fasoracetam

An early placebo-first/open ADHD study reported improvement with fasoracetam. Three later randomized ADHD trials, a 22q11.2 crossover, and an earlier Japanese phase 3 program did not confirm a clinical benefit.

Small-molecule investigational racetam

ASCEND Parts A and B both missed their six-week primary endpoint; in genetically enriched Part A, the ADHD score numerically improved more with placebo.
The 37-person 22q11.2 crossover found no significant whole-sample global, anxiety, ADHD or social-responsiveness benefit.
Healthy adults were studied for pharmacokinetics, not cognitive enhancement.
Older men had higher exposure and lower clearance after the same 100 mg dose in a tiny PK comparison.

What is fasoracetam?

Fasoracetam is the compound formerly called NS-105 and later NFC-1, AEVI-001 and NB-001. Later trials used fasoracetam-monohydrate capsules.

The clinical program spans healthy PK work, an abandoned Japanese cerebrovascular/dementia program, pediatric ADHD studies and a 22q11.2-deletion trial. Those populations and formulations answer different questions.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

PubChem: fasoracetam chemical identity

Government chemical identity database

National Library of Medicine. PubChem CID 198695.

C10H16N2O2; molecular weight 196.25 g/mol.

Participants / model: Chemical record
Follow-up: Living database
Study design: Curated structure record

Formula and molecular weight establish the recorded structure, not the contents of a retail product or an effective dose.

Read the original source

SAGA protocol: Study of AEVI-001 in adolescents with ADHD and mGluR-network variants.

Primary sponsor protocol

Aevi Genomic Medicine. NCT02777931 sponsor protocol, sections 5.2 and 9.7.

The protocol states that earlier Japanese phase 3 development ended after NS-105 failed defined efficacy endpoints and summarizes unpublished PK and safety history.

Participants / model: Historical healthy-volunteer and cerebrovascular/dementia development cohorts; later SAGA adolescents
Follow-up: Historical program plus six-week SAGA plan
Study design: Sponsor protocol containing prior-program summary

The protocol reports the phase 3 failure without enrollment or a numerical result, so the outcome cannot be quantified.

Read the original source

What did the controlled ADHD program find?

The early 30-person study reported improvement after everyone switched from one week of placebo to open active treatment. Later randomized studies did not establish the primary benefit.

SAGA randomized 101 genetically selected adolescents. ADHD-RS change was −14.2 with fasoracetam versus −12.1 with placebo, while CGI-I response was 26/46 versus 16/50; the posted record supplies no between-group test settling either co-primary outcome. Only 37/49 active and 39/52 placebo participants completed.

ASCEND Part A analyzed 67 genetically selected children/adolescents: ADHD-RS change was −9.17 with drug versus −11.34 with placebo. Part B analyzed 106 without the selected variants: −11.04 versus −10.38. The sponsor reported that both parts missed the primary endpoint.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD

Trial registry with posted results

ClinicalTrials.gov. NCT02777931. Posted results.

ADHD-RS least-squares mean change −14.2 with drug versus −12.1 with placebo; the posted result does not supply a between-group p value.

Participants / model: 101 randomized adolescents; 96 in the modified intention-to-treat analysis
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Efficacy groups were 46 drug and 50 placebo. This is distinct from the earlier 30-person study and the later ASCEND trials.

Read the original source

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03265119. Posted results.

ADHD-RS change −9.17 with drug versus −11.34 with placebo.

Participants / model: 69 enrolled; 67 in efficacy analysis, 33 drug and 34 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

One serious irritability event was posted in the active safety group. Sponsor reported failure of the primary endpoint.

Read the original source

PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03609619. Posted results.

ADHD-RS change −11.04 with drug versus −10.38 with placebo.

Participants / model: 109 enrolled; 106 in efficacy analysis, 52 drug and 54 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Sponsor reported failure of the primary endpoint. Do not pool enrolled, efficacy and safety denominators.

Read the original source

ASCEND sponsor results, January 2019

Primary sponsor disclosure

Aevi Genomic Medicine. 2 January 2019. Sponsor-issued release carried by PR Newswire.

Neither ASCEND part met its primary ADHD-RS endpoint at six weeks.

Participants / model: Children with ADHD in ASCEND Parts A and B
Follow-up: 6 weeks
Study design: Top-line disclosure for two randomized controlled trial cohorts
Funding: Issued by the developer, Aevi Genomic Medicine.

A sponsor statement, not a peer-reviewed paper. The trial registries report the numerical results.

Read the original source

What happened in the 22q11.2 deletion trial?

The randomized crossover did not establish a whole-sample clinical benefit. Thirty-seven youth were randomized, 32 completed, and 33 entered the efficacy analysis.

CGI-I least-squares means were 3.34 with fasoracetam and 3.69 with placebo (p=0.0672). CGI-S, anxiety, ADHD inattention, ADHD hyperactivity and social responsiveness were all nonsignificant.

The 2025 conference abstract reports this same cohort, not a replication. Nominal subgroup findings do not override the registered whole-sample nulls.

NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

Trial registry with posted results

ClinicalTrials.gov. NCT05290493. Posted results.

CGI-I 3.34 with drug versus 3.69 with placebo, p=0.0672; secondary anxiety, ADHD and social-responsiveness comparisons were also nonsignificant.

Participants / model: 37 children randomized; 32 completed; efficacy set 33
Follow-up: 6-week treatment periods
Study design: Quadruple-masked crossover: six weeks per treatment, separated by one week of washout
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Safety was the primary outcome. Treatment-emergent events occurred in 18/33 drug exposures and 24/34 placebo exposures, with no serious events. The same participants crossed over; these are not independent groups.

Read the original source

343. Fasoracetam for Neuropsychiatric Symptoms in Children and Adolescents With 22q11.2 Deletion Syndrome: A Randomized Crossover Phase II Clinical Trial

Primary conference abstract

Baribeau D, Chadehumbe M, Hopkins S, et al. Biological Psychiatry. 2025. DOI 10.1016/j.biopsych.2025.02.581.

The report describes the same 37-person 22q11.2 crossover already posted in the registry; whole-sample clinical outcomes remained nonsignificant.

Participants / model: Youth aged 6 to 17 with pathogenic 22q11.2 deletion
Follow-up: Six weeks per treatment with one-week washout
Study design: Conference report of randomized placebo crossover

Exploratory subgroup p values were not shown to be multiplicity-controlled and are not independent replication.

Read the original source

What happened in the original NS-105 program?

A later sponsor protocol says Japanese phase 3 development in cerebrovascular disease/dementia stopped because the trial failed its defined efficacy endpoints.

The same protocol summarizes earlier healthy phase 1 and phase 2 cohorts of 169 and 288 patients. It reports only that the Japanese phase 3 program failed, without enrollment or a numerical result, so no effect size can be assigned.

SAGA protocol: Study of AEVI-001 in adolescents with ADHD and mGluR-network variants.

Primary sponsor protocol

Aevi Genomic Medicine. NCT02777931 sponsor protocol, sections 5.2 and 9.7.

The protocol states that earlier Japanese phase 3 development ended after NS-105 failed defined efficacy endpoints and summarizes unpublished PK and safety history.

Participants / model: Historical healthy-volunteer and cerebrovascular/dementia development cohorts; later SAGA adolescents
Follow-up: Historical program plus six-week SAGA plan
Study design: Sponsor protocol containing prior-program summary

The protocol reports the phase 3 failure without enrollment or a numerical result, so the outcome cannot be quantified.

Read the original source

Did glutamate-related genetics identify responders?

No validated treatment-selection test emerged. The mGluR-network hypothesis guided enrollment, but the enriched ASCEND Part A point estimate favored placebo on the primary ADHD scale.

Animal studies show activity in scopolamine, lesion, ischemia and other induced-amnesia models. They do not establish attention or memory improvement in a healthy human.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03265119. Posted results.

ADHD-RS change −9.17 with drug versus −11.34 with placebo.

Participants / model: 69 enrolled; 67 in efficacy analysis, 33 drug and 34 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

One serious irritability event was posted in the active safety group. Sponsor reported failure of the primary endpoint.

Read the original source

Involvement of cholinergic and GABAergic systems in the reversal of memory disruption by NS-105, a cognition enhancer.

Preclinical primary study

Ogasawara T, Itoh Y, Tamura M, Mushiroi T, Ukai Y, Kise M, Kimura K. Pharmacology Biochemistry and Behavior. 1999. PMID 10494996. DOI 10.1016/S0091-3057(99)00108-2.

Fasoracetam improved selected outcomes across several induced-impairment rat models and altered cholinergic measures.

Participants / model: Rats with pharmacologic, lesion, ischemia or electroconvulsive-shock impairment
Follow-up: Acute or repeated animal exposures
Study design: Controlled induced-impairment experiments

Every positive memory model began with induced impairment; this does not show healthy-human enhancement.

Read the original source

Does this support adult ADHD, studying or brain-fog claims?

No. The efficacy studies enrolled children or adolescents with diagnosed ADHD or 22q11.2 deletion. The cited studies include no healthy-rested cognition trial or adult ADHD efficacy trial.

Healthy volunteers contributed exposure data only. No human sleep-loss, strength, endurance or longevity outcome was found.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD

Trial registry with posted results

ClinicalTrials.gov. NCT02777931. Posted results.

ADHD-RS least-squares mean change −14.2 with drug versus −12.1 with placebo; the posted result does not supply a between-group p value.

Participants / model: 101 randomized adolescents; 96 in the modified intention-to-treat analysis
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Efficacy groups were 46 drug and 50 placebo. This is distinct from the earlier 30-person study and the later ASCEND trials.

Read the original source

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03265119. Posted results.

ADHD-RS change −9.17 with drug versus −11.34 with placebo.

Participants / model: 69 enrolled; 67 in efficacy analysis, 33 drug and 34 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

One serious irritability event was posted in the active safety group. Sponsor reported failure of the primary endpoint.

Read the original source

PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03609619. Posted results.

ADHD-RS change −11.04 with drug versus −10.38 with placebo.

Participants / model: 109 enrolled; 106 in efficacy analysis, 52 drug and 54 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Sponsor reported failure of the primary endpoint. Do not pool enrolled, efficacy and safety denominators.

Read the original source

NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

Trial registry with posted results

ClinicalTrials.gov. NCT05290493. Posted results.

CGI-I 3.34 with drug versus 3.69 with placebo, p=0.0672; secondary anxiety, ADHD and social-responsiveness comparisons were also nonsignificant.

Participants / model: 37 children randomized; 32 completed; efficacy set 33
Follow-up: 6-week treatment periods
Study design: Quadruple-masked crossover: six weeks per treatment, separated by one week of washout
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Safety was the primary outcome. Treatment-emergent events occurred in 18/33 drug exposures and 24/34 placebo exposures, with no serious events. The same participants crossed over; these are not independent groups.

Read the original source

What is known about pharmacokinetics?

In adolescents, single-dose peak concentrations occurred around 1.3 to 1.9 hours and pooled mean half-life was 4.82 hours. Tiny adult data show age-related exposure differences.

After 100 mg, seven older men had higher Cmax and AUC and lower total clearance than seven younger men; renal clearance correlated with creatinine clearance. A four-to-five-hour half-life does not define focus duration or a healthy-use schedule.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

Comparison of pharmacokinetics of NS-105, a novel agent for cerebrovascular disease, in elderly and young subjects.

Primary human pharmacokinetic study

Kumagai Y et al. International Journal of Clinical Pharmacology Research. 1999. PMID 10450537.

Older men had higher peak concentration and exposure and lower total clearance after 100 mg; renal clearance correlated with creatinine clearance.

Participants / model: Seven healthy men aged 68 to 79 and seven aged 20 to 32; one young participant excluded from urine analysis
Follow-up: Single oral dose after breakfast
Study design: Parallel age-group pharmacokinetic study

The groups were tiny and all male; this measured exposure, not cognition or clinical benefit.

Read the original source

SAGA protocol: Study of AEVI-001 in adolescents with ADHD and mGluR-network variants.

Primary sponsor protocol

Aevi Genomic Medicine. NCT02777931 sponsor protocol, sections 5.2 and 9.7.

The protocol states that earlier Japanese phase 3 development ended after NS-105 failed defined efficacy endpoints and summarizes unpublished PK and safety history.

Participants / model: Historical healthy-volunteer and cerebrovascular/dementia development cohorts; later SAGA adolescents
Follow-up: Historical program plus six-week SAGA plan
Study design: Sponsor protocol containing prior-program summary

The protocol reports the phase 3 failure without enrollment or a numerical result, so the outcome cannot be quantified.

Read the original source

What did the clinical program report about risk?

Headache and fatigue were common in the early study, and short pediatric trials cannot settle uncommon or chronic risks.

In the 30-person study, headache occurred in 19 and fatigue in 11; three serious events were judged unrelated. SAGA posted no serious events; ASCEND Part A posted one serious irritability event in the active arm; the 22q crossover posted no serious event.

The historical sponsor summary mentions laboratory elevations and two phase 3 serious events of unknown relationship, but does not report enough detail for a complete pooled safety analysis.

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD

Trial registry with posted results

ClinicalTrials.gov. NCT02777931. Posted results.

ADHD-RS least-squares mean change −14.2 with drug versus −12.1 with placebo; the posted result does not supply a between-group p value.

Participants / model: 101 randomized adolescents; 96 in the modified intention-to-treat analysis
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Efficacy groups were 46 drug and 50 placebo. This is distinct from the earlier 30-person study and the later ASCEND trials.

Read the original source

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03265119. Posted results.

ADHD-RS change −9.17 with drug versus −11.34 with placebo.

Participants / model: 69 enrolled; 67 in efficacy analysis, 33 drug and 34 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

One serious irritability event was posted in the active safety group. Sponsor reported failure of the primary endpoint.

Read the original source

PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03609619. Posted results.

ADHD-RS change −11.04 with drug versus −10.38 with placebo.

Participants / model: 109 enrolled; 106 in efficacy analysis, 52 drug and 54 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Sponsor reported failure of the primary endpoint. Do not pool enrolled, efficacy and safety denominators.

Read the original source

NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

Trial registry with posted results

ClinicalTrials.gov. NCT05290493. Posted results.

CGI-I 3.34 with drug versus 3.69 with placebo, p=0.0672; secondary anxiety, ADHD and social-responsiveness comparisons were also nonsignificant.

Participants / model: 37 children randomized; 32 completed; efficacy set 33
Follow-up: 6-week treatment periods
Study design: Quadruple-masked crossover: six weeks per treatment, separated by one week of washout
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Safety was the primary outcome. Treatment-emergent events occurred in 18/33 drug exposures and 24/34 placebo exposures, with no serious events. The same participants crossed over; these are not independent groups.

Read the original source

SAGA protocol: Study of AEVI-001 in adolescents with ADHD and mGluR-network variants.

Primary sponsor protocol

Aevi Genomic Medicine. NCT02777931 sponsor protocol, sections 5.2 and 9.7.

The protocol states that earlier Japanese phase 3 development ended after NS-105 failed defined efficacy endpoints and summarizes unpublished PK and safety history.

Participants / model: Historical healthy-volunteer and cerebrovascular/dementia development cohorts; later SAGA adolescents
Follow-up: Historical program plus six-week SAGA plan
Study design: Sponsor protocol containing prior-program summary

The protocol reports the phase 3 failure without enrollment or a numerical result, so the outcome cannot be quantified.

Read the original source

Studies and sources

Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling.

Primary human study

Elia J et al. Nature communications. 2018. PMID 29339723. DOI 10.1038/s41467-017-02244-2.

Exploratory ADHD ratings improved in a sequential placebo-first study; the primary aims were safety and pharmacokinetics.

Participants / model: 30 adolescents aged 12 to 17 with ADHD and selected genetic variants
Follow-up: 5 weeks
Study design: Single-blind fixed placebo week followed by open dose escalation; genotype status blinded
Funding: Funded by neuroFix Therapeutics. Senior author founded neuroFix and held equity; the paper describes independent clinical evaluators and statistical analysis.

30 enrolled, 29 completed. No concurrent randomized placebo arm. Three serious events occurred; authors did not attribute them to treatment. Common events included headache in 19/30 and fatigue in 11/30.

Read the original source

PubChem: fasoracetam chemical identity

Government chemical identity database

National Library of Medicine. PubChem CID 198695.

C10H16N2O2; molecular weight 196.25 g/mol.

Participants / model: Chemical record
Follow-up: Living database
Study design: Curated structure record

Formula and molecular weight establish the recorded structure, not the contents of a retail product or an effective dose.

Read the original source

Efficacy and Safety of NFC-1 in Adolescents With Genetic Disorders Impacting mGluR and ADHD

Trial registry with posted results

ClinicalTrials.gov. NCT02777931. Posted results.

ADHD-RS least-squares mean change −14.2 with drug versus −12.1 with placebo; the posted result does not supply a between-group p value.

Participants / model: 101 randomized adolescents; 96 in the modified intention-to-treat analysis
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Efficacy groups were 46 drug and 50 placebo. This is distinct from the earlier 30-person study and the later ASCEND trials.

Read the original source

PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03265119. Posted results.

ADHD-RS change −9.17 with drug versus −11.34 with placebo.

Participants / model: 69 enrolled; 67 in efficacy analysis, 33 drug and 34 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

One serious irritability event was posted in the active safety group. Sponsor reported failure of the primary endpoint.

Read the original source

PART B: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and Without mGluR Mutations

Trial registry with posted results

ClinicalTrials.gov. NCT03609619. Posted results.

ADHD-RS change −11.04 with drug versus −10.38 with placebo.

Participants / model: 109 enrolled; 106 in efficacy analysis, 52 drug and 54 placebo
Follow-up: 6-week treatment periods
Study design: Randomized, double-blind, parallel placebo-controlled trial
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Sponsor reported failure of the primary endpoint. Do not pool enrolled, efficacy and safety denominators.

Read the original source

NB-001 in Children and Adolescents With 22q11 Deletion Syndrome

Trial registry with posted results

ClinicalTrials.gov. NCT05290493. Posted results.

CGI-I 3.34 with drug versus 3.69 with placebo, p=0.0672; secondary anxiety, ADHD and social-responsiveness comparisons were also nonsignificant.

Participants / model: 37 children randomized; 32 completed; efficacy set 33
Follow-up: 6-week treatment periods
Study design: Quadruple-masked crossover: six weeks per treatment, separated by one week of washout
Funding: Industry-sponsored trial; sponsor-submitted registry results.

Safety was the primary outcome. Treatment-emergent events occurred in 18/33 drug exposures and 24/34 placebo exposures, with no serious events. The same participants crossed over; these are not independent groups.

Read the original source

Comparison of pharmacokinetics of NS-105, a novel agent for cerebrovascular disease, in elderly and young subjects.

Primary human pharmacokinetic study

Kumagai Y et al. International Journal of Clinical Pharmacology Research. 1999. PMID 10450537.

Older men had higher peak concentration and exposure and lower total clearance after 100 mg; renal clearance correlated with creatinine clearance.

Participants / model: Seven healthy men aged 68 to 79 and seven aged 20 to 32; one young participant excluded from urine analysis
Follow-up: Single oral dose after breakfast
Study design: Parallel age-group pharmacokinetic study

The groups were tiny and all male; this measured exposure, not cognition or clinical benefit.

Read the original source

SAGA protocol: Study of AEVI-001 in adolescents with ADHD and mGluR-network variants.

Primary sponsor protocol

Aevi Genomic Medicine. NCT02777931 sponsor protocol, sections 5.2 and 9.7.

The protocol states that earlier Japanese phase 3 development ended after NS-105 failed defined efficacy endpoints and summarizes unpublished PK and safety history.

Participants / model: Historical healthy-volunteer and cerebrovascular/dementia development cohorts; later SAGA adolescents
Follow-up: Historical program plus six-week SAGA plan
Study design: Sponsor protocol containing prior-program summary

The protocol reports the phase 3 failure without enrollment or a numerical result, so the outcome cannot be quantified.

Read the original source

343. Fasoracetam for Neuropsychiatric Symptoms in Children and Adolescents With 22q11.2 Deletion Syndrome: A Randomized Crossover Phase II Clinical Trial

Primary conference abstract

Baribeau D, Chadehumbe M, Hopkins S, et al. Biological Psychiatry. 2025. DOI 10.1016/j.biopsych.2025.02.581.

The report describes the same 37-person 22q11.2 crossover already posted in the registry; whole-sample clinical outcomes remained nonsignificant.

Participants / model: Youth aged 6 to 17 with pathogenic 22q11.2 deletion
Follow-up: Six weeks per treatment with one-week washout
Study design: Conference report of randomized placebo crossover

Exploratory subgroup p values were not shown to be multiplicity-controlled and are not independent replication.

Read the original source

Involvement of cholinergic and GABAergic systems in the reversal of memory disruption by NS-105, a cognition enhancer.

Preclinical primary study

Ogasawara T, Itoh Y, Tamura M, Mushiroi T, Ukai Y, Kise M, Kimura K. Pharmacology Biochemistry and Behavior. 1999. PMID 10494996. DOI 10.1016/S0091-3057(99)00108-2.

Fasoracetam improved selected outcomes across several induced-impairment rat models and altered cholinergic measures.

Participants / model: Rats with pharmacologic, lesion, ischemia or electroconvulsive-shock impairment
Follow-up: Acute or repeated animal exposures
Study design: Controlled induced-impairment experiments

Every positive memory model began with induced impairment; this does not show healthy-human enhancement.

Read the original source

Why is fasoracetam in D tier?

D reflects one weak pediatric ADHD signal followed by a wider controlled program that did not establish efficacy. Healthy cognition, adult focus, anxiety, sleep performance, and longevity remain untested or unestablished.