ClinicalTrials.gov record NCT07439510

A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Oct 10, 2026

A Phase 2b/3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Codabakalner in Male Participants With Full Mutation Fragile X Syndrome and Adult Participants With Autism Spectrum Disorders

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2/3A study that tests whether a treatment works while continuing to check safety, in a larger group than a phase 2 study alone. It combines the aims of phase 2 and phase 3 in one protocol.
Age18 years old to 45 years old
SexMale
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2b/3 randomized, double-blind, placebo-controlled study evaluates codabakalner in male participants with Fragile X syndrome and adult participants with autism spectrum disorder. Phase 2b is a 4-arm, dose-regimen finding stage with 4 weeks of daily treatment and 4 weeks of follow-up. Phase 3 is a 2-arm stage using the dose chosen from Phase 2b, with 12 weeks of daily treatment and 4 weeks of follow-up.

What it is studying. The study is measuring the effects, safety, tolerability, and blood pharmacokinetics of codabakalner compared with placebo. Phase 2b compares multiple dose regimens to identify a dose for Phase 3; Phase 3 tests that selected dose over a longer treatment period. Outcomes include cognitive tests, adaptive behavior measures, clinician and caregiver ratings, EEG measures, safety events, and drug levels in blood.

What the researchers hope to learn. To identify suitable dose regimens of codabakalner, characterize its safety and tolerability, measure pharmacokinetic properties, and assess its effects on cognition, adaptive behavior, EEG measures, and clinician/caregiver-reported symptoms in FXS and ASD.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a Study to Investigate the Effects and Safety of Codabakalner for the Treatment of Fragile X Syndrome in Male Participants and Adult Participants with Autism Spectrum Disorders

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Codabakalner
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Phase 2b: Adult males aged 18 to 45 years, inclusive Phase 3: Males aged 13-45 years, inclusive Mental age >/= 36mon
  • Diagnosis of Fragile X as confirmed with genetic testing and Stanford Binet testing or
  • Confirmed diagnosis ASD per DSM5-TR and EEG criteria
  • Patient must have caregiver
  • Must be in good health with no significant medical history

Exclusion criteria

  • Any physical or psychological condition that prohibits study completion
  • Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months.
  • Auditory or visual impairments that cannot be corrected
  • History of suicidal behaviour or suicidal ideation
  • Screening vital signs that are abnormal per protocol specification
  • ECG that are abnormal and clinically significant
  • History of substance abuse or dependence within 6 months
  • Other investigational products within 30 days
  • Unable to swallow capsules

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This Phase 2b/3, randomized, double-blind, placebo-controlled, 2-part study will evaluate the efficacy, safety and tolerability of different dose regimens of codabakalner in adult male participants with Fragile X syndrome. The results from Phase 2b will be hypothesis generating and will inform the Phase 3 design. Phase 2b, dose-regimen finding, 4-arm parallel design: Eligible participants will be randomized 1:1:1:1 to receive codabakalner at different doses or matched placebo. Participants will receive study intervention over 4 weeks and will be followed up for 4 weeks after last dose of study intervention. Phase 3, 2-arm parallel design: Eligible participants will be randomized 1:1 to receive codabakalner or matched placebo. The dose of codabakalner administered in the Phase 3 will be defined based on analysis of Phase 2b. Participants will receive study intervention over 12 weeks and will be followed up for 4 weeks after last dose of study intervention.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

If you join, you will be randomized to receive daily study medication (codabakalner) or matched placebo; Phase 2b dosing is given for 4 weeks with a 4-week follow-up, and Phase 3 dosing is given for 12 weeks with a 4-week follow-up. During study visits you will complete cognitive testing (NIH Toolbox), adaptive behavior assessments (Vineland-III), clinician ratings (CGI-S and CGI-I), caregiver questionnaires and numerical rating scales, and suicidality assessments (C-SSRS). The study also includes EEG resting-state measurements, measurements of codabakalner concentrations in plasma, and monitoring for adverse events and serious adverse events. The registry lists "unable to swallow capsules" as an exclusion, so participants must be able to swallow capsules to take the study medication.

How the study is structured. Participants are randomized and quadruple-masked to receive either codabakalner or matching placebo. Phase 2b uses a 4-arm parallel design (multiple codabakalner dose regimens and placebo) with daily dosing for 4 weeks and a 4-week follow-up. Phase 3 uses a 2-arm parallel design (selected codabakalner dose versus placebo) with daily dosing for 12 weeks and a 4-week follow-up. During the study participants complete cognitive testing (NIH Toolbox), adaptive behavior scales (Vineland), clinician and caregiver questionnaires, EEG recordings, safety assessments, and blood sampling for pharmacokinetics as described in the registry.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on fragile X syndrome

Clinical trials20 trials listed for fragile X syndromeEvery trial the registry lists for fragile X syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in fragile X syndrome researchLatest monthly update: September 2026. Includes: Multiple behavior challenges. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 1, 2026 (estimated)
Primary completion
Feb 1, 2030 (estimated)
Study completion
Jun 30, 2030 (estimated)
Target enrollment
120 participants (estimated)
Last updated on registry
Oct 9, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Spinogenix

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

Phase 3
A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
ECG
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
1:1:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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