Phase 1 Motor Neurone Disease Trial, Recruiting NCT07290062 Sponsor: Insmed Gene Therapy LLC Condition: Motor Neurone Disease
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Phase 1 Motor Neurone Disease Trial, Recruiting

NCT07290062
Recruiting Phase 1

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • Your Body Mass Index (BMI) — a measure of body weight relative to height — must be 18 or above.
  • You must have been diagnosed with ALS (also known as Lou Gehrig's disease) with symptoms, confirmed using a specific set of diagnostic guidelines called the Gold Coast criteria.
  • For participants without a family history of ALS: you must have tested negative for known gene changes that are linked to inherited (familial) ALS.
  • For a specific group in this trial (Cohorts 2 and 3 only): you must have a confirmed change in a gene called SOD1 that is known to cause ALS, and must have tested negative for other gene changes linked to inherited ALS.
  • Any other gene variations found may need to be reviewed by the trial organisers to check if you are still eligible (confirm with trial site).
  • Your score on a standard ALS symptom and function rating scale (called the ALSFRS-R) must be 24 or higher at the start of the trial.
  • You must have been living with ALS for 42 months (3.5 years) or less since your symptoms began.

Who may not be able to join:

  • You cannot join if you have previously received cell therapy or gene therapy as a treatment for ALS.
  • You cannot join if you are currently taking or have recently taken any experimental medication or been part of another clinical trial, whether for ALS or any other condition.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 1: approximately ~10% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Phone: 1-844-446-7633

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 1
Sponsor
Insmed Gene Therapy LLC
Registry
ClinicalTrials.gov
Start date
9 January 2026
Est. completion
31 March 2030

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov