Phase 3 Spinal Muscular Atrophy Trial, Recruiting NCT06862596 Sponsor: Masahisa Katsuno Condition: Spinal Muscular Atrophy
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Phase 3 Spinal Muscular Atrophy Trial, Recruiting

NCT06862596
Recruiting Phase 3

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:

  • You are a male with a confirmed diagnosis of SBMA (Spinal and Bulbar Muscular Atrophy), confirmed by genetic testing showing a specific gene repeat count of 38 or more
  • You have muscle weakness in your arms or legs, muscle wasting, or weakness in the muscles used for swallowing or speaking, caused by damage to the lower motor nerves
  • Your overall physical function score (measured by a standard scale called ALSFRS-R) falls within a specific range — between 24 and 42 — at the time of screening
  • You are aged 18 or older but under 80 years old at the time of giving consent
  • You are willing and able to give written consent to take part in the study (if you are unable to sign due to your condition, a legal representative may sign on your behalf with your verbal agreement)

Who may not be able to join:

  • You have taken part in, or are currently taking part in, another clinical trial within the past 12 weeks
  • You have had an allergic reaction to any ingredient in the study drug
  • You have certain heart rhythm conduction problems, such as second- or third-degree heart block without a pacemaker, or a specific electrical pattern in the heart called left bundle branch block
  • You have a specific heart rhythm pattern on an ECG known as Brugada-type pattern
  • You have severe heart failure or serious heart conditions such as a previous heart attack, heart valve disease, or disease of the heart muscle
  • Your resting heart rate is slower than 50 beats per minute
  • Your systolic (upper number) blood pressure is 90 mmHg or below
  • Your blood potassium level is below 3.5 mmol/L
  • You are currently taking medicines to control heart rhythm (antiarrhythmic drugs)
  • You are currently taking certain epilepsy medicines that affect sodium channels in the body (confirm with trial site)
  • You are currently taking a medicine called theophylline (often used for breathing conditions)
  • You are currently taking narcotic (opioid) medicines
  • You have used a medicine called Mexiletine within the past month, or have used it specifically to try to manage SBMA symptoms
  • You have other serious health complications (confirm with trial site for specifics)
  • You are unwilling or unable to use contraception throughout the study period
  • The trial doctor considers you unsuitable for any other reason

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 3: approximately ~65% 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

Principal Investigator: Masahisa Katsuno, PhD, MD, Nagoya University Hospital

Phone: +81527442389

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 3
Sponsor
Masahisa Katsuno
Registry
ClinicalTrials.gov
Start date
28 February 2025
Est. completion
31 December 2027

Where this trial is recruiting

🇯🇵 Japan

Primary endpoints

Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score

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