UnknownPhase 2

TXA127 in Non-Ambulant Patients With DMD Cardiomyopathy

Israel10 participantsStarted 2023-08-31

Plain-language summary

This open-label, single-arm multi-center study studying the safety and efficacy of TXA127 on non-ambulant patients with DMD Cardiomyopathy will comprise of two phases: 1. 6-month open-label treatment phase: Male DMD patients with documented cardiomyopathy, will receive a daily subcutaneous injection of TXA127 0.5 mg/kg. Treatment will be provided for 6 months. Treatment safety will be assessed by collection and review of AEs, vital signs, ECGs, physical examinations, PFTs, and laboratory parameters on Day 1, Month 1, and Month 6. Ejection Fraction, upper extremity strength and biomarker levels will be assessed at these study visits as well. In addition, an abbreviated safety visit will be conducted at Month 3. 2. 12-month optional extension phase: Patients will continue the same study drug regime for an additional 12 months. The primary objective of this phase is to obtain long-term safety data. Efficacy data will also be collected. Safety, efficacy, and exploratory biomarkers will be assessed at Month 12 and Month 18, using the same methods as in the treatment phase. In addition, abbreviated safety visits will be conducted at Month 9 and Month 15.

Who can participate

Age range16 Years

SexMALE

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Inclusion criteria

✓. Male subjects 16 years of age or older who provide informed consent and can follow up with protocol procedures. Parental or guardian consent is required for subjects at least 16 years of age but younger than 18 years of age.
✓. Documented diagnosis of Duchenne muscular dystrophy by genetic mutation analysis.
✓. Documented cardiomyopathy, as assessed by echocardiogram with:
✓. For a patient ≤ 20 years of age, EF \> 35% and \< 55% and fractional shortening of ≤ 28% at the time of screening.
✓. For a patient \> 20 years of age, EF \> 20% and fractional shortening ≤ 28% at the time of screening.
✓. Reproducible (+/- 10%) difference between screening and baseline of percent predicted FVC , using the best out of 3 efforts at each visit:
✓. For a patient ≤ 20 years of age, FVC between 45% and 85%, inclusive. Patient should not utilize non-invasive ventilation such as CPAP or BiPAP.
✓. For a patient \>20 years of age, all of the following should exist: FVC \> 20%, EF \> 20% in baseline ECHO and ability to be off non-invasive ventilation, such as CPAP and BiPAP, for at least 4 consecutive hours a day (24 hours period).

Exclusion criteria

What they're measuring

To evaluate the safety of TXA127 in patients with DMD

Timeframe: 6 months plus 12 month extension

To evaluate the effects of treatment on ejection fraction (EF)

Timeframe: 6 months plus 12 month extension

Trial details

NCT IDNCT06013839

SponsorConstant Therapeutics LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08

Contact for this trial

Richard L Franklin, MD, PhD

1-617-245-0289 rfranklin@constanttherapeutics.com

View on ClinicalTrials.gov More DMD-Associated Dilated Cardiomyopathy trials