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Sarepta Therapeutics clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, Ireland, India, China and EU (CTIS) registers with Sarepta Therapeutics as lead sponsor. Synced 26 Sep 2026
13Current trials
3Recruiting now
2Drugs in these trials
6Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Sarepta Therapeutics is the lead sponsor of 13 current clinical trials in our USA, UK, Ireland, India, China and EU (CTIS) registers. 3 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Eteplirsen and Sirolimus. The most-studied condition is Duchenne Muscular Dystrophy.

ClinicalTrials.gov trials by status

  • Recruiting325%
  • Not yet open18%
  • Active, closed542%
  • By invitation325%

Trials by phase

  • Phase 3433%
  • Phase 1325%
  • Observational325%
  • Phase 1/218%
  • Phase 418%
01

Clinical trials sponsored by Sarepta Therapeutics

12 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's Disease
NCT07536061 · ClinicalTrials.gov ↗ · Huntington's Disease
Phase 1 Recruiting 2026-05 UK
Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
NCT06138743 · ClinicalTrials.gov ↗ · Myotonic Dystrophy 1
Phase 1/2 Recruiting 2024-03 UK, China
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
NCT04626674 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Phase 1 Recruiting 2020-11 USA
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
NCT07542314 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Drug: Sirolimus
Phase 4 Not yet open 2026-08 USA
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
NCT05967351 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Phase 3 By invitation 2023-09 UK, USA
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
NCT06606340 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Observational By invitation 2019-01 USA
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
NCT06270719 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Observational By invitation 2024-02 USA
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
NCT03992430 · ClinicalTrials.gov ↗ · Muscular Dystrophy, Duchenne
Phase 3 Active, closed 2020-07 Ireland, UK, USA, India
A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice
NCT04475926 · ClinicalTrials.gov ↗ · Limb-girdle Muscular Dystrophy
Observational Active, closed 2021-04 UK, USA
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
NCT06246513 · ClinicalTrials.gov ↗ · Limb-girdle Muscular Dystrophy
Phase 3 Active, closed 2024-01 UK, USA
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
NCT05881408 · ClinicalTrials.gov ↗ · Duchenne Muscular Dystrophy
Phase 3 Active, closed 2023-05 UK, USA
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
NCT05876780 · ClinicalTrials.gov ↗ · Limb Girdle Muscular Dystrophy
Phase 1 Active, closed 2022-12 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Sarepta Therapeutics in the EU trials register (CTIS)

1 trial

Authorised or ongoing trials in the EU Clinical Trials Information System with Sarepta Therapeutics as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who are ≥18 to ≤65 Years
2024-513579-42-00 · CTIS ↗ · Nervous System Diseases
Phase 1 Ongoing Spain, Germany, Belgium + 1 more
03

Drugs in Sarepta Therapeutics trials

2 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Sarepta Therapeutics trials

6 conditions
Duchenne Muscular Dystrophy6Limb-girdle Muscular Dystrophy2Muscular Dystrophy, Duchenne1Huntington's Disease1Myotonic Dystrophy 11Limb Girdle Muscular Dystrophy1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA101
Clinical trials in UK72
Clinical trials in Ireland10
Clinical trials in India10
Clinical trials in China11
EU register (CTIS)1—

A trial with sites in more than one country appears in each of those registers.

06

Other sponsors in Duchenne Muscular Dystrophy

SponsorTrials
Italfarmaco3
Solid Biosciences3
NS Pharma3
REGENXBIO3
Edgewise Therapeutics2
Wave Life Sciences USA2

Sponsors with the most current trials registered for this condition.

07

Questions about Sarepta Therapeutics trials

How many clinical trials does Sarepta Therapeutics sponsor?

Sarepta Therapeutics is the lead sponsor of 12 current trials on ClinicalTrials.gov in our USA, UK, Ireland, India and China registers, and 1 authorised or ongoing trial in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Sarepta Therapeutics trials recruiting?

Yes. 3 of its 12 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Sarepta Therapeutics testing?

Its current trials name 2 drugs and biologicals as interventions. The most frequent are Eteplirsen (2) and Sirolimus (1). The number in brackets is how many of its trials name each drug.

Does Sarepta Therapeutics have clinical trials in Ireland?

Yes. 1 trial in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.