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Duchenne Muscular DystrophyPhase 2MOMENTUMNCT04004065

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

A Phase 2 study testing Vesleteplirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 25 sites in 8 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Read more about this study

Who is it for?
Ages 7 to 21 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Vesleteplirsen
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
62 participants
Where
25 sitesin 8 countries
Study dates
26 Jun 2019to 7 Feb 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 7 to 21 years
  • Men only
  • No healthy volunteers
  • Child, Adult

Inclusion Criteria for participants previously treated with Vesleteplirsen:

  • Has received prior Vesleteplirsen treatment in Part A of this study or in Study 5051-102.

Exclusion Criteria for participants previously treated with Vesleteplirsen and new participants enrolling into Part B:

  • Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any other condition that, in the Investigator's opinion, could interfere with participation in the trial.

Inclusion Criteria for treatment-naïve participants enrolling into Part B:

  • Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
  • Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration.
  • Has stable pulmonary function (forced vital capacity [FVC] ≥40% of predicted and no requirement for nocturnal ventilation).

Exclusion Criteria for treatment-naive participants enrolling into Part B:

  • History of hypomagnesemia within 12 weeks prior to Screening.
  • Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
  • Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
  • Has a left ventricular ejection fraction (LVEF) <40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit.
  • Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.

Other inclusion/exclusion criteria apply.

Trial locations and contacts

25 sites in 8 countries.

25 sites in 8 countries

United States11 sites

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30318

  • Austin Neuromuscular Center

    Austin, Texas, 78756

  • Children's Health Ambulatory Pavilion

    Dallas, Texas, 75207

  • Connecticut Children's

    Farmington, Connecticut, 06032

  • Northwest Florida Clinical Research Group, LLC

    Gulf Breeze, Florida, 32561

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • University of Kansas Medical Center Research Inst.

    Kansas City, Kansas, 66103

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • University of California Davis Health

    Sacramento, California, 95817

  • Seattle Children's

    Seattle, Washington, 98105

  • University of Massachusetts

    Worcester, Massachusetts, 01655

Other trials in United States

United Kingdom4 sites

  • Royal Hospital for Children (Glasgow)

    Glasgow, G51 4TF

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, Lancashire, L12 2AP

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3JH

  • Oxford University Hospial NHS Foundation Trust, John Radcliffe Hospital

    Oxford, OX3 9DU

Other trials in United Kingdom

Belgium2 sites

  • Universitair Ziekenhuis Gent

    Ghent, 9000

  • UZ Leuven

    Leuven, 3000

Other trials in Belgium

Germany2 sites

  • University of Essen - Children's Hospital

    Essen, D-45147

  • Klinikum der Universität München

    Munich, 80337

Other trials in Germany

Italy2 sites

  • Fondazione Policlinico Universitario A Gemelli

    Rome, 168

  • A.O.U. Citta della Salute e della Scienza di Torino - SS Malattie Neuromuscolari, Department of Neurosciences

    Torino, 10139

Other trials in Italy

Spain2 sites

  • Hospital Sant Joan de Déu. U.B.

    Barcelona, 08950

  • Hospital Universitari I Politecnic La Fe de Valencia

    Valencia, 46026

Other trials in Spain

Canada1 site

  • Children's Hospital - London Health Sciences Centre (LHSC)

    London, Ontario, N6A 5W9

Other trials in Canada

Netherlands1 site

  • Leiden University Medical Center

    Leiden, 2333

Other trials in Netherlands

About this study

Official title: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2) Part B will be conducted to further evaluate the vesleteplirsen doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 (NCT03675126) and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.

Being tested
Vesleteplirsen
Keywords
DMD, Duchenne, Dystrophy, Dystrophin, Exon Skipping, Ambulatory, Duchenne Muscular Dystrophy, Exon 51, Nonambulatory, Pediatric, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VesleteplirsenDrug

    Also called SRP-5051

    Vesleteplirsen injection, for IV use

Study groups in NCT04004065
Study groupRoleWhat they receive
Part A: VesleteplirsenExperimentalParticipants received escalating dose levels of vesleteplirsen, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.
Receives: Drug: Vesleteplirsen
Part B: VesleteplirsenExperimentalParticipants received vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This included the participants who rolled over from Part A, as well as the additional participants who enrolled at the beginning of Part B.
Receives: Drug: Vesleteplirsen

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A: Incidence of Adverse Events (AEs)

    Measured: Part A: Baseline up to 75 weeks

  2. Part B: Change From Baseline in Dystrophin Protein Level at Week 28

    Measured: Part B: Baseline, Week 28

Secondary outcomes (7)

  1. Part A: Pharmacokinetics (PK): Plasma Concentration of Vesleteplirsen

    Measured: Pre-dose and at multiple time points (up to 32 hours) after end of infusion

  2. Part A: PK: Urine Concentration of Vesleteplirsen

    Measured: Pre-dose and at multiple time periods (up to 48 hours) after end of infusion

  3. Part B: Change From Baseline in Exon-Skipping Levels at Week 28

    Measured: Part B: Baseline, Week 28

  4. Part B: Incidence of Adverse Events (AEs)

    Measured: Part B: Baseline up to Week 304

  5. Part B: PK: Plasma Concentration of Vesleteplirsen

    Measured: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion

  6. Part B: PK: Urine Concentration of Vesleteplirsen

    Measured: Part B predose and at multiple timepoints (up to 48 hours) after end of infusion

  7. Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay at Week 28

    Measured: Part B: Baseline, Week 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Jul 2019
  2. Study start26 Jun 2019
  3. Primary completion30 Oct 2023
  4. Study completion7 Feb 2025

Why it stopped: The study was terminated following review of safety data.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
62 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04004065
Sponsor's protocol ID
5051-201
Other IDs
2019-000601-77, 2019
Acronym
MOMENTUM
Status last verified
Mar 2025
First submitted
27 Jun 2019
Last updated
10 Mar 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Vesleteplirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 25 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 10 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 25 sites in United States, United Kingdom, Belgium, Germany, Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: The study was terminated following review of safety data.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A: Incidence of Adverse Events (AEs) (time frame: Part A: Baseline up to 75 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04004065.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2025.

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