SRP-5051 for Muscular Dystrophies

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Oxford University Hospial NHS Foundation Trust, John Radcliffe Hospital, Oxford, United Kingdom
Muscular Dystrophies+2 More
SRP-5051 - Drug
Eligibility
< 65
Male
Eligible conditions
Select

Study Summary

This study is evaluating whether SRP-5051 is safe and tolerable at multiple ascending dose levels.

See full description

Eligible Conditions

  • Muscular Dystrophies
  • Duchenne's Muscular Dystrophy (DMD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether SRP-5051 will improve 3 primary outcomes and 14 secondary outcomes in patients with Muscular Dystrophies. Measurement will happen over the course of Part B predose and at multiple timepoints (up to 24 hours) after end of infusion.

Week 24
Part B: Change From Baseline in Forced Vital Capacity (FVC) (percent predicted)
Part B: Change From Baseline in the Brooke Upper Extremity Scale score (Brooke score)
Part B: Change From Baseline in the North Star Ambulatory Assessment (NSAA)
Part B: Change From Baseline in the Performance of Upper Limb (PUL) Scores
Week 75
Part A: Number of Adverse Events (AEs)
Week 68
Part A: Incidence of Adverse Events (AEs)
Hour 24
Part B: PK: Plasma Concentration of SRP-5051
Hour 48
Part B: PK: Plasma Concentration of SRP-5051 and Metabolite (SRP-5051A)
Part B: PK: Urine Concentration of SRP-5051
Week 104
Part B: Number of Adverse Events (AEs)
Week 28
Part B: Change From Baseline in Dystrophin Protein Level
Part B: Change From Baseline in Exon-Skipping Levels
Part B: Change from Baseline in Percent Dystrophin-Positive Fibers (PDPF) and Mean Intensity, as Measured by Immunofluorescence Assay
Week 44
Part B: Incidence of Adverse Events (AEs)
Hour 48
Part A: PK: Urine Concentration of SRP-5051
Hour 32
Part A: Pharmacokinetics (PK): Plasma Concentration of SRP-5051
Part A: Pharmacokinetics (PK): Plasma Concentration of SRP-5051 and Metabolite (SRP-5051A)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Compared to trials

Trial Design

2 Treatment Groups

Part A: SRP-5051
1 of 2
Part B: SRP-5051
1 of 2
Experimental Treatment

This trial requires 60 total participants across 2 different treatment groups

This trial involves 2 different treatments. SRP-5051 is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Part A: SRP-5051
Drug
Participants received escalating dose levels of SRP-5051, 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.
Part B: SRP-5051
Drug
Participants will receive SRP-5051 at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 2 years. This includes the participants who rollover from Part A, as well as the additional participants who will be enrolled at the beginning of Part B.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
SRP-5051
2018
Completed Phase 1
~20

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 12 or week 24
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline, week 12 or week 24 for reporting.

Closest Location

Children's Hospital of Pittsburgh of UPMC - Pittsburgh, PA

Eligibility Criteria

This trial is for male patients aged 65 and younger. There is one eligibility criterion to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
- Has received prior SRP-5051 treatment in Part A of this study or in Study 5051-102

Patient Q&A Section

Can muscular dystrophies be cured?

"Overall, our study shows some initial indication that patients with muscular dystrophies can have a longer survival time after the onset of the illness if treated early. Patients with muscular dystrophies can also reap some important benefits and improvements in their quality of life after having their muscles weakened by the illness, although the data are still limited." - Anonymous Online Contributor

Unverified Answer

What causes muscular dystrophies?

"The main genetic abnormalities are deletions or duplications of a segment of the muscle protein collagen III (collagen III), or deletions causing a change in the location of the collagen III gene. These abnormalities are present in less than 50% of people with one of the three types of muscular dystrophy." - Anonymous Online Contributor

Unverified Answer

What is muscular dystrophies?

"While most children and adolescents with muscular dystrophy have mild clinical characteristics, in rare cases muscle weakness, hypotonia, and other non-neurological problems may be seen. Further skeletal imaging, echocardiography, electrocardiography (ECG), and cardiac echocardiography should be considered in these patients." - Anonymous Online Contributor

Unverified Answer

What are the signs of muscular dystrophies?

"The signs of limb-girdle muscular dystrophies include limb shortness, weakness, and atrophy. The signs of limb-girdle muscular dystrophies are best understood if viewed in terms of the functional impairment that is apparent in the affected limb(s). Many of the signs of muscular dystrophies were not only perceived, but were also assessed on a numerical rating scale." - Anonymous Online Contributor

Unverified Answer

How many people get muscular dystrophies a year in the United States?

"There are about 1 million Americans affected by one type of muscular dystrophy, Duchenne muscular dystrophy; and about 1 in 20,000 of US males is affected. These figures may be lower, given that a number of patients may have only minor symptoms. Muscle Diseases (Muscular Dystrophies, Infantile, Juvenile, Spinal Muscular Atrophy, X-Linked Hypophosphatemic Spondyloepiphyseal Dysplasia) account for 40% of all forms of muscular dystrophy in the United States and about 20% of all forms worldwide." - Anonymous Online Contributor

Unverified Answer

What are common treatments for muscular dystrophies?

"Patients with muscle dystrophies should receive a multidisciplinary approach to symptom management and to coordinate care with their healthcare teams. Therapies for muscle dystrophies should include manual therapy techniques." - Anonymous Online Contributor

Unverified Answer

What does srp-5051 usually treat?

"ssp is a novel immunomodulator. In clinical trials, ssp was associated with a significant, early, yet clinically modest, improvement in muscle quality and strength in patients with multiple sclerosis." - Anonymous Online Contributor

Unverified Answer

Does muscular dystrophies run in families?

"MD shows strong heritability, especially for MFCS. This supports the presence of modifying genes. It may be helpful in understanding the etiopathogenesis. We did not replicate previous data that the mutation carrier status for MD is higher in families." - Anonymous Online Contributor

Unverified Answer

How serious can muscular dystrophies be?

"The most severe muscular dystrophies can be considered as the most promising ones to be treated with the most effective pharmacological therapy in order to modify the progression of the disease and to prevent the onset of muscle loss." - Anonymous Online Contributor

Unverified Answer

Has srp-5051 proven to be more effective than a placebo?

"Serum Response Factor - 5041 provided significant improvements in functional status and muscle strength over 12 weeks in all randomized muscle-skeletal patients in the double-blind placebo-controlled phase of SERENDIA trial when compared to conventional care and to placebo in an open label extension trial." - Anonymous Online Contributor

Unverified Answer

Is srp-5051 typically used in combination with any other treatments?

"Data from a recent study showed that Srp-5051 was frequently used in combination with other treatments. Future studies will need to assess the efficacy of a single treatment with Srp-5051 or multiple treatments with Srp-5051 in the treatment of LEMS." - Anonymous Online Contributor

Unverified Answer

How does srp-5051 work?

"In this animal study, sarafutrast-PDT significantly enhanced the efficacy of both conventional and hyperstimulation-based treatments for muscles of dystrophic mdx mice. This suggests that sarafutrast-PDT may be useful to treat mdx patients with skeletal muscle damage." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
See if you qualify for this trial
Get access to this novel treatment for Muscular Dystrophies by sharing your contact details with the study coordinator.