Apitegromab for Muscular Atrophy

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Muscular Atrophy+10 More
Apitegromab - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new drug, apitegromab, as an adjunctive therapy to nusinersen or risdiplam, for patients with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA). The trial will confirm the efficacy and safety of apitegromab as an adjunctive therapy to nusinersen and evaluate the efficacy and safety of apitegromab as an adjunctive therapy to risdiplam.

Eligible Conditions
  • Muscular Atrophy
  • Spinal Muscular Atrophy (SMA)
  • LY2495655
  • Oppenheim Disease
  • Type 2 Spinal Muscular Atrophy
  • Neuromuscular Manifestations
  • Spinal Muscular Atrophy
  • Atrophy
  • SMA
  • Muscular Atrophy, Spinal

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Muscular Atrophy

Study Objectives

1 Primary · 7 Secondary · Reporting Duration: Baseline up to 12 months.

Month 12
Main Efficacy Population and Exploratory Subpopulation combined: Apitegromab concentrations in serum from blood samples.
Main Efficacy Population and Exploratory Subpopulation combined: Circulating latent myostatin concentrations in blood samples.
Main Efficacy Population and Exploratory Subpopulation combined: Incidence of Treatment Emergent Adverse Events (TEAEs) and Severe Adverse Events (SAEs) by severity.
Main Efficacy Population and Exploratory Subpopulation combined: Presence or absence of ADA against apitegromab in serum from blood samples.
Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
Upper Extremity
Main Efficacy Population: Change from Baseline in number of WHO motor development milestones attained at 12 months.
Main Efficacy Population: Proportion of patients with ≥3-point change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Muscular Atrophy

Trial Design

5 Treatment Groups

Exploratory Subpopulation (Apitegromab)
1 of 5
Main Efficacy Population (Apitegromab 10 mg/kg)
1 of 5
Main Efficacy Population (Apitegromab 20 mg/kg)
1 of 5
Main Efficacy Population (Placebo)
1 of 5
Exploratory Subpopulation (Placebo)
1 of 5
Experimental Treatment
Non-Treatment Group

204 Total Participants · 5 Treatment Groups

Primary Treatment: Apitegromab · Has Placebo Group · Phase 3

Exploratory Subpopulation (Apitegromab)
Drug
Experimental Group · 1 Intervention: Apitegromab · Intervention Types: Drug
Main Efficacy Population (Apitegromab 10 mg/kg)
Drug
Experimental Group · 1 Intervention: Apitegromab · Intervention Types: Drug
Main Efficacy Population (Apitegromab 20 mg/kg)
Drug
Experimental Group · 1 Intervention: Apitegromab · Intervention Types: Drug
Main Efficacy Population (Placebo)
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
Exploratory Subpopulation (Placebo)
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline up to 12 months.

Who is running the clinical trial?

Scholar Rock, Inc.Lead Sponsor
2 Previous Clinical Trials
258 Total Patients Enrolled
1 Trials studying Muscular Atrophy
58 Patients Enrolled for Muscular Atrophy

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 1st, 2021

Last Reviewed: October 1st, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

How old are they?
18 - 65100.0%
What site did they apply to?
Stanford University Medical Center100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%