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Apitegromab for Spinal Muscular Atrophy (SAPPHIRE Trial)

Phase 3
Waitlist Available
Research Sponsored by Scholar Rock, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Documented diagnosis of 5q SMA
Diagnosed with later-onset SMA (i.e., Type 2 and Type 3 SMA) before receiving an approved SMN upregulator therapy (i.e., either nusinersen or risdiplam)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to 12 months.
Awards & highlights

SAPPHIRE Trial 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.

Who is the study for?
This trial is for patients aged 2-21 with later-onset spinal muscular atrophy (Types 2 and 3) who are already taking nusinersen or risdiplam. Participants must be nonambulatory but able to sit independently, have a stable nutritional status, and not use invasive ventilation over 16 hours daily. They can't join if they've had ZOLGENSMA treatment, severe scoliosis/contractures, hypersensitivity to monoclonal antibodies, or used certain muscle-related therapies recently.Check my eligibility
What is being tested?
The study tests the effectiveness of apitegromab as an additional therapy alongside approved SMN upregulator treatments (nusinersen or risdiplam) in improving motor function in SMA patients. It's a Phase 3 trial where some participants will receive apitegromab while others will get a placebo for comparison.See study design
What are the potential side effects?
Potential side effects of apitegromab may include reactions similar to those seen with other monoclonal antibodies such as infusion reactions, allergic responses, and possible impacts on immune function. The exact side effects are being studied.

SAPPHIRE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with 5q spinal muscular atrophy.
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I was diagnosed with Type 2 or 3 SMA before starting treatments like nusinersen or risdiplam.
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I cannot walk but can sit up straight without support.
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I am currently on one SMA treatment and plan to stay on it during the trial.
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My motor function score is between 10 and 45.
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I can receive medication through an IV and give blood samples for the study.

SAPPHIRE Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to 12 months.
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 12 months. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.
Secondary outcome measures
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.
+4 more

SAPPHIRE Trial Design

5Treatment groups
Experimental Treatment
Placebo Group
Group I: Main Efficacy Population (Apitegromab 20 mg/kg)Experimental Treatment1 Intervention
Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 12 years old at Screening. Participants will be randomized to receive apitegromab 20 mg/kg for up to 52 weeks.
Group II: Main Efficacy Population (Apitegromab 10 mg/kg)Experimental Treatment1 Intervention
Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 12 years old at Screening. Participants will be randomized to receive apitegromab 10 mg/kg for up to 52 weeks.
Group III: Exploratory Subpopulation (Apitegromab)Experimental Treatment1 Intervention
Type 2 SMA and Nonambulatory Type 3 SMA, ages 13 through 21 years old at Screening. Participants will be randomized to receive apitegromab 20 mg/kg for up to 52 weeks.
Group IV: Main Efficacy Population (Placebo)Placebo Group1 Intervention
Type 2 SMA and Nonambulatory Type 3 SMA, ages 2 through 12 years old at Screening. Participants will be randomized to receive placebo for up to 52 weeks.
Group V: Exploratory Subpopulation (Placebo)Placebo Group1 Intervention
Type 2 SMA and Nonambulatory Type 3 SMA, ages 13 through 21 years old at Screening. Participants will be randomized to receive placebo for up to 52 weeks.

Find a Location

Who is running the clinical trial?

Scholar Rock, Inc.Lead Sponsor
3 Previous Clinical Trials
430 Total Patients Enrolled

Media Library

Apitegromab Clinical Trial Eligibility Overview. Trial Name: NCT05156320 — Phase 3
Spinal Muscular Atrophy Research Study Groups: Main Efficacy Population (Apitegromab 10 mg/kg), Main Efficacy Population (Apitegromab 20 mg/kg), Main Efficacy Population (Placebo), Exploratory Subpopulation (Apitegromab), Exploratory Subpopulation (Placebo)
Spinal Muscular Atrophy Clinical Trial 2023: Apitegromab Highlights & Side Effects. Trial Name: NCT05156320 — Phase 3
Apitegromab 2023 Treatment Timeline for Medical Study. Trial Name: NCT05156320 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many different areas are conducting this experiment?

"There are 24 centres conducting this trial, with the primary locations being University of Utah in Salt Lake City, Seattle Children's Hospital in Seattle, and Rady's Children's Hospital/UCSD in San Diego."

Answered by AI

Has Apitegromab received approval from the FDA?

"Apitegromab received a safety score of 3 from our team at Power. This is due to the fact that Apitegromab is in Phase 3 trials, meaning that there is some data which suggests that the medication is effective as well as multiple rounds of data which support the safety of the medication."

Answered by AI

What else is known about Apitegromab from a medical standpoint?

"There are currently two clinical trials underway for Apitegromab, one of which is in Phase 3. Although the majority of trials for Apitegromab are situated in Warsaw and Utah, research is being conducted in 46 different locations."

Answered by AI

Does this clinical trial pave the way for other similar studies?

"At the moment, there are two different ongoing trials concerning Apitegromab. They are being conducted across 22 cities and 7 different countries. The first study happened in 2019. That particular trial, which was sponsored by Scholar Rock, Inc., included 58 patients. It completed its Phase 2 drug approval stage. Since 2019, a total of 18240 trials have been conducted."

Answered by AI

Are researchers looking for more test subjects?

"The listing on clinicaltrials.gov says that this trial is still enrolling patients. The study was first publicized on February 24th, 2022 and received its latest update on October 27th, 2022. They are looking for a total of 204 patients from 21 different recruitment sites."

Answered by AI

Do elderly people fit the age bracket required for this clinical trial?

"From the information available, it seems that the youngest age this trial can accept is 2 years old, while the oldest age is 21."

Answered by AI

How many participants are being allowed in this trial?

"In order to meet the needs of this study, a total of 204 patients are required who fit the inclusion criteria. Scholar Rock, Inc. will be sponsoring the trial and running it out of various sites including University of Utah in Salt Lake City, Utah and Seattle Children's Hospital in Seattle, Washington."

Answered by AI

May I sign up for this research trial?

"So long as they meet the age criteria of being between 2 and 21 years old and have muscular atrophy, patients are eligible to participate in this trial. Up to 204 patients will be accepted."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Stanford University Medical Center
University of Utah
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
2022. "Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05156320.
All > Spinal Muscular Atrophy
~30 spots leftby Oct 2024
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