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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)

Must not have

Received ZOLGENSMA® (onasemnogene abeparvovec-xioi) at any time and previous treatment with apitegromab

Use of invasive ventilation and tracheostomy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to 12 months.

Awards & highlights

Pivotal 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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been treated with ZOLGENSMA® and apitegromab.

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I use a machine to help me breathe through a tube in my throat.

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I use a ventilator during the day for more than 16 hours.

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I haven't had any serious health issues or infections in the last week.

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I haven't had major surgery in the last 6 months that could affect my movement.

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I've had allergic reactions to certain protein-based treatments.

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I haven't used muscle or nerve affecting drugs or supplements in the last 60 days.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to 12 months.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.

Secondary study objectives

Upper Extremity

Main Efficacy Population: Proportion of patients with ≥3-point change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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.

0 / 13Eligibility criteria met