What side effects are associated with this type of intervention?

Common treatments for Spinal Muscular Atrophy (SMA) include nusinersen, risdiplam, and onasemnogene abeparvovec-xioi. Nusinersen can cause side effects such as lower respiratory infections, constipation, and post-lumbar puncture syndrome. Risdiplam may lead to fever, diarrhea, rash, and upper respiratory tract infections. Onasemnogene abeparvovec-xioi is associated with elevated liver enzymes, vomiting, and thrombocytopenia. These side effects vary in severity and frequency, and patients should discuss them with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Spinal Muscular Atrophy (SMA). Nusinersen (Spinraza) was the first approved treatment, which works by increasing the production of the SMN protein. Risdiplam (Evrysdi) is an oral medication that also aims to increase SMN protein levels. Onasemnogene abeparvovec-xioi (Zolgensma) is a gene therapy that delivers a functional copy of the SMN1 gene to address the genetic root cause of SMA. These treatments have significantly improved outcomes for SMA patients and are similar to the adjunctive therapy being studied with taldefgrobep alfa.

What other types of research exist?

Promising novel alternatives to Taldefgrobep Alfa for Spinal Muscular Atrophy patients include nusinersen, risdiplam, and onasemnogene abeparvovec-xioi. These treatments are already established and may have ongoing advancements or new formulations by 2024. Additionally, ongoing research and clinical trials may introduce new therapies targeting different mechanisms or improving efficacy and safety profiles.

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Taldefgrobep Alfa for Spinal Muscular Atrophy (RESILIENT Trial)

Phase 3

Waitlist Available

Research Sponsored by Biohaven Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Ambulant or Non-Ambulant

Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number

Must not have

Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)

Must weigh at least 15kg

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 48

Awards & highlights

Pivotal Trial

Summary

This trial will test if taldefgrobep alfa can help people with SMA who are already on other treatments. The drug works by blocking a protein that stops muscle growth, potentially making muscles stronger and improving movement. Taldefgrobep alfa has shown promise in increasing muscle mass and function in SMA patients.

Who is the study for?

This trial is for people with Spinal Muscular Atrophy who are already on a stable dose of specific SMA treatments like nusinersen or risdiplam, or have had gene therapy. Participants must weigh at least 15kg and can be ambulant or non-ambulant. Those needing daytime ventilation, recent spinal fusion patients, and those with certain implants or previous anti-myostatin therapies cannot join.

What is being tested?

The study tests taldefgrobep alfa's effectiveness and safety as an additional treatment for Spinal Muscular Atrophy alongside existing medications compared to a placebo. It aims to see if adding taldefgrobep alfa improves patient outcomes.

What are the potential side effects?

While the side effects of taldefgrobep alfa aren't specified here, similar drugs may cause injection site reactions, muscle pain, headaches, nausea, and potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can walk with or without assistance.

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I have been genetically diagnosed with Spinal Muscular Atrophy.

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I am currently on a stable dose of SMA treatment and plan to stay on it.

Exclusion Criteria

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

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I need a ventilator to breathe during the day.

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I weigh at least 15kg.

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I have never taken anti-myostatin therapies.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 48

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 48

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 48 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score

Awards & Highlights

Pivotal Trial

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

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: taldefgrobep alfaExperimental Treatment1 Intervention

taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week Open label Extension (OLE) phase.

Group II: PlaceboPlacebo Group2 Interventions

Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for 48-week OLE phase.

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Nusinersen is an antisense oligonucleotide that modifies the splicing of SMN2 gene transcripts to increase the production of functional SMN protein, which is deficient in SMA patients. Risdiplam is a small molecule that also targets SMN2 gene splicing, enhancing the production of the SMN protein. Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene using a viral vector, directly addressing the genetic cause of SMA. These treatments are crucial for SMA patients as they aim to increase the levels of SMN protein, which is essential for motor neuron survival and function, thereby slowing disease progression and improving motor function.