SMANCT06877689
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
A study testing Apitegromab in people with SMA, run by Scholar Rock, Inc..
Trial summary
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency . Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- SMA
- What is being tested?
- Apitegromab
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- ≥2 years of age.
- Documented diagnosis of 5q SMA.
Who cannot join
- Previous history of a hypersensitivity reaction to a monoclonal antibody or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab.
- Enrolled in a clinical study for any investigational drug.
- Pregnant or breastfeeding.
About this study
Official title: Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency . A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
- Conditions
- SMA
- Being tested
- Apitegromab
- Keywords
- Expanded Access, Pre-Approval Access, Compassionate Use
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApitegromabDrug
Also called SRK-015
Apitegromab (SRK-015) is a fully human anti-proMyostatin monoclonal antibody (mAb) that specifically binds to human pro/latent myostatin, inhibiting myostatin activation. Apitegromab will be administered every 4 weeks by intravenous (IV) infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SMAStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Scholar Rock, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06877689
- Sponsor's protocol ID
- SRK-015-008
- Status last verified
- Apr 2026
- First submitted
- 10 Mar 2025
- Last updated
- 28 Apr 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT06877689
- Acronym / sponsor ID
- SRK-015-008
- Conditions
- SMA
- Keywords
- Expanded Access, Pre-Approval Access, Compassionate Use
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Scholar Rock, Inc.Industry (pharma, biotech, devices)
- First posted
- 14 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this expanded access program (EAP) is to provide access to apitegromab for eligible patients with spinal muscular atrophy (SMA) prior to approval by the local regulatory agency . A medical doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Study design
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- ≥2 years of age.
- Documented diagnosis of 5q SMA.
Key exclusion criteria (3 listed)
- Previous history of a hypersensitivity reaction to a monoclonal antibody or recombinant protein bearing an Fc domain (eg, a soluble receptor-Fc fusion protein), apitegromab, or excipients of apitegromab.
- Enrolled in a clinical study for any investigational drug.
- Pregnant or breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ApitegromabAlso: SRK-015Apitegromab (SRK-015) is a fully human anti-proMyostatin monoclonal antibody (mAb) that specifically binds to human pro/latent myostatin, inhibiting myostatin activation. Apitegromab will be administered every 4 weeks by intravenous (IV) infusion. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Apitegromab in people with SMA, run by Scholar Rock, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026.