Trial record 1 of 2 for:
apitegromab
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (ONYX)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT05626855 |
|
Recruitment Status :
Active, not recruiting
First Posted : November 25, 2022
Last Update Posted : April 12, 2024
|
Sponsor:
Scholar Rock, Inc.
Information provided by (Responsible Party):
Scholar Rock, Inc.
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Spinal Muscular Atrophy Spinal Muscular Atrophy Type 3 Spinal Muscular Atrophy Type II SMA Neuromuscular Diseases Muscular Atrophy Atrophy Muscular Atrophy, Spinal Neuromuscular Manifestations Anti-myostatin | Drug: Apitegromab | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 260 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Intervention Model Description: | Open Label Extension Study |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab |
| Actual Study Start Date : | April 17, 2023 |
| Estimated Primary Completion Date : | November 1, 2026 |
| Estimated Study Completion Date : | January 1, 2027 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Spinal muscular atrophy
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Treatment Period
Patients who are ≥2 years of age with Type 2 and Type 3 SMA will receive apitegromab 20 mg/kg every 4 weeks by intravenous (IV) infusion during the 104-week Treatment Period
|
Drug: Apitegromab
Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength. |
Primary Outcome Measures :
- Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA [ Time Frame: Up to 6 years ]Incidence of TEAEs and SAEs by severity
Secondary Outcome Measures :
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]Hammersmith Functional Motor Scale Expanded (HFMSE) total score at prespecified time points (excludes ambulatory patients)
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]Revised Upper Limb Module (RULM) total score at prespecified time points (excludes ambulatory patients)
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]Number of World Health Organization (WHO) motor development milestones attained at prespecified time points (excludes ambulatory patients)
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]Revised Hammersmith Scale (RHS) total score
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]Results for 6-Minute Walk Test
- Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points [ Time Frame: Up to 6 years ]30-Second Sit-to-Stand
- Further evaluate the immunogenicity of apitegromab [ Time Frame: Up to 6 years ]Presence or absence of antidrug antibody (ADA) against apitegromab in serum from blood samples
Other Outcome Measures:
- Further characterize the PK of apitegromab [ Time Frame: Up to 6 years ]Apitegromab concentrations in serum from blood samples at prespecified time points
- Further evaluate the pharmacodynamic (PD) effects of apitegromab [ Time Frame: Up to 6 years ]Circulating latent myostatin concentrations in blood samples at prespecified time points
- To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior [ Time Frame: Up to 6 years ]Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) at prespecified time points
- To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior [ Time Frame: Up to 6 years ]Patient-reported Outcomes Measurement Information System (PROMIS) Fatigue Questionnaire at prespecified time points
- To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior [ Time Frame: Up to 6 years ]Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) at prespecified time points
- To further evaluate the effect of apitegromab on patient/caregiver-reported disability, fatigability, and suicidal ideation and behavior [ Time Frame: Up to 6 years ]Columbia-Suicide Severity Rating Scale (C-SSRS) at prespecified time points
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial. (For TOPAZ, completed is defined as completion of Visit EC14 in Extension Period C or participating in TOPAZ at the time the trial is ended. For SAPPHIRE, completed is defined as completion of Visit 14 or participating in SAPPHIRE at the time the trial is ended)
- Estimated life expectancy >2 years from Baseline (Day 1)
- Able to receive study drug infusions and provide blood samples through the use of a peripheral IV or a long-term IV access device that the patient has placed for reasons independent from the trial
- Able to adhere to the requirements of the protocol, including travel to the trial site and completing all trial procedures and trial visits
- Females of childbearing potential must have a negative pregnancy test at Baseline and agree to use at least 1 acceptable method of contraception throughout the trial and for 20 weeks after the last dose of apitegromab
Exclusion Criteria:
- Patient permanently discontinued study treatment during the feeder trial (i.e., TOPAZ or SAPPHIRE)
- Nutritional status that was not stable over the past 6 months and is not anticipated to be stable throughout the trial or medical necessity for a gastric/nasogastric feeding tube, where the majority of feeds are given by this route, as assessed by the Investigator
- Patient is currently enrolled in any investigational drug trial other than TOPAZ or SAPPHIRE
- Prior history of severe hypersensitivity reaction or intolerance to SMN-targeted therapies
- Prior history of severe hypersensitivity reaction or intolerance to apitegromab
- Use of chronic daytime noninvasive ventilatory support for >16 hours daily in the 2 weeks before dosing, or anticipated to regularly receive such daytime ventilator support chronically throughout the trial
- Any acute or comorbid condition interfering with the well-being of the patient at the patient's last visit in TOPAZ or SAPPHIRE, (including active systemic infection, the need for acute treatment, or inpatient observation due to any reason). After resolution of the condition, the patient can be enrolled in the trial if they meet all the other eligibility criteria.
- Pregnant or breastfeeding
- Any other condition or clinically significant laboratory result or ECG value that, in the opinion of the Investigator, may compromise safety or compliance, would preclude the patient from successful completion of the trial, or interfere with the interpretation of the results
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05626855
Locations
Show 40 study locations
Sponsors and Collaborators
Scholar Rock, Inc.
| Responsible Party: | Scholar Rock, Inc. |
| ClinicalTrials.gov Identifier: | NCT05626855 |
| Other Study ID Numbers: |
SRK-015-004 |
| First Posted: | November 25, 2022 Key Record Dates |
| Last Update Posted: | April 12, 2024 |
| Last Verified: | April 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
|
Muscular Atrophy Muscular Atrophy, Spinal Neuromuscular Diseases Spinal Muscular Atrophies of Childhood Neuromuscular Manifestations Atrophy Pathological Conditions, Anatomical Neurologic Manifestations |
Nervous System Diseases Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn |