Clinical trial NCT05626855: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Spinal Muscular Atrophy
Spinal Muscular Atrophy Type 3
Spinal Muscular Atrophy Type 2
SMA
Neuromuscular Diseases
Muscular Atrophy
Atrophy
Muscular Atrophy, Spinal
Neuromuscular Manifestations
Anti-myostatin
Lead sponsor: Scholar Rock, Inc.
Sponsor class: INDUSTRY
First posted: 2022-11-25
Enrollment: 238
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open Label Extension Study
Primary purpose: TREATMENT

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.


Eligibility 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 the Baseline Visit (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 the Baseline Visit and agree to use at least 1 highly effective 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

Primary outcomes:
measure: Evaluate the long-term safety and tolerability of apitegromab in patients with Type 2 and Type 3 SMA
timeFrame: Up to 6 years
Secondary outcomes:
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Evaluate the long-term efficacy of apitegromab by assessing changes in motor function outcome measures at prespecified time points
timeFrame: Up to 6 years
measure: Further evaluate the immunogenicity of apitegromab
timeFrame: Up to 6 years
Other outcomes:
measure: Further characterize the PK of apitegromab
timeFrame: Up to 6 years
measure: Further evaluate the pharmacodynamic (PD) effects of apitegromab
timeFrame: Up to 6 years
measure: To further evaluate the effect of apitegromab on patient/caregiver-reported disability, and fatigability.
timeFrame: Up to 6 years
measure: To further evaluate the effect of apitegromab on patient/caregiver-reported disability, and fatigability.
timeFrame: Up to 6 years
measure: To further evaluate the effect of apitegromab on patient/caregiver-reported disability, and fatigability.
timeFrame: Up to 6 years
Locations:
city: Phoenix
country: United States
facility: Phoenix Childrens Hospital
state: Arizona
city: La Jolla
country: United States
facility: UCSD Altman Clinical and Translational Research
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford Neuroscience Health Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Orlando
country: United States
facility: Nemours Biomedical Research
state: Florida
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Fairway
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns HopkinsHospital
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Saint Paul
country: United States
facility: Gillette Children's Specialty Healthcare
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University Medical Campus
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest University School of Medicine
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Dallas
country: United States
facility: University of Texas Southwestern - Pediatric Neurology
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Newport News
country: United States
facility: Children's Specialty Group PLLC (Children's Hospital of The King's Daughters)
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Madison
country: United States
facility: University of Wisconsin Hospital
state: Wisconsin
city: Gent
country: Belgium
facility: UZ Gent
city: Leuven
country: Belgium
facility: UZ Leuven
city: Liege
country: Belgium
facility: CHR Citadelle
city: Lille
country: France
facility: CHRU de Lille - Hpital Jeanne de Flandre
city: Paris
country: France
facility: Hopital Trousseau - I-Motion
city: Toulouse
country: France
facility: CHU Toulouse Hopital des Enfants
city: Bonn
country: Germany
facility: Universitätskinderklinik Bonn, Abteilung für Neuropädiatrie und SPZ
city: Essen
country: Germany
facility: Universitatsklinikum Essen
city: Freiburg
country: Germany
facility: Universitatsklinikum Freiburg
city: Munchen, Bayern
country: Germany
facility: Klinikum der Universitat Munchen, Dr. von Haunersches Kinderspital, Abteilung fur Kinderneurologie und Entwicklungsneurologie
city: Genova
country: Italy
facility: Istituto Giannina Gaslini, Centro Traslazionale di Miologia e Patologie Neurodegenerative
city: Messina
country: Italy
facility: UOC NEUROLOGIA E MALATTIE NEUROMUSCOLARI A.O.U Policlinico G. Martino
city: Milano
country: Italy
facility: Carlo Besta Neurological Research Institute
city: Milano
country: Italy
facility: NeuroMuscular Omnicentre
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario A. Gemelli
city: Utrecht
country: Netherlands
facility: UMC Utrecht
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne
city: Poznań
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Poznaniu, Oddział Kliniczny Neurologii Dzieci i Młodzieży
city: Warsaw
country: Poland
facility: Instytut Pomnik - Centrum Zdrowia Dziecka
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Deau
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
city: Leeds
country: United Kingdom
facility: Leeds Children's Hospital Clinical Research
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: Oxford
country: United Kingdom
facility: University of Oxford
Study officials:
References: