Clinical trial • Phase III • Neurology|Rare Disease

APITEGROMAB for Spinal muscular atrophy (Type 2)|Spinal muscular atrophy (Type 3)

Phase III trial of APITEGROMAB for Spinal muscular atrophy (Type 2)|Spinal muscular atrophy (Type 3). open-label, none/not specified-controlled.

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Spinal muscular atrophy (Type 2)|Spinal muscular atrophy (Type 3)
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
04-03-2024
First CTIS Authorization Date
25-03-2024

Trial design

open-label, none/not specified-controlled Phase III trial in Belgium, Germany, Spain and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
111
Trial Duration For Participant
868

Eligibility

Recruits 111 paediatric patients.

Pregnancy Exclusion
Pregnant or breastfeeding.
Vulnerable Population
Includes vulnerable participants (minors). If the patient is legally a minor, informed consent must be signed by the patient's parent or legal guardian and the patient's oral or written assent must be obtained where applicable and in accordance with local regulatory and legal requirements. Age-specific assent and parental/guardian information/ICF documents are provided (examples include assent forms for ages 2-5, 6-12, 12-17 and parental/guardian ICFs; multiple language versions are available per country).

Inclusion criteria

  • {"criterion_text":"-Informed consent document signed by the patient if the patient is legally an adult. If the patient is legally a minor, informed consent document signed by the patient's parent or legal guardian and patient's oral or written assent obtained, if applicable and in accordance with the regulatory and legal requirements of the participating location."}
  • {"criterion_text":"-Patients who have completed the Phase 2 TOPAZ (Study SRK-015-002) trial or the Phase 3 SAPPHIRE (Study SRK-015-003) trial."}
  • {"criterion_text":"-Estimated life expectancy >2 years from Baseline (Day 1)."}
  • {"criterion_text":"-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 (i.e., for background medical care and not for the purpose of receiving apitegromab in the trial), throughout the trial."}
  • {"criterion_text":"-Able to adhere to the requirements of the protocol."}
  • {"criterion_text":"-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. Female patients who are expected to have reached reproductive maturity by the end of the trial must agree to adhere to trial-specific contraception requirements."}

Exclusion criteria

  • {"criterion_text":"-El paciente suspendió permanentemente el tratamiento del estudio durante el ensayo preliminar (es decir, TOPAZ o SAPPHIRE)"}
  • {"criterion_text":"-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."}
  • {"criterion_text":"-Patient is currently enrolled in any investigational drug trial other than TOPAZ or SAPPHIRE."}
  • {"criterion_text":"-Prior history of severe hypersensitivity reaction or intolerance to SMN-targeted therapies."}
  • {"criterion_text":"-Prior history of severe hypersensitivity reaction or intolerance to apitegromab."}
  • {"criterion_text":"-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."}
  • {"criterion_text":"-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."}
  • {"criterion_text":"-Pregnant or breastfeeding."}
  • {"criterion_text":"-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."}

Endpoints

Primary endpoints

  • {"endpoint_text":"-Incidence of treatment-emergent adverse event (TEAEs) and serious adverse event (SAEs) by severity","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"-HFMSE total score at prespecified time points (excludes TOPAZ Cohort 1 patients)","definition_or_measurement_approach":""}
  • {"endpoint_text":"-RULM total score at prespecified time points (excludes TOPAZ Cohort 1 patients)","definition_or_measurement_approach":""}
  • {"endpoint_text":"-Number of WHO motor development milestones attained at prespecified time points (excludes TOPAZ Cohort 1 patients)","definition_or_measurement_approach":""}
  • {"endpoint_text":"-RHS total score and results for 6-Minute Walk Test, 30-Second Sit-to-Stand, 10-Meter Walk/Run (from the RHS), and timed rise from floor (from the RHS) at prespecified time points (TOPAZ Cohort 1 patients only)","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
111
Recruitment Window Months
73
Consent Approach
Adults sign informed consent. For legally minor patients, a parent or legal guardian must sign the informed consent and the patient must provide oral or written assent where applicable and in accordance with local regulatory/legal requirements. Age-specific assent and parental/guardian ICFs are prepared (examples: assent forms for 2-5, 6-12, 12-17 years; parental/guardian ICFs). Multiple language versions are available across participating countries (e.g., English, French, Dutch, German, Italian, Polish and country-specific versions).

Geography

Total Number Of Sites
21
Total Number Of Participants
125

Belgium

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
25-07-2025
Processing Time Days
497
Number Of Sites
3
Number Of Participants
9

Sites

Site Name
Universitair Ziekenhuis Gent
Department Name
Neuromusculair Referentiecentrum
Contact Person Name
Nicolas Deconinck
Contact Person Email
Nicolas.deconinck@uzgent.be
Site Name
UZ Leuven
Department Name
Neuromuscular Diseases
Contact Person Name
Liesbeth De Waele
Contact Person Email
Liesbeth.dewaele@uzleuven.be
Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Neuro pediatry
Contact Person Name
Aurore Daron
Contact Person Email
Aurore.daron@chrcitadelle.be

Germany

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
28-07-2025
Processing Time Days
500
Number Of Sites
4
Number Of Participants
15

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik fuer Kinderheilkunde I
Contact Person Name
Heike Koelbel
Contact Person Email
heike.koelbel@uk-essen.de
Site Name
Medical Center - University Of Freiburg
Department Name
Zentrum fuer Kinder- und Jugendmedizin Klinik fuer Neuropaediatrie und Muskelerkrankungen
Contact Person Name
Astrid Pechmann
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Dr. von Haunersches Kinderspital Abteilung fuer Kinderneurologie und Entwicklungsneurologie
Contact Person Name
Astrid Blaschek
Site Name
Universitaetsklinikum Bonn AöR
Department Name
Zentrum fuer Kinderheilkunde Abteilung Neuropaediatrie
Contact Person Name
Dorothea Holzwarth
Contact Person Email
Dorothea.holzwarth@ukbonn.de

Spain

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
28-07-2025
Processing Time Days
500
Number Of Sites
2
Number Of Participants
21

Sites

Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neuromuscular Disease Unit
Contact Person Name
Inmaculada Pitarch Castellano
Contact Person Email
inmapitarch@hotmail.com
Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Neuromuscular Unit
Contact Person Name
Andrés Nascimento
Contact Person Email
andres.nascimento@sjd.es

Netherlands

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
01-08-2025
Processing Time Days
504
Number Of Sites
1
Number Of Participants
8

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
Department of Neurology
Contact Person Name
Ludo Van der Pol
Contact Person Email
W.L.vanderPol@umcutrecht.nl

Italy

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
28-07-2025
Processing Time Days
500
Number Of Sites
5
Number Of Participants
40

Sites

Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
Unita Operativa Complessa di Neuropsichiatria infantile
Contact Person Name
Eugenio Maria Mercuri
Site Name
Centro Clinico Nemo
Department Name
Centro Clinico Nemo
Contact Person Name
Valeria Ada Sansone
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Developmental Neurology Unit
Contact Person Name
Riccardo Masson
Site Name
Azienda Ospedaliera Universitaria Gaetano Martino Messina
Department Name
U.O.C. Neurologia e Malattie Neuromuscolari
Contact Person Name
Sonia Messina
Contact Person Email
smessina@unime.it
Site Name
Giannina Gaslini Institute For Scientific Hospitalization And Care
Department Name
UOSD Centro di Miologia e Patologie Neurodegenerative
Contact Person Name
Claudio Bruno
Contact Person Email
claudiobruno@gaslini.org

Poland

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
28-07-2025
Processing Time Days
500
Number Of Sites
3
Number Of Participants
16

Sites

Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Neurologii Rozwojowej
Contact Person Name
Maria Mazurkiewicz-Bełdzińska
Contact Person Email
mmazur@gumed.edu.pl
Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
Oddzial Kliniczny Neurologii Dzieci i Modziezy
Contact Person Name
Barbara Steinborn
Contact Person Email
bstein@ump.edu.pl
Site Name
Instytut Pomnik Centrum Zdrowia Dziecka
Department Name
Oddział Neurologii i Epileptologii
Contact Person Name
Katarzyna Kotulska-Jóźwiak
Contact Person Email
k.kotulska@ipczd.pl

France

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
04-08-2025
Processing Time Days
507
Number Of Sites
3
Number Of Participants
16

Sites

Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Neurology and Neuropediatric
Contact Person Name
Jean-Baptiste DAVION
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
Pediatric Neurology
Contact Person Name
Claude CANCES
Contact Person Email
cances.c@chu-toulouse.fr
Site Name
Trousseau Hospital
Department Name
Institut de myologie
Contact Person Name
Andreea SEFERIAN

Sponsor

Primary sponsor

Full Name
Scholar Rock Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Pharmaceutical Product Development LLC
Responsibilities
code: 8
Name
Syneos Health Inc.
Responsibilities
Multiple operational responsibilities; SUSAR reporting and regulatory (codes: 1,2,7,9,10,11; code 15 includes 'SUSAR reporting, Regulatory')
Name
Eresearchtechnology Inc.
Responsibilities
ECG analysis/ review (code: 15)

Third parties

  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"code: 8","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ECG analysis/ review (code: 15)","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"Videotaping (code: 15)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"codes: 1,10,11,2,7,9; SUSAR reporting, Regulatory (code: 15)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Randomization Trial Supply Management (RTSM) – EDC Platform (code: 15)","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
APITEGROMAB
Active Substance
APITEGROMAB
Modality
Monoclonal antibody
Routes Of Administration
Intravenous
Route
Intravenous
Orphan Designation
Yes
Starting Dose
20 mg/kg
Dose Levels
20 mg/kg
Frequency
Every 4 weeks
Maximum Dose
20 mg/kg per infusion; max total 520 mg/kg
Combination Treatment
Yes

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