Clinical trial • Phase II • Neurology|Rare Disease|Musculoskeletal
APITEGROMAB for Spinal muscular atrophy
Phase II trial of APITEGROMAB for Spinal muscular atrophy. None/Not specified-controlled. 22 participants.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease|Musculoskeletal
- Trial Disease
- Spinal muscular atrophy
- Trial Stage
- Phase II
- Drug Modality
- Monoclonal antibody
- Paediatric Trial
- Yes
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 02-07-2025
- First CTIS Authorization Date
- 21-10-2025
Trial design
None/Not specified-controlled Phase II trial in Belgium, France, Italy and others.
- Comparator
- None/Not specified
- Target Sample Size
- 22
- Trial Duration For Participant
- 336
Eligibility
Recruits 22 paediatric patients.
- Vulnerable Population
- Participants are infants/toddlers (<2 years old); vulnerable population selected. Informed consent is provided by parent/guardian (Parent-guardian ICF and Subject Information Sheets are available in multiple languages). Assent is not applicable due to participant age.
Inclusion criteria
- {"criterion_text":"- Is <2 years old at the time of the informed consent\n- Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth\n- Has confirmed diagnosis of 5q autosomal recessive SMA\n- Has confirmed presence of SMN2 gene copy(ies)\n- Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)\n- Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit\n- Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score <55."}
Exclusion criteria
- {"criterion_text":"- Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route, as assessed by the Investigator and/or an Oral and Swallowing Abilities Tool score of 0.\n- Major orthopedic issues such as severe scoliosis or severe contractures or interventional procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject to be evaluated on any motor function outcome measures, within 6 months before Screening or anticipated during the study.\n- Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Population PK model-based parameters such as CL, Vd, Vc and Vp","definition_or_measurement_approach":"Population pharmacokinetic (PK) model-based estimation of parameters (e.g., CL, Vd, Vc, Vp) from collected PK data."}
- {"endpoint_text":"- Apitegromab and total latent myostatin concentrations in serum","definition_or_measurement_approach":"Measurement of apitegromab and total latent myostatin concentrations in serum (serum concentration assays)."}
- {"endpoint_text":"- Change from baseline in the raw score of the BSID-4 GMS at 48 weeks","definition_or_measurement_approach":"Assessment of change from baseline in Bayley Scales of Infant and Toddler Development, 4th Edition (BSID-4) Gross Motor Scale (GMS) raw score at 48 weeks."}
Secondary endpoints
- {"endpoint_text":"- Relationship of apitegromab concentrations and total latent myostatin concentration in serum","definition_or_measurement_approach":"Analysis of the relationship/correlation between apitegromab serum concentrations and total latent myostatin serum concentrations."}
- {"endpoint_text":"- Population PK/PD model-based parameters such as Imax, IC50, and Kout","definition_or_measurement_approach":"Population PK/PD modeling to estimate parameters (e.g., Imax, IC50, Kout) from PK and PD data."}
- {"endpoint_text":"- Incidence of TEAEs and SAEs by severity","definition_or_measurement_approach":"Collection and tabulation of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) categorized by severity."}
Recruitment
- Registry Or Advocacy Recruitment
- True, Advocacy PAG (patient advocacy group) clinical trial listing materials are used
- Digital Remote Recruitment
- True, includes website pages, social media posts, and e-newsletter content in multiple languages for outreach
- Planned Sample Size
- 22
- Recruitment Window Months
- 40
- Consent Approach
- Informed consent obtained from parent/guardian using Parent-guardian ICF and Subject Information Sheets (L1 SIS and ICF Parent-Guardian documents). ICF/SIS materials available in multiple languages (English, French, Dutch, Spanish, Italian as per document titles). Assent is not applicable due to participant age (<2 years).
Methods
- Website text – country-specific website recruitment pages (documents: Website_BE_DUT, Website_BE_FRE, Website_FRE_Redacted, Website_IT_Redacted, Website_SPA_Redacted).
- Social media posts – country/language specific social media recruitment posts (documents: Social media posts_BE_DUT, _BE_FRE, _IT, _SPA).
- Posters with flyer – printed poster/flyer materials for clinics (documents: Poster with flyer_BE_FRE, _BE_DUT, _IT, _SPA).
- Dr to parent/guardian letter – clinician-to-parent invitation letters (documents: Dr to parent guardian letter_BE_FRE, _BE_DUT, _IT, _SPA).
- Parent/guardian brochure – information brochures for parents/guardians (documents: Parent guardian brochure_BE_DUT_Redacted, _BE_FRE_Redacted, Parent-Guardian brochure_Redacted, parent_guardian_brochure_IT_Redacted, Parent-Guardian Brochure_SPA_Redacted).
- Advocacy/PAG clinical trial listing – materials for patient advocacy groups (documents: Advocacy PAG clinical trial listing_BE_DUT_Redacted, _BE_FRE_Redacted, Clinical Trial Listing_SPA_Redacted, advocacy materials_IT).
- E-newsletter / eNewsletter content – electronic newsletter content for advocacy/targeted audiences (documents: PAG enewsletter_BE_DUT, _BE_FRE, eNewsletter Content_SPA, eNewsletter content_IT).
- GP/clinician letters and clinic-based recruitment – GP letter and clinic referral materials (documents: GP letter_IT, Dr to Parent-Guardian letters).
Geography
- Total Number Of Sites
- 11
- Total Number Of Participants
- 30
Belgium
- Earliest CTIS Part Ii Submission Date
- 10-10-2025
- Latest Decision Or Authorization Date
- 21-10-2025
- Processing Time Days
- 11
- Number Of Sites
- 3
- Number Of Participants
- 6
Sites
- Site Name
- UZ Leuven
- Department Name
- Neuromuscular Diseases
- Principal Investigator Name
- Liesbeth De Waele
- Principal Investigator Email
- Liesbeth.dewaele@uzleuven.be
- Contact Person Name
- Liesbeth De Waele
- Contact Person Email
- Liesbeth.dewaele@uzleuven.be
- Site Name
- Universitair Ziekenhuis Gent
- Department Name
- Neuromusculair Referentiecentrum
- Principal Investigator Name
- Nicolas Deconinck
- Principal Investigator Email
- Nicolas.deconinck@uzgent.be
- Contact Person Name
- Nicolas Deconinck
- Contact Person Email
- Nicolas.deconinck@uzgent.be
- Site Name
- Centre Hospitalier Regional De La Citadelle
- Department Name
- Neuro paediatry
- Principal Investigator Name
- Aurore Daron
- Principal Investigator Email
- Aurore.daron@chrcitadelle.be
- Contact Person Name
- Aurore Daron
- Contact Person Email
- Aurore.daron@chrcitadelle.be
France
- Earliest CTIS Part Ii Submission Date
- 25-08-2025
- Latest Decision Or Authorization Date
- 24-10-2025
- Processing Time Days
- 60
- Number Of Sites
- 2
- Number Of Participants
- 8
Sites
- Site Name
- Association Institut De Myologie
- Department Name
- I-Motion, Hopital Armand Trousseau
- Principal Investigator Name
- Andreea SEFERIAN
- Principal Investigator Email
- a.seferian@institut-myologie.org
- Contact Person Name
- Andreea SEFERIAN
- Contact Person Email
- a.seferian@institut-myologie.org
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Hopital Jeanne de Flandre - Centre d investigation clinique pediatrique
- Principal Investigator Name
- Jean-Baptiste DAVION
- Principal Investigator Email
- Jeanbaptiste.DAVION@chu-lille.fr
- Contact Person Name
- Jean-Baptiste DAVION
- Contact Person Email
- Jeanbaptiste.DAVION@chu-lille.fr
Italy
- Earliest CTIS Part Ii Submission Date
- 15-07-2025
- Latest Decision Or Authorization Date
- 23-10-2025
- Processing Time Days
- 100
- Number Of Sites
- 3
- Number Of Participants
- 9
Sites
- Site Name
- Centro Clinico Nemo
- Department Name
- Centro Clinico Nemo
- Principal Investigator Name
- Valeria Ada Sansone
- Principal Investigator Email
- valeria.sansone@centrocliniconemo.it
- Contact Person Name
- Valeria Ada Sansone
- Contact Person Email
- valeria.sansone@centrocliniconemo.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Unita Operativa Complessa di Neuropsichiatria infantile
- Principal Investigator Name
- Eugenio Maria Mercuri
- Principal Investigator Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Contact Person Name
- Eugenio Maria Mercuri
- Contact Person Email
- eugeniomaria.mercuri@policlinicogemelli.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- Developmental Neurology Unit
- Principal Investigator Name
- Riccardo Masson
- Principal Investigator Email
- riccardo.masson@istituto-besta.it
- Contact Person Name
- Riccardo Masson
- Contact Person Email
- riccardo.masson@istituto-besta.it
Spain
- Earliest CTIS Part Ii Submission Date
- 01-09-2025
- Latest Decision Or Authorization Date
- 23-10-2025
- Processing Time Days
- 52
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Neuromuscular Disease Unit
- Principal Investigator Name
- Inmaculada Pitarch Castellano
- Principal Investigator Email
- inmapitarch@hotmail.com
- Contact Person Name
- Inmaculada Pitarch Castellano
- Contact Person Email
- inmapitarch@hotmail.com
- Site Name
- Hospital Sant Joan De Deu Barcelona
- Department Name
- Neuromuscular Unit
- Principal Investigator Name
- Andrés Nascimento Osorio
- Principal Investigator Email
- andres.nascimento@sjd.es
- Contact Person Name
- Andrés Nascimento Osorio
- Contact Person Email
- andres.nascimento@sjd.es
Netherlands
- Earliest CTIS Part Ii Submission Date
- 26-09-2025
- Latest Decision Or Authorization Date
- 21-10-2025
- Processing Time Days
- 25
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Universitair Medisch Centrum Utrecht
- Department Name
- Department of Neurology
- Principal Investigator Name
- Ludo Van der Pol
- Principal Investigator Email
- W.L.vanderPol@umcutrecht.nl
- Contact Person Name
- Ludo Van der Pol
- Contact Person Email
- W.L.vanderPol@umcutrecht.nl
Sponsor
Primary sponsor
- Full Name
- Scholar Rock Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Syneos Health Inc.
- Responsibilities
- Multiple duties (codes present); includes Patient Recruitment, IDMC Oversight
- Name
- PPD Development LP
- Responsibilities
- code 8
- Name
- Charles River Laboratories Inc.
- Responsibilities
- PK/PD/ADA
- Name
- Catalent Cts (Kansas City) LLC
- Responsibilities
- code 14
Third parties
- {"country":"United States","full_name":"Gray Consulting Inc.","duties_or_roles":"Patient Travel Support","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Safety","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"Multiple duties (codes present); includes Patient Recruitment, IDMC Oversight","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"code 8","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code 7","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Netherlands","full_name":"LabConnect Europe B.V.","duties_or_roles":"Kit Production and Long-Term Sample Storage","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Caerus Marketing Group LLC","duties_or_roles":"Patient Recruitment","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"videotaping","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Charles River Laboratories Inc.","duties_or_roles":"PK/PD/ADA","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Labor Dr. Wisplinghoff GbR","duties_or_roles":"Short-Term Storage; code 4","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"IP shipment and depot","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Catalent Cts (Kansas City) LLC","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"LabConnect GmbH","duties_or_roles":"Short-Term Storage; code 9","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Germany","full_name":"PCI Pharma Services Germany GmbH","duties_or_roles":"IP shipment and depot","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- APITEGROMAB
- Active Substance
- APITEGROMAB
- Modality
- Monoclonal antibody
- Routes Of Administration
- INTRAVENOUS USE
- Route
- INTRAVENOUS USE
- Orphan Designation
- Yes
- Combination Treatment
- Yes
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