A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: garetosmab.
- Who it may be relevant to
- Registry conditions: Fibrodysplasia Ossificans Progressiva (FOP). Basic parameters: 2 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Center list to be confirmed — check the primary protocol.
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva
Overview
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP. The aim of the study is to see how safe, tolerable, and effective the study drug is. The study is looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the study drug less effective or could lead to side effects)
Interventions
- Drug garetosmab
Administered per the protocol
Primary outcome measures
- Occurrence of Treatment-Emergent Adverse Event (TEAEs) [Time frame: Baseline to week 28]
- Occurrence of TEAEs [Time frame: Baseline to week 56]
- Severity of TEAEs [Time frame: Baseline to week 28]
- Severity of TEAEs [Time frame: Baseline to week 56]
- Concentrations of functional garetosmab in serum [Time frame: Through week 56]
Secondary outcome measures (12)
- Total volume of new Heterotopic Ossification (HO) lesion [Time frame: At week 28 and week 56]
- Number of new HO lesions [Time frame: At week 28 and week 56]
- Occurrence of new HO lesions [Time frame: At week 28 and week 56]
- Number of clinician-assessed flare-ups [Time frame: Through week 28 and week 56]
- Occurrence of clinician-assessed flare-ups [Time frame: Through week 28 and week 56]
- Number of patient/caregiver-reported flare-ups [Time frame: Through Week 28 and week 56]
- Occurrence of patient/caregiver-reported flare-ups [Time frame: Through week 28 and week 56]
- Change from baseline in Tanner puberty scale [Time frame: At week 28 and week 56]
- Characteristics of menstrual cycles for female participants who reached menarche [Time frame: Over 28 weeks and 56 weeks]
- Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children [Time frame: Through week 56]
- Concentrations of total activin A in serum [Time frame: Through week 56]
- Occurence of Anti-Drug Antibody (ADA) to garetosmab [Time frame: Through week 56]
Eligibility criteria
Inclusion criteria
- For USA participants, age criteria are 4 to < 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to < 18 years old
- Must have a confirmation of FOP diagnosis, as described in the protocol
- At the time of enrollment, participants must weight:
- Cohort 1 > 30 kg
- Cohort 2 > 30 kg
- Cohort 3 ≤ 30 kg
Exclusion criteria
- Cumulative Analog Joint Involvement Scale (CAJIS) score > 19 at the time of screening
- Participant has significant concomitant illness or history of significant illness, as described in the protocol
- Previous history or diagnosis of cancer
- Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration
- History of severe respiratory compromise requiring oxygen, respiratory support
- Known history of cerebral vascular malformation
- Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise
- Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol
NOTE: Other Protocol-defined Inclusion/Exclusion Criteria Apply
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Center list to be confirmed — check the primary protocol.
Identifiers
NCT: NCT07559513 · R2477-FOP-2413 · 2024-518415-19-01