Fibrodysplasia Ossificans Progressiva (FOP)
Conditions
Keywords
Type I Activin A Receptor (ACVR1) FOP-causing mutation
Brief summary
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
DRUGgaretosmab
Administered per the protocol
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. 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 2. Must have a confirmation of FOP diagnosis, as described in the protocol 3. At the time of enrollment, participants must weight: 1. Cohort 1 \> 30 kg 2. Cohort 2 \> 30 kg 3. Cohort 3 ≤ 30 kg Key
Exclusion criteria
1. Cumulative Analog Joint Involvement Scale (CAJIS) score \> 19 at the time of screening 2. Participant has significant concomitant illness or history of significant illness, as described in the protocol 3. Previous history or diagnosis of cancer 4. Ongoing significant viral or bacterial illness, within 2 weeks of the first study drug administration 5. History of severe respiratory compromise requiring oxygen, respiratory support 6. Known history of cerebral vascular malformation 7. Participants with a history of severe, non-traumatic bleeding requiring transfusion or hospitalization for hemodynamic compromise 8. Participants with a known pre-existing medical history of a bleeding diathesis, as described in the protocol NOTE: Other Protocol-defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of Treatment-Emergent Adverse Event (TEAEs) | Baseline to week 28 |
| Occurrence of TEAEs | Baseline to week 56 |
| Severity of TEAEs | Baseline to week 28 |
| Concentrations of functional garetosmab in serum | Through week 56 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Total volume of new Heterotopic Ossification (HO) lesion | At week 28 and week 56 | — |
| Number of new HO lesions | At week 28 and week 56 | — |
| Occurrence of new HO lesions | At week 28 and week 56 | — |
| Number of clinician-assessed flare-ups | Through week 28 and week 56 | — |
| Occurrence of clinician-assessed flare-ups | Through week 28 and week 56 | — |
| Number of patient/caregiver-reported flare-ups | Through Week 28 and week 56 | — |
| Occurrence of patient/caregiver-reported flare-ups | Through week 28 and week 56 | — |
| Change from baseline in Tanner puberty scale | At week 28 and week 56 | Tanner puberty scale or stages: Staging of sexual development is graded on a 5-point ordinal scale ranging from 1 (prepubertal) to 5 (adultlike) for female breast development, male external genitals, and pubic hair |
| Characteristics of menstrual cycles for female participants who reached menarche | Over 28 weeks and 56 weeks | — |
| Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children | Through week 56 | Participants 5-19 years of age Z-score represents standardized measure of how far an individual deviated from study cohort average at baseline. A higher Z-score reflects better performance. |
| Concentrations of total activin A in serum | Through week 56 | — |
| Occurence of Anti-Drug Antibody (ADA) to garetosmab | Through week 56 | — |
| Magnitude of ADA to garetosmab | Through week 56 | — |
| Change from baseline in hearing function as assessed by audiometry | At week 28 and week 56 | — |
| Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores | At week 28 and week 56 | Age-appropriate PedsQL Generic Core Scales will be used to measure HRQoL in children and adolescents. Response options include 5-point Likert scale (or 3-point Likert scale for the young children self-report) for each item asking about experience within the past week. Global scores are transformed to a 0 to 100 scale with higher scores indicating better quality of life. |
| Acceptability and tolerability assessment via exit interview | Up to week 30 | Each interview will be conducted by trained external interviewers following a semi-structured interview guide of questions for the participants about their overall experience in the trial. |
Contacts
CONTACTClinical Trials Administrator
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed