A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA-2)
20 augusti 2026 uppdaterad av: Regeneron Pharmaceuticals
Phase 3 Evaluation of the Safety, Pharmacokinetics, and Efficacy of Garetosmab (Anti-Activin A Monoclonal Antibody) in Children and Adolescents With Fibrodysplasia Ossificans Progressiva
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)
Studieöversikt
Status
Har inte rekryterat ännu
Betingelser
Intervention / Behandling
Studietyp
Interventionell
Inskrivning (Beräknad)
18
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studiekontakt
- Namn: Clinical Trials Administrator
- Telefonnummer: 844-734-6643
- E-post: clinicaltrials@regeneron.com
Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
Tar emot friska volontärer
Nej
Beskrivning
Key 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
Key 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
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Icke-randomiserad
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Cohort 1: Adolescents
|
Administered per the protocol
Andra namn:
|
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Experimentell: Cohort 2: Children
|
Administered per the protocol
Andra namn:
|
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Experimentell: Cohort 3: Children and Adolescents
|
Administered per the protocol
Andra namn:
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
Tidsram: Baseline to week 28
|
Baseline to week 28
|
|
Occurrence of TEAEs
Tidsram: Baseline to week 56
|
Baseline to week 56
|
|
Severity of TEAEs
Tidsram: Baseline to week 28
|
Baseline to week 28
|
|
Severity of TEAEs
Tidsram: Baseline to week 56
|
Baseline to week 56
|
|
Concentrations of functional garetosmab in serum
Tidsram: Through week 56
|
Through week 56
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Total volume of new Heterotopic Ossification (HO) lesion
Tidsram: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of new HO lesions
Tidsram: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Occurrence of new HO lesions
Tidsram: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of clinician-assessed flare-ups
Tidsram: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Occurrence of clinician-assessed flare-ups
Tidsram: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Number of patient/caregiver-reported flare-ups
Tidsram: Through Week 28 and week 56
|
Through Week 28 and week 56
|
|
|
Occurrence of patient/caregiver-reported flare-ups
Tidsram: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Change from baseline in Tanner puberty scale
Tidsram: 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
|
At week 28 and week 56
|
|
Characteristics of menstrual cycles for female participants who reached menarche
Tidsram: Over 28 weeks and 56 weeks
|
Over 28 weeks and 56 weeks
|
|
|
Height-for-age Z-Scores according to the World Health Organization (WHO) Growth Reference Data for Children
Tidsram: 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.
|
Through week 56
|
|
Concentrations of total activin A in serum
Tidsram: Through week 56
|
Through week 56
|
|
|
Occurence of Anti-Drug Antibody (ADA) to garetosmab
Tidsram: Through week 56
|
Through week 56
|
|
|
Magnitude of ADA to garetosmab
Tidsram: Through week 56
|
Through week 56
|
|
|
Change from baseline in hearing function as assessed by audiometry
Tidsram: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores
Tidsram: 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.
|
At week 28 and week 56
|
|
Acceptability and tolerability assessment via exit interview
Tidsram: 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.
|
Up to week 30
|
Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Utredare
- Studierektor: Clinical Trial Management, Regeneron Pharmaceuticals
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart (Beräknad)
2 februari 2027
Primärt slutförande (Beräknad)
10 december 2028
Avslutad studie (Beräknad)
21 december 2029
Studieregistreringsdatum
Först inskickad
22 april 2026
Först inskickad som uppfyllde QC-kriterierna
22 april 2026
Första postat (Faktisk)
30 april 2026
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
24 augusti 2026
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
20 augusti 2026
Senast verifierad
1 augusti 2026
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- R2477-FOP-2413
- 2024-518415-19-01 (Ctis)
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
JA
IPD-planbeskrivning
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
Tidsram för IPD-delning
When Regeneron has:
- received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
- made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry)
- the legal authority to share the data, and
- ensured the ability to protect participant privacy
Kriterier för IPD Sharing Access
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli.
Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
IPD-delning som stöder informationstyp
- STUDY_PROTOCOL
- SAV
- ICF
- ANALYTIC_CODE
- CSR
Läkemedels- och apparatinformation, studiedokument
Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Ja
Studerar en amerikansk FDA-reglerad produktprodukt
Nej
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