A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA-2)
20 augustus 2026 bijgewerkt door: 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)
Studie Overzicht
Toestand
Nog niet aan het werven
Interventie / Behandeling
Studietype
Ingrijpend
Inschrijving (Geschat)
18
Fase
- Fase 3
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studiecontact
- Naam: Clinical Trials Administrator
- Telefoonnummer: 844-734-6643
- E-mail: clinicaltrials@regeneron.com
Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
Accepteert gezonde vrijwilligers
Nee
Beschrijving
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
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
|
Experimenteel: Cohort 1: Adolescents
|
Administered per the protocol
Andere namen:
|
|
Experimenteel: Cohort 2: Children
|
Administered per the protocol
Andere namen:
|
|
Experimenteel: Cohort 3: Children and Adolescents
|
Administered per the protocol
Andere namen:
|
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
|
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
Tijdsspanne: Baseline to week 28
|
Baseline to week 28
|
|
Occurrence of TEAEs
Tijdsspanne: Baseline to week 56
|
Baseline to week 56
|
|
Severity of TEAEs
Tijdsspanne: Baseline to week 28
|
Baseline to week 28
|
|
Severity of TEAEs
Tijdsspanne: Baseline to week 56
|
Baseline to week 56
|
|
Concentrations of functional garetosmab in serum
Tijdsspanne: Through week 56
|
Through week 56
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Total volume of new Heterotopic Ossification (HO) lesion
Tijdsspanne: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of new HO lesions
Tijdsspanne: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Occurrence of new HO lesions
Tijdsspanne: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Number of clinician-assessed flare-ups
Tijdsspanne: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Occurrence of clinician-assessed flare-ups
Tijdsspanne: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Number of patient/caregiver-reported flare-ups
Tijdsspanne: Through Week 28 and week 56
|
Through Week 28 and week 56
|
|
|
Occurrence of patient/caregiver-reported flare-ups
Tijdsspanne: Through week 28 and week 56
|
Through week 28 and week 56
|
|
|
Change from baseline in Tanner puberty scale
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: Through week 56
|
Through week 56
|
|
|
Occurence of Anti-Drug Antibody (ADA) to garetosmab
Tijdsspanne: Through week 56
|
Through week 56
|
|
|
Magnitude of ADA to garetosmab
Tijdsspanne: Through week 56
|
Through week 56
|
|
|
Change from baseline in hearing function as assessed by audiometry
Tijdsspanne: At week 28 and week 56
|
At week 28 and week 56
|
|
|
Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores
Tijdsspanne: 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
Tijdsspanne: 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
|
Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Onderzoekers
- Studie directeur: Clinical Trial Management, Regeneron Pharmaceuticals
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Geschat)
2 februari 2027
Primaire voltooiing (Geschat)
10 december 2028
Studie voltooiing (Geschat)
21 december 2029
Studieregistratiedata
Eerst ingediend
22 april 2026
Eerst ingediend dat voldeed aan de QC-criteria
22 april 2026
Eerst geplaatst (Werkelijk)
30 april 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
24 augustus 2026
Laatste update ingediend die voldeed aan QC-criteria
20 augustus 2026
Laatst geverifieerd
1 augustus 2026
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- R2477-FOP-2413
- 2024-518415-19-01 (Ctis)
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
JA
Beschrijving IPD-plan
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
IPD-tijdsbestek voor delen
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
IPD-toegangscriteria voor delen
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 delen Ondersteunend informatietype
- LEERPROTOCOOL
- SAP
- ICF
- ANALYTIC_CODE
- MVO
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Ja
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .