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A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP) (OPTIMA-2)

22. april 2026 opdateret af: 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)

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

18

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

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/Exclusion Criteria Apply

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Cohort 1: Adolescents
Medicin: garetosmab
Administered per the protocol
Andre navne:
  • REGN2477
Eksperimentel: Cohort 2: Children
Medicin: garetosmab
Administered per the protocol
Andre navne:
  • REGN2477
Eksperimentel: Cohort 3: Children and Adolescents
Medicin: garetosmab
Administered per the protocol
Andre navne:
  • REGN2477

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Tidsramme
Occurrence of Treatment-Emergent Adverse Event (TEAEs)
Tidsramme: Baseline to week 28
Baseline to week 28
Occurrence of TEAEs
Tidsramme: Baseline to week 56
Baseline to week 56
Severity of TEAEs
Tidsramme: Baseline to week 28
Baseline to week 28
Severity of TEAEs
Tidsramme: Baseline to week 56
Baseline to week 56
Concentrations of functional garetosmab in serum
Tidsramme: Through week 56
Through week 56

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Total volume of new Heterotopic Ossification (HO) lesion
Tidsramme: At week 28 and week 56
At week 28 and week 56
Number of new HO lesions
Tidsramme: At week 28 and week 56
At week 28 and week 56
Occurrence of new HO lesions
Tidsramme: At week 28 and week 56
At week 28 and week 56
Number of clinician-assessed flare-ups
Tidsramme: Through week 28 and week 56
Through week 28 and week 56
Occurrence of clinician-assessed flare-ups
Tidsramme: Through week 28 and week 56
Through week 28 and week 56
Number of patient/caregiver-reported flare-ups
Tidsramme: Through Week 28 and week 56
Through Week 28 and week 56
Occurrence of patient/caregiver-reported flare-ups
Tidsramme: Through week 28 and week 56
Through week 28 and week 56
Change from baseline in Tanner puberty scale
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: Through week 56
Through week 56
Occurence of Anti-Drug Antibody (ADA) to garetosmab
Tidsramme: Through week 56
Through week 56
Magnitude of ADA to garetosmab
Tidsramme: Through week 56
Through week 56
Change from baseline in hearing function as assessed by audiometry
Tidsramme: At week 28 and week 56
At week 28 and week 56
Change from baseline in Pediatric Quality of Life inventory (PedsQL) scores
Tidsramme: 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
Tidsramme: 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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Regeneron Pharmaceuticals

Efterforskere

  • Studieleder: Clinical Trial Management, Regeneron Pharmaceuticals

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

30. juli 2026

Primær færdiggørelse (Anslået)

10. december 2028

Studieafslutning (Anslået)

21. december 2029

Datoer for studieregistrering

Først indsendt

22. april 2026

Først indsendt, der opfyldte QC-kriterier

22. april 2026

Først opslået (Faktiske)

30. april 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

30. april 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

22. april 2026

Sidst verificeret

1. marts 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • R2477-FOP-2413
  • 2024-518415-19-01 (Ctis)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.

IPD-delingstidsramme

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-delingsadgangskriterier

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-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Fibrodysplasia Ossificans Progressiva (FOP)

Kliniske forsøg med garetosmab

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Belarus

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner