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Fibrodyplasia Ossificans Progressiva (FOP)Phase 3NCT04577820

Study to Assess the Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans Progressiva (FOP)

A Phase 3 study testing garetosmab in people with Fibrodyplasia Ossificans Progressiva (FOP) and Heterotopic Ossification (HO), run by Regeneron Pharmaceuticals.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

The primary safety objective of the study is to assess the safety and tolerability of garetosmab in Japanese male and female adult patients with FOP. The primary efficacy objective of the study is to assess the effect of garetosmab on Heterotopic ossification (HO) in Japanese adult patients with FOP, as determined by… Read more about this study

Who is it for?
Ages 18 to 60 years
What is being tested?
garetosmab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
13 Oct 2021to 8 Oct 2022
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 60 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive HO)
  • Confirmation of FOP diagnosis with documentation of any Type I activin A receptor (ACVR1) mutation
  • FOP disease activity, as defined in the protocol, within 1 year of screening visit
  • Willing and able to undergo PET and CT imaging procedures and other procedures as defined in this study
  • Able to understand and complete study-related questionnaires and diaries (assistance from caregivers is allowed)

Who cannot join

  • Patient has significant concomitant illness or history of significant illness such as but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study
  • Previous history or diagnosis of cancer
  • Severely impaired renal function defined as estimated glomerular filtration rate <30 mL/min/1.73 m2 calculated by the Modification of Diet in Renal Disease equation (1 retest is allowed)
  • Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) >9% at screening (1 retest allowed)
  • History of severe respiratory compromise, as defined in protocol
  • Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures
  • Pregnant or breastfeeding women
  • NOTE: Other protocol defined inclusion/exclusion criteria apply.

About this study

Official title: Evaluation of Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans Progressiva

The primary safety objective of the study is to assess the safety and tolerability of garetosmab in Japanese male and female adult patients with FOP.

The primary efficacy objective of the study is to assess the effect of garetosmab on Heterotopic ossification (HO) in Japanese adult patients with FOP, as determined by the number of new heterotopic bone lesions identified by computed tomography (CT).

Being tested
garetosmab
Medical subject headings
Ossification, Heterotopic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • garetosmabDrug

    Also called REGN2477

    Repeated doses administered intravenously (IV) every four weeks (Q4W)

Study groups in NCT04577820
Study groupRoleWhat they receive
garetosmabExperimentalNot described
Receives: Drug: garetosmab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and severity of treatment-emergent adverse event (TEAEs)

    Measured: Through week 28

  2. Number of new HO lesions as assessed by CT

    Measured: At week 28

Secondary outcomes (39)

  1. Total volume of new HO lesions as assessed by CT

    Measured: At week 28

  2. Number of new HO lesions as assessed by positron emission tomography (PET)

    Measured: At week 28

  3. Total lesion activity in new HO lesions as assessed by PET

    Measured: At week 28

  4. Percent of patients with new HO lesions as assessed by CT

    Measured: At week 28

  5. Percent of patients with new HO lesions as assessed by PET

    Measured: At week 28

  6. Percent of patients with investigator-assessed flare-ups

    Measured: Baseline to week 28

  7. Percent of patients with investigator-assessed flare-ups

    Measured: Baseline to week 56

  8. Percent of patients with flare-ups assessed by patient e-diary

    Measured: Baseline to week 28

  9. Percent of patients with flare-ups assessed by patient e-diary

    Measured: Baseline to week 56

  10. Number of new HO lesions as assessed by CT

    Measured: At week 56

  11. Total volume of new HO lesions as assessed by CT

    Measured: At week 56

  12. Percent of patients with new HO lesions as assessed by CT

    Measured: At week 56

  13. Number of new HO lesions as assessed by PET

    Measured: At week 56

  14. Total lesion activity in new HO lesions as assessed by PET

    Measured: At week 56

  15. Percent of patients with new HO lesions as assessed by PET

    Measured: At week 56

  16. Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET

    Measured: Baseline and week 28

  17. Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET

    Measured: Baseline and week 56

  18. Percent change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET

    Measured: Baseline and week 28

  19. Percent change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET

    Measured: Baseline and week 56

  20. Change in total lesion activity by PET

    Measured: Baseline and week 28

  21. Change in total lesion activity by PET

    Measured: Baseline and week 56

  22. Percent change in total lesion activity by PET

    Measured: Baseline and week 28

  23. Percent change in total lesion activity by PET

    Measured: Baseline and week 56

  24. Change in the total volume of HO lesions as assessed by CT

    Measured: Baseline and week 28

  25. Change in the total volume of HO lesions as assessed by CT

    Measured: Baseline and week 56

  26. Percent change in the total volume of HO lesions as assessed by CT

    Measured: Baseline and week 28

  27. Percent change in the total volume of HO lesions as assessed by CT

    Measured: Baseline and week 56

  28. Change in number of HO lesions as assessed by PET

    HO lesions defined as target and new lesions relative to baseline.

    Measured: Baseline and week 28

  29. Change in number of HO lesions as assessed by PET

    Defined above

    Measured: Baseline and week 56

  30. Change in the number of HO lesions detectable by CT

    Defined above

    Measured: Baseline and week 28

  31. Change in the number of HO lesions detectable by CT

    Defined above

    Measured: Baseline and week 56

  32. Time-weighted average (standardized area under curve [AUC]) change in daily pain due to FOP, as measured using the daily numeric rating scale (NRS)

    The NRS is a categorical rating scale used by patients to rate their pain associated with FOP. Patients will be asked to rate their pain on a scale that ranges from "0" (no pain) to "10" (worst possible pain).

    Measured: Baseline through week 28

  33. Time-weighted average (standardized AUC) change in daily pain due to FOP, as measured using the daily NRS

    Measured: Baseline through week 56

  34. Total dosage of glucocorticoids use

    Measured: Through week 56

  35. Incidence and severity of TEAEs

    Measured: Through week 56

  36. Concentration of total activin A in serum over time

    Measured: Through week 56

  37. Pharmacokinetic (Pk) Profile - concentrations of garetosmab in serum over time

    Measured: Through week 56

  38. Immunogenicity as measured by Anti-drug antibodies (ADA) to garetosmab over time

    Measured: Through week 28

  39. Percent change from baseline in biomarkers of bone formation levels in serum

    Measured: Through week 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Oct 2020
  2. Study start13 Oct 2021 (estimated)
  3. Primary completion1 Mar 2022 (estimated)
  4. Study completion8 Oct 2022 (estimated)

Why it stopped: Phase 2 Study R2477-FOP-1940 has been withdrawn and the next phase of the development program is being planned

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04577820
Sponsor's protocol ID
R2477-FOP-1940
Status last verified
Oct 2021
First submitted
30 Sep 2020
Last updated
1 Nov 2021
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing garetosmab in people with Fibrodyplasia Ossificans Progressiva (FOP) and Heterotopic Ossification (HO), run by Regeneron Pharmaceuticals.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 1 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of treatment-emergent adverse event (TEAEs) (time frame: Through week 28). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04577820.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Nov 2021.

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