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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT04577820
Other study ID # R2477-FOP-1940
Secondary ID
Status Withdrawn
Phase Phase 3
First received
Last updated
Start date October 13, 2021
Est. completion date October 8, 2022

Study information

Verified date October 2021
Source Regeneron Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical 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 the number of new heterotopic bone lesions identified by computed tomography (CT).


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
garetosmab
Repeated doses administered intravenously (IV) every four weeks (Q4W)

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Regeneron Pharmaceuticals

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence and severity of treatment-emergent adverse event (TEAEs) Through week 28
Primary Number of new HO lesions as assessed by CT At week 28
Secondary Total volume of new HO lesions as assessed by CT At week 28
Secondary Number of new HO lesions as assessed by positron emission tomography (PET) At week 28
Secondary Total lesion activity in new HO lesions as assessed by PET At week 28
Secondary Percent of patients with new HO lesions as assessed by CT At week 28
Secondary Percent of patients with new HO lesions as assessed by PET At week 28
Secondary Percent of patients with investigator-assessed flare-ups Baseline to week 28
Secondary Percent of patients with investigator-assessed flare-ups Baseline to week 56
Secondary Percent of patients with flare-ups assessed by patient e-diary Baseline to week 28
Secondary Percent of patients with flare-ups assessed by patient e-diary Baseline to week 56
Secondary Number of new HO lesions as assessed by CT At week 56
Secondary Total volume of new HO lesions as assessed by CT At week 56
Secondary Percent of patients with new HO lesions as assessed by CT At week 56
Secondary Number of new HO lesions as assessed by PET At week 56
Secondary Total lesion activity in new HO lesions as assessed by PET At week 56
Secondary Percent of patients with new HO lesions as assessed by PET At week 56
Secondary Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET Baseline and week 28
Secondary Change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET Baseline and week 56
Secondary Percent change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET Baseline and week 28
Secondary Percent change in mean maximum standard uptake volume (SUVmax) of individual active HO site(s) by PET Baseline and week 56
Secondary Change in total lesion activity by PET Baseline and week 28
Secondary Change in total lesion activity by PET Baseline and week 56
Secondary Percent change in total lesion activity by PET Baseline and week 28
Secondary Percent change in total lesion activity by PET Baseline and week 56
Secondary Change in the total volume of HO lesions as assessed by CT Baseline and week 28
Secondary Change in the total volume of HO lesions as assessed by CT Baseline and week 56
Secondary Percent change in the total volume of HO lesions as assessed by CT Baseline and week 28
Secondary Percent change in the total volume of HO lesions as assessed by CT Baseline and week 56
Secondary Change in number of HO lesions as assessed by PET HO lesions defined as target and new lesions relative to baseline. Baseline and week 28
Secondary Change in number of HO lesions as assessed by PET Defined above Baseline and week 56
Secondary Change in the number of HO lesions detectable by CT Defined above Baseline and week 28
Secondary Change in the number of HO lesions detectable by CT Defined above Baseline and week 56
Secondary 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). Baseline through week 28
Secondary Time-weighted average (standardized AUC) change in daily pain due to FOP, as measured using the daily NRS Baseline through week 56
Secondary Total dosage of glucocorticoids use Through week 56
Secondary Incidence and severity of TEAEs Through week 56
Secondary Concentration of total activin A in serum over time Through week 56
Secondary Pharmacokinetic (Pk) Profile - concentrations of garetosmab in serum over time Through week 56
Secondary Immunogenicity as measured by Anti-drug antibodies (ADA) to garetosmab over time Through week 28
Secondary Percent change from baseline in biomarkers of bone formation levels in serum Through week 28