A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS)

Conditions

Fibrodysplasia Ossificans Progressiva

Brief summary

Occurrence of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24.

Detailed description

• Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Total volume of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Change in the total volume of all HO lesions as assessed by low dose WBCT (excluding the head) from baseline to Week 24., • Number of new flares based on FOP PROMPT from baseline to Week 24., • AEs and SAEs, assessed by changes in vital signs, ECGs, echocardiograms, physical examinations, PFTs, BMD, laboratory data, and knee epiphyseal closure (2 to < 21 years of age)., • Occurrence of new HO lesions as assessed by low dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Total volume of new HO lesions as assessed by low-dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Change in the total volume of all HO lesions as assessed by low dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., INCB000928 PK parameters in plasma and/or saliva: Cmax, tmax, Cmin, and AUCt., • Number of new flares based on FOP PROMPT from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period

Related papers

No related papers found yet.

Interventions

DRUGINCB000928

DRUGPlacebo

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Occurrence of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24.	—

Secondary

Measure	Time frame
• Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Total volume of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Change in the total volume of all HO lesions as assessed by low dose WBCT (excluding the head) from baseline to Week 24., • Number of new flares based on FOP PROMPT from baseline to Week 24., • AEs and SAEs, assessed by changes in vital signs, ECGs, echocardiograms, physical examinations, PFTs, BMD, laboratory data, and knee epiphyseal closure (2 to < 21 years of age)., • Occurrence of new HO lesions as assessed by low dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Total volume of n	—

Measure

Time frame

• Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Total volume of new HO lesions as assessed by low-dose WBCT (excluding the head) from baseline to Week 24., • Change in the total volume of all HO lesions as assessed by low dose WBCT (excluding the head) from baseline to Week 24., • Number of new flares based on FOP PROMPT from baseline to Week 24., • AEs and SAEs, assessed by changes in vital signs, ECGs, echocardiograms, physical examinations, PFTs, BMD, laboratory data, and knee epiphyseal closure (2 to < 21 years of age)., • Occurrence of new HO lesions as assessed by low dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Number of new HO lesions as assessed by low-dose WBCT (excluding the head) from Week 24 to Week 48 compared to baseline to Week 24 in participants randomized to placebo during the DB period., • Total volume of n

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Countries

France, Germany, Italy, Netherlands, Portugal, Spain

Outcome results

None listed