This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data. Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate…
ID
Source
Brief title
Condition
- Musculoskeletal and connective tissue disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Total volume of new HO as assessed by low dose WBCT (excluding the head) at
Week 24.
Secondary outcome
• Total number of new flares (annualized) from baseline to Week 24.
• Proportion of participants with a clinically meaningful improvement in the
flare-related symptoms assessed by the FOP-PROMPT at Week 24.
• Frequency and severity of AEs and SAEs, including the results of vital signs,
ECGs, echocardiograms, physical examinations, PFTs, BMD, laboratory data, and
knee epiphyseal closure (>= 12 to < 21 years of age).
• Proportion of participants with new HO over the 24-week period.
• Total number of new HO lesions as assessed by low-dose WBCT (excluding the
head) from baseline to Week 24.
• Total volume of new HO 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 double-blind period.
• Total number of new flares (annualized) from Week 24 to Week 48 compared to
baseline to Week 24 in participants randomized to placebo during the
double-blind period.
• Proportion of participants with new HO from Week 24 to Week 48 compared to
baseline to Week 24 in participants randomized to placebo during the
double-blind 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 double-blind period.
• INCB000928 PK for plasma and or saliva: Cmax, tmax, Cmin, and AUCt.
Background summary
See protocol section 2.1
Study objective
This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data.
Primary:
To determine the efficacy of INCB000928 for the prevention of new HO in
participants with FOP.
Key Secondary:
To further evaluate the efficacy of INCB000928 in the reduction of flares and
improvement in flare-related symptoms.
Secondary:
- To further evaluate the efficacy of INCB000928 in the reduction of flares and
improvement in flare-related symptoms.
- To evaluate the safety and tolerability of INCB000928 in participants with
FOP.
- To further evaluate the efficacy of INCB000928 in participants with FOP.
- To confirm the efficacy of INCB000928 at Week 48 in participants randomized
to placebo during the double-blind period.
- To characterize the PK of INCB000928 in participants with FOP.
Study design
This is a Phase 2, multicenter, randomized, double-blind, placebo-controlled
study to evaluate the efficacy, safety, tolerability, and PK of INCB000928 over
a 24-week treatment period followed by a 148-week, open-label extension period
(including OLE 1 Treatment Period [52 Weeks] and OLE 2
Treatment Period [96 Weeks]);It is estimated that an individual will
participate for approximately 3.5 years.
Intervention
Participants will be randomized 1:1 to the INCB000928 100 mg QD treatment group
or the placebo group (~ 30 participants per group), stratified by joint
function based on CAJIS score at baseline.
Up to 32 days for screening; continuous treatment in the double-blind,
placebo-controlled period for 24 weeks; a 52-week, open-label extension period;
and a 4-week follow-up period. It is estimated that an individual will
participate for approximately 21 months.
Study burden and risks
See protocol section 2.3. Benefit/Risk Assessment,
Augustine Cutt-Off 1801
Wilmington DE 19803
US
Augustine Cutt-Off 1801
Wilmington DE 19803
US
Listed location countries
Age
Inclusion criteria
-Female and male adults and adolescents >= 12 years of age.
-Clinical diagnosis of FOP (based on findings of congenital malformation of the
great
toes, episodic soft-tissue swelling, and/or progressive HO).
- Participant-reported FOP disease activity within 1 year of the screening
visit. This is
defined as pain, swelling, and other signs and symptoms associated with FOP
flare-ups or
worsening of joint function or radiographic progression of HO (increase in site
or number
of HO lesions) with or without an association with flare-up episodes.
Exclusion criteria
-CAJIS score >= 24.
-FOP disease severity that in the investigator's opinion precludes
participation (eg,
ankyloses of most or all joints, symptomatic thoracic insufficiency syndrome, or
recurrent respiratory infections).
-History of uncontrolled or unstable cardiovascular, respiratory, renal,
gastrointestinal,
endocrine, hematopoietic, psychiatric, and/or neurological disease within 6
months of
screening.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EU-CTR | CTIS2023-504129-38-00 |
| EudraCT | EUCTR2021-002286-17-NL |
| ClinicalTrials.gov | NCT05090891 |
| CCMO | NL78845.029.22 |