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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05090891




Registration number
NCT05090891
Ethics application status
Date submitted
8/10/2021
Date registered
25/10/2021
Date last updated
19/04/2024

Titles & IDs
Public title
To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
Secondary ID [1] 0 0
INCB 00928-201
Universal Trial Number (UTN)
Trial acronym
Progress
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva (FOP) 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Injuries and Accidents 0 0 0 0
Other injuries and accidents

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - INCB000928
Treatment: Drugs - placebo

Experimental: Group A: INCB000928 - Participants will receive INCB000928 for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 52 weeks.

Placebo Comparator: Group B: Placebo followed by INCB000928 - Participants will receive placebo for 24 weeks (double-blind period). Participants who completed the double-blind period will receive INCB000928 in the 52 week open-label extension period.


Treatment: Drugs: INCB000928
INCBG000928 will be administered QD orally.

Treatment: Drugs: placebo
placebo will be administered QD orally
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Double Blind Period: Total volume of new heterotopic ossification (HO)
Timepoint [1] 0 0
Week 24
Secondary outcome [1] 0 0
Double-blind Period: Total number of new flares
Timepoint [1] 0 0
Baseline, Week 12 and Week 24
Secondary outcome [2] 0 0
Double-blind Period: Proportion of participants with a clinically meaningful improvement in the flare-related symptoms
Timepoint [2] 0 0
Baseline, Week 12 and Week 24
Secondary outcome [3] 0 0
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Timepoint [3] 0 0
Up to 80 weeks

Eligibility
Key inclusion criteria
- Female and male adults and adolescents = 12 years of age with a diagnosis of FOP.

- Willingness to avoid pregnancy or fathering children based on the criteria below.

- Willing and able to undergo low-dose WBCT (excluding the head) imaging without
requiring intubation.

- Further inclusion criteria apply.
Minimum age
12 Years
Maximum age
99 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Pregnant or breast-feeding.

- CAJIS score = 24.

- FOP disease severity that in the investigator's opinion precludes participation.

- Any clinically significant medical condition other than FOP that would, in the
investigator's judgment, interfere with full participation in the study, pose a
significant risk to the participant, or interfere with interpretation of study data.

- Chronic or current active infectious disease requiring systemic antibiotic,
antifungal, or antiviral treatment.

- HIV, HBV, or HCV infection. Note:

- Further exclusion criteria apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/04/2028
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal North Shore Hospital - St Leonards
Recruitment hospital [2] 0 0
Murdoch Children'S Research Institute - Parkville
Recruitment hospital [3] 0 0
Westmead Hospital - Westmead
Recruitment postcode(s) [1] 0 0
02065 - St Leonards
Recruitment postcode(s) [2] 0 0
03052 - Parkville
Recruitment postcode(s) [3] 0 0
02145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Minnesota
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania
Country [4] 0 0
Argentina
State/province [4] 0 0
Ciudad Autonoma Buenos Aires
Country [5] 0 0
Brazil
State/province [5] 0 0
San Paolo
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
Chile
State/province [7] 0 0
Santiago
Country [8] 0 0
China
State/province [8] 0 0
Beijing
Country [9] 0 0
China
State/province [9] 0 0
Shanghai
Country [10] 0 0
France
State/province [10] 0 0
Paris
Country [11] 0 0
Germany
State/province [11] 0 0
Koln
Country [12] 0 0
Italy
State/province [12] 0 0
Genova
Country [13] 0 0
Italy
State/province [13] 0 0
Rome
Country [14] 0 0
Korea, Republic of
State/province [14] 0 0
Seoul
Country [15] 0 0
Mexico
State/province [15] 0 0
Tlalpan
Country [16] 0 0
Netherlands
State/province [16] 0 0
Amsterdam
Country [17] 0 0
New Zealand
State/province [17] 0 0
Auckland
Country [18] 0 0
Portugal
State/province [18] 0 0
Lisbon
Country [19] 0 0
Russian Federation
State/province [19] 0 0
Irkutsk
Country [20] 0 0
Russian Federation
State/province [20] 0 0
Moscow
Country [21] 0 0
Russian Federation
State/province [21] 0 0
St. Petersburg
Country [22] 0 0
South Africa
State/province [22] 0 0
Cape Town
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid
Country [24] 0 0
Turkey
State/province [24] 0 0
Izmir
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Manchester
Country [26] 0 0
United Kingdom
State/province [26] 0 0
Stanmore

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Incyte Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the
Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a
clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05090891
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kurt Brown, MD
Address 0 0
Incyte Corporation
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Incyte Corporation Call Center (US)
Address 0 0
Country 0 0
Phone 0 0
1.855.463.3463
Fax 0 0
Email 0 0
medinfo@incyte.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05090891