Register a trial

Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements. New passwords must include at least one lowercase letter, one uppercase letter, one number and one special character (e.g. !#$%&@).
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT05090891
Ethics application status
Date submitted
8/10/2021
Date registered
25/10/2021

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: Cohort 1 - Participants (= 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Experimental: Cohort 2 - Participants (6 to \< 12 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.

Experimental: Cohort 3 - Participants (2 to \< 6 years of age) will receive INCB000928 or placebo as defined in the protocol for 24 weeks (double-blind period). Participants who complete the double-blind period will continue into open-label extension period for an additional 292 weeks.


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: Occurrence of new heterotopic ossification (HO) lesions from baseline
Timepoint [1] 0 0
Week 24
Secondary outcome [1] 0 0
Double Blind Period: Number of new HO lesions from baseline
Timepoint [1] 0 0
Week 24
Secondary outcome [2] 0 0
Double Blind Period: Total volume of new HO lesions from baseline
Timepoint [2] 0 0
Week 24
Secondary outcome [3] 0 0
Double Blind Period: Change in the total volume of all HO lesions from baseline
Timepoint [3] 0 0
Week 24
Secondary outcome [4] 0 0
Double Blind Period: Number of new flares from baseline
Timepoint [4] 0 0
Week 24
Secondary outcome [5] 0 0
Number of Participants with Treatment Emergent Adverse Events (TEAE)
Timepoint [5] 0 0
Up to 316 weeks
Secondary outcome [6] 0 0
Open-Label Extension: Occurrence of new HO lesions from Week 24
Timepoint [6] 0 0
Week 48
Secondary outcome [7] 0 0
Open-Label Extension: Number of new HO lesions from Week 24
Timepoint [7] 0 0
Week 48
Secondary outcome [8] 0 0
Open-Label Extension: Total volume of new HO lesions from Week 24
Timepoint [8] 0 0
Week 48
Secondary outcome [9] 0 0
Open-Label Extension: Change in the total volume of all HO lesions from Week 24
Timepoint [9] 0 0
Week 48
Secondary outcome [10] 0 0
Open-Label Extension: Number of new flares from Week 24
Timepoint [10] 0 0
Week 48
Secondary outcome [11] 0 0
Pharmacokinetics Parameter: Cmax of INCB000928
Timepoint [11] 0 0
Baseline, Weeks 12, 24, 48 and 76
Secondary outcome [12] 0 0
Pharmacokinetics Parameter: Tmax of INCB000928
Timepoint [12] 0 0
Baseline, Weeks 12, 24, 48 and 76
Secondary outcome [13] 0 0
Pharmacokinetics Parameter: Cmin of INCB000928
Timepoint [13] 0 0
Baseline, Weeks 12, 24, 48 and 76
Secondary outcome [14] 0 0
Pharmacokinetics Parameter: AUCt of INCB000928
Timepoint [14] 0 0
Baseline, Weeks 12, 24, 48 and 76

Eligibility
Key inclusion criteria
* Female and male participants:

* Cohort 1: = 12 years of age.
* Cohort 2: 6 to < 12 years of age.
* Cohort 3: 2 to < 6 years of age (after eDMC review of interim data from Cohort 2).
* Clinical 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
2 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
23/12/2032
Actual
Sample size
Target
98
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 postcode(s) [1] 0 0
02065 - St Leonards
Recruitment postcode(s) [2] 0 0
03052 - Parkville
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
South Africa
State/province [19] 0 0
Cape Town
Country [20] 0 0
Spain
State/province [20] 0 0
Madrid
Country [21] 0 0
Turkey
State/province [21] 0 0
Izmir
Country [22] 0 0
United Kingdom
State/province [22] 0 0
Manchester
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Stanmore

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Amanda McBride, 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
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05090891