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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: 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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
Available to whom?
Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.incyte.com/our-company/compliance-and-transparency


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