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    Summary
    EudraCT Number:2021-002286-17
    Sponsor's Protocol Code Number:INCB00928-201
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-11-29
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2021-002286-17
    A.3Full title of the trial
    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)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
    A.4.1Sponsor's protocol code numberINCB00928-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05090891
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/025/2022
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorIncyte Corporation
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportIncyte Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIncyte Corporation
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address1801 Augustine Cut-Off
    B.5.3.2Town/ cityWolmington
    B.5.3.3Post codeDE 19803
    B.5.3.4CountryUnited States
    B.5.5Fax number1302 4252734
    B.5.6E-mailregaffairs@incyte.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namezilurgisertib
    D.3.2Product code INCB000928
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNzilurgisertib
    D.3.9.1CAS number 2173390-29-7
    D.3.9.2Current sponsor codeINCB000928
    D.3.9.3Other descriptive nameINCB000928 fumarate dihydrate
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fibrodysplasia ossificans progressiva
    E.1.1.1Medical condition in easily understood language
    Fibrodysplasia ossificans progressiva (FOP)
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10068715
    E.1.2Term Fibrodysplasia ossificans progressiva
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.
    E.2.2Secondary objectives of the trial
    - 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.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent/assent:
    a. For adult participants (≥ 18 years of age), ability to comprehend and willingness to sign an ICF.
    b. For adolescent participants (≥ 12 to < 18 years of age), written informed consent of the parent(s) or legal guardian and written assent from the adolescent participant.
    2. Female and male adults and adolescents ≥ 12 years of age.
    3. Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft-tissue swelling, and/or progressive HO).
    4. 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.
    5. Ability to swallow and retain orally administered tablets, either whole or crushed and dispersed in foods or liquids, or ability to receive and retain crushed tablets via a feeding tube
    6. Willingness to avoid pregnancy or fathering children based on the criteria below.
    a. Male participants with reproductive potential must agree to take appropriate precautions to avoid fathering children from screening through 90 days after the last dose of study drug and must refrain from donating sperm during this period.
    b. Female participants of childbearing potential must have a negative pregnancy test at screening (serum) and before the first dose on Day 1 (urine). Female participants of childbearing potential must agree to take appropriate precautions to avoid pregnancy from screening through 190 days after the last dose of study drug
    c. Women without childbearing potential are eligible.
    7. Willing and able to undergo low-dose WBCT (excluding the head) imaging without requiring intubation.
    8. Willing and able to comply with study procedures and requirements and attend all study visits as defined in this Protocol.
    E.4Principal exclusion criteria
    1. Pregnant or breast-feeding.
    2. CAJIS score ≥ 24.
    3. FOP disease severity that in the investigator's opinion precludes participation
    4. History of uncontrolled or unstable cardiovascular, respiratory, renal, gastrointestinal, endocrine, hematopoietic, psychiatric, and/or neurological disease within 6 months of screening.
    5. 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.
    6. Presence of a clinically significant finding on echocardiogram
    7. Presence of an abnormal finding on ECG at screening that in the investigator's opinion is clinically significant and/or the following ECG parameters: QTcF interval > 450 milliseconds, QRS interval > 120 milliseconds, PR interval > 220 milliseconds, ECG evidence of Brugada syndrome, atrial fibrillation or atrial flutter, or Mobitz II or higher grade atrioventricular block.
    8. Current treatment with a potent/strong inhibitor or inducer of CYP3A4 within 5 half-lives before the first dose of study treatment or expected to receive such treatment during the study
    9. Use of the following medications:
    a. Imatinib 30 days prior to baseline
    b. Any medication that might interfere with HO formation in the 30 days or 5 half lives, whichever is longer before baseline.
    10. Participation in an investigational drug study for the treatment of FOP or any other indication within 90 days or 5 half lives, whichever is longer before baseline.
    11. Planning to receive a live vaccine during the course of the study or within 6 weeks after the last dose of study drug.
    12. Known or suspected allergy to INCB000928 or any component of the study drug.
    13. Known history of clinically significant drug or alcohol abuse as defined by the investigator in the l year before baseline
    14. Chronic or current active infectious disease requiring systemic antibiotic, antifungal, or antiviral treatment.
    15. HIV, HBV, or HCV infection.
    16. Participants with laboratory values at screening defined in protocol
    17. Weight < 30 kg at screening.
    18. The following participants are excluded in France: a) Vulnerable populations according to article L.1121-6 of the French Public Health
    Code; b) Adults under legal protection or who are unable to express their consent per article L.1121-8 of the French Public Health Code; c) Individuals not affiliated with the social security system.
    E.5 End points
    E.5.1Primary end point(s)
    • Total volume of new HO as assessed by low-dose WBCT (excluding the head) at Week 24.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24 and Safety assessments will be performed throughout the study
    E.5.2Secondary end point(s)
    • 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.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 24 and Week 48 and Safety assessments will be performed throughout the study
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Chile
    New Zealand
    Switzerland
    Australia
    Brazil
    Canada
    China
    Korea, Republic of
    Mexico
    South Africa
    Turkey
    United Kingdom
    United States
    France
    Germany
    Italy
    Netherlands
    Portugal
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 20
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 38
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 60
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Included in protocol section 6.7
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-11-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-06-28
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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