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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05039515




Registration number
NCT05039515
Ethics application status
Date submitted
2/09/2021
Date registered
9/09/2021

Titles & IDs
Public title
A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).
Scientific title
A Phase 2 Study to Assess the Efficacy and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva in Male and Female Paediatric and Adult Participants.
Secondary ID [1] 0 0
2020-002858-24
Secondary ID [2] 0 0
D-CA-60130-452
Universal Trial Number (UTN)
Trial acronym
FALKON
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fibrodysplasia Ossificans Progressiva 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 - IPN60130
Treatment: Drugs - IPN60130
Treatment: Drugs - Placebo

Experimental: IPN60130 high dosage - Oral capsule, swallowed whole or sprinkled onto food, once daily

Experimental: IPN60130 low dosage - Oral capsule, swallowed whole or sprinkled onto food, once daily

Placebo comparator: Placebo - Oral capsule, swallowed whole or sprinkled onto food, once daily


Treatment: Drugs: IPN60130
Immediate-release capsule containing high dose of the drug substance.

Treatment: Drugs: IPN60130
Immediate-release capsule containing low dose of the drug substance.

Treatment: Drugs: Placebo
Placebo will be supplied as powder filled hard capsules
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized change in HO volume as assessed by low-dose WBCT (excluding the head) in treated participants receiving IPN60130 compared with placebo.
Timepoint [1] 0 0
From baseline to 12 months
Primary outcome [2] 0 0
Incidence of Adverse Events / Serious Adverse Events (AEs/SAE)
Timepoint [2] 0 0
From baseline until the end of study (63 months)
Primary outcome [3] 0 0
Change from baseline in clinically significant abnormal values in laboratory parameters (haematology, biochemistry, and urinalysis)
Timepoint [3] 0 0
From baseline until the end of study (63 months)
Primary outcome [4] 0 0
Change from baseline in physical examination findings
Timepoint [4] 0 0
From baseline until the end of study (63 months)
Primary outcome [5] 0 0
Change from baseline in clinically significant vital signs
Timepoint [5] 0 0
From baseline until the end of study (63 months)
Primary outcome [6] 0 0
Change from baseline in clinically significant Electrocardiogram (ECG) readings
Timepoint [6] 0 0
From baseline until the end of study (63 months)
Secondary outcome [1] 0 0
Change in HO volume of new HO lesions as detected by WBCT in participants receiving IPN60130 compared with placebo recipients
Timepoint [1] 0 0
From baseline up to 12 months
Secondary outcome [2] 0 0
Change in number of HO lesions by WBCT in participants receiving IPN60130 compared with placebo recipients
Timepoint [2] 0 0
From baseline up to 12 months
Secondary outcome [3] 0 0
Flare-up rate and number of flare-up days in participants receiving IPN60130 compared with placebo recipients
Timepoint [3] 0 0
From baseline up to 12 months
Secondary outcome [4] 0 0
The number of body regions with new HO in participants receiving IPN60130 compared with placebo recipients
Timepoint [4] 0 0
From baseline up to 12 months
Secondary outcome [5] 0 0
Change in pain intensity
Timepoint [5] 0 0
From baseline up to 12 months
Secondary outcome [6] 0 0
The proportion of participants with any new HO in participants receiving IPN60130 compared with placebo recipients
Timepoint [6] 0 0
From baseline up to 12 months
Secondary outcome [7] 0 0
Change from baseline in HO volume as detected by WBCT in participants receiving IPN60130 compared with placebo recipients and with participants receiving the standard of care in the Natural history study (NHS)
Timepoint [7] 0 0
From baseline up to 60 months
Secondary outcome [8] 0 0
Change from baseline in Cumulative Analogue Joint Involvement Scale for FOP (CAJIS) by treatment arm compared with placebo recipients and participants receiving the standard of care in the NHS across all available timepoints
Timepoint [8] 0 0
From baseline up to 60 months
Secondary outcome [9] 0 0
Change in the FOP Physical Function Questionnaire (FOP-PFQ) by treatment arm compared with placebo recipients and participants receiving the standard of care in the NHS from baseline across all available timepoints
Timepoint [9] 0 0
From baseline up to 60 months
Secondary outcome [10] 0 0
Pharmacokinetic (PK) parameter: Cmax of IPN60130
Timepoint [10] 0 0
From baseline up to Month 24
Secondary outcome [11] 0 0
PK parameter: AUC of IPN60130
Timepoint [11] 0 0
Every 6 months up to Month 24
Secondary outcome [12] 0 0
PK parameter: Ctrough of IPN60130
Timepoint [12] 0 0
Every 6 months up to Month 24
Secondary outcome [13] 0 0
PK parameter: Cmin of IPN60130
Timepoint [13] 0 0
Every 6 months up to Month 24
Secondary outcome [14] 0 0
Assessment of the exposure-response relationship
Timepoint [14] 0 0
From baseline up to 60 months

Eligibility
Key inclusion criteria
Key

* Participants must be at least 5 years of age, to be confirmed (entry for younger paediatric participants <15 years of age will only be once safety in adult and older paediatric participants =15 years of age has been established) at the time of signing the informed participant/parent consent and, for participants who are minors, age-appropriate assent.
* Participants must be at least 15 years of age at the time of signing the informed participant/parent consent for the main study and, for participants who are minors, age-appropriate assent
* Participants must be clinically diagnosed with FOP, with the R206H ACVR1 mutation or other FOP variants associated with progressive HO.
* Participants must have disease progression in the preceding year of the screening visit.
* Participants who have participated in a prior clinical study using another investigational product for the treatment of FOP may be enrolled after a washout of at least 5 half-lives of the other investigational product. Participants with prior treatment such as, but not limited to, imatinib, isotretinoin, garetosmab, or palovarotene may be enrolled 30 days after discontinuation or after washout of at least 5 half-lives, whichever is longer.

1. Washout period for palovarotene is 30 days
2. Washout period for garetosmab is 4 months
* Participants must be able to perform pulmonary function tests adequately and reliably.
* Participants must be able to have an adequate echocardiography assessment at screening for evaluation of left ventricular structure and function as defined by the protocol.
* Participants must be accessible for treatment and follow-up and be able to undergo all study procedures. Participants living at distant locations from the investigational site must be able and willing to travel to a site for the initial and all on-site follow-up visits. Participants must be able to undergo low-dose WBCT (excluding head) without sedation.
* Body weight =10 kg.
* Abstinent or using two highly effective forms of birth control. Females must also have a negative blood or urine pregnancy test prior to administration of study drug.
* Participants must be capable of giving written, signed, and dated informed participant/parent consent; and for participants who are minors, age-appropriate assent and/or legal guardian consent (performed according to local regulations)

Key
Minimum age
5 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participants with complete heart block and left bundle branch block on screening electrocardiogram.
* Participants with screening echocardiography showing septal or left ventricular free wall thickness >12 mm for adult participants or a z-score >3 compared with population norms for children and adolescent participants or left ventricular ejection fraction (LVEF) <50%.
* Participants with severe mitral or tricuspid regurgitation on echocardiography at screening.
* Participants with significant underlying lung disease requiring supplementary oxygen or forced vital capacity <35% of predicted at screening.
* Participants with uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or another significant disease as judged by the investigator.
* Participants with severe hepatic impairment.
* Concomitant medications that are strong inhibitors (including grapefruit juice) or inducers (including St John's Wort) of cytochrome P450 (CYP) 3A4 activity; or kinase inhibitors such as imatinib.
* Prior use in the past year and concomitant use of bisphosphonates for participants in the PET-CT sub study.
* Concurrent participation in another interventional clinical study, or a noninterventional study with radiographic measures or invasive procedures (e.g. collection of blood or tissue samples).
* Amylase or lipase >2× the upper limit of normal (ULN) or with a history of chronic pancreatitis.
* Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >5×ULN.
* Participants with hematologic abnormalities:

* Hgb<10g/dL
* Platelets<75,000/mm3
* WBC<2000/mm3
* Participants with coagulation test measurements outside of the normal range at screening.

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 administering the treatment/s
The people assessing the outcomes
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/03/2029
Actual
Sample size
Target
Accrual to date
Final
113
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal North Shore Hospital - New South Wales - Sidney
Recruitment postcode(s) [1] 0 0
- Sidney
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
Buenos Aires
Country [5] 0 0
Belgium
State/province [5] 0 0
Leuven
Country [6] 0 0
Canada
State/province [6] 0 0
Edmonton
Country [7] 0 0
Canada
State/province [7] 0 0
Toronto
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
Italy
State/province [11] 0 0
Genoa
Country [12] 0 0
Japan
State/province [12] 0 0
Nagoya
Country [13] 0 0
Japan
State/province [13] 0 0
Tokyo
Country [14] 0 0
Japan
State/province [14] 0 0
Obu
Country [15] 0 0
Korea, Republic of
State/province [15] 0 0
Seoul
Country [16] 0 0
Mexico
State/province [16] 0 0
Ciudad de mexico
Country [17] 0 0
Portugal
State/province [17] 0 0
Lisboa
Country [18] 0 0
Spain
State/province [18] 0 0
Pozuelo De Alarcón
Country [19] 0 0
Spain
State/province [19] 0 0
Valencia
Country [20] 0 0
Sweden
State/province [20] 0 0
Umeå

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Clementia Pharmaceuticals Inc.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Ipsen
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Ipsen Medical Director
Address 0 0
Ipsen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ipsen Clinical Study Enquiries
Address 0 0
Country 0 0
Phone 0 0
see email
Fax 0 0
Email 0 0
clinical.trials@ipsen.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?
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.

Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.
When will data be available (start and end dates)?
Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.
Available to whom?
Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/members/ourmembers/


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/NCT05039515