272. 進行性骨化性線維異形成症 Fibrodysplasia ossificans progressiva Clinical trials / Disease details


臨床試験数 : 40 薬物数 : 36 - (DrugBank : 6) / 標的遺伝子数 : 27 - 標的パスウェイ数 : 95

  
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PhaseCountries
1NCT05027802
(ClinicalTrials.gov)
March 15, 202225/8/2021A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years With Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit From Palovarotene Therapy.Fibrodysplasia Ossificans Progressiva (FOP)Drug: PalovaroteneIpsenNULLNot yet recruiting14 YearsN/AAll87Phase 3NULL
2EUCTR2020-002858-24-SE
(EUCTR)
24/01/202227/11/2020A study assessing the efficacy and safety of an investigational drug called IPN60130 for the treatment of fibrodysplasia ossificans progressiva in participants aged 5 years of age and olderA Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older - FALKON fibrodysplasia ossificans progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: IPN60130 10 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Product Name: IPN60130 50 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Clementia Pharmaceuticals Inc, an Ipsen CompanyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2United States;Spain;Russian Federation;Colombia;Italy;United Kingdom;France;Mexico;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;China;Korea, Republic of;Sweden
3NCT05090891
(ClinicalTrials.gov)
December 20, 20218/10/2021To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans ProgressivaA Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans ProgressivaFibrodysplasia Ossificans Progressiva (FOP)Drug: INCB000928;Drug: placeboIncyte CorporationNULLNot yet recruiting12 Years99 YearsAll60Phase 2NULL
4NCT05039515
(ClinicalTrials.gov)
December 1, 20212/9/2021Study to Assess the Efficacy and Safety of 2 Dosage Regimens of Oral IPN60130 for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP).A Phase 2, Two-part, Placebo-controlled, Parallel-group, Double-blind Study to Assess the Efficacy and Safety of 2 Dosage Regimens of Oral IPN60130 for the Treatment of Fibrodysplasia Ossificans Progressiva in Male and Female Participants 5 Years of Age and Older.Fibrodysplasia Ossificans ProgressivaDrug: IPN60130;Drug: PlaceboClementia Pharmaceuticals Inc.IpsenRecruiting5 YearsN/AAll90Phase 2United States;France;Italy;Korea, Republic of;Spain;Sweden;United Kingdom
5EUCTR2020-002858-24-ES
(EUCTR)
19/10/202127/07/2021A study assessing the efficacy and safety of an investigational drug called IPN60130 for the treatment of fibrodysplasia ossificans progressiva in participants aged 5 years of age and olderA Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older fibrodysplasia ossificans progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: IPN60130 10 mg capsules
INN or Proposed INN: not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Product Name: IPN60130 50 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Clementia Pharmaceuticals Inc, an Ipsen CompanyNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of
6NCT04577820
(ClinicalTrials.gov)
October 13, 202130/9/2020Study to Assess the Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans Progressiva (FOP)Evaluation of Efficacy and Safety of Garetosmab in Japanese Adult Patients With Fibrodysplasia Ossificans ProgressivaFibrodyplasia Ossificans Progressiva (FOP);Heterotopic Ossification (HO)Drug: garetosmabRegeneron PharmaceuticalsNULLWithdrawn18 Years60 YearsAll0Phase 3NULL
7EUCTR2020-002858-24-IT
(EUCTR)
09/06/202104/06/2021A study assessing the efficacy and safety of an investigational drug calledIPN60130 for the treatment of fibrodysplasia ossificans progressiva inparticipants aged 5 years of age and olderA Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older - - fibrodysplasia ossificans progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: IPN60130 10 mg capsules
Product Code: [-]
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate
Product Name: IPN60130 50 mg capsules
Product Code: [-]
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate
CLEMENTIA PHARMACEUTICALS INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2United States;Spain;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Germany;Netherlands;China;Japan;Sweden;Korea, Republic of
8NCT04818398
(ClinicalTrials.gov)
April 1, 202124/3/2021Study of Single-Ascending Doses of DS-6016a in Healthy Japanese SubjectsSingle-Ascending Dose Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of DS-6016a After Subcutaneous Injection in Healthy Japanese SubjectsFibrodysplasia Ossificans ProgressivaDrug: DS-6016a;Drug: PlaceboDaiichi Sankyo Co., Ltd.NULLActive, not recruiting20 Years45 YearsMale48Phase 1Japan
9NCT04307953
(ClinicalTrials.gov)
August 5, 202011/3/2020Saracatinib Trial TO Prevent FOPSaracatinib Trial TO Prevent FOPFibrodysplasia Ossificans ProgressivaDrug: AZD0530 Difumarate;Drug: Matching placeboVU University Medical CenterRoyal National Orthopaedic Hospital NHS Trust;Klinikum Garmisch-Patenkirchen;University of Oxford;Brigham and Women's Hospital;AstraZeneca;Innovative Medicines InitiativeRecruiting18 Years65 YearsAll20Phase 2Germany;Netherlands;United Kingdom
10EUCTR2019-003324-20-NL
(EUCTR)
08/01/202016/10/2019A clinical trial to judge the safety, tolerability and effects on abnormal bone formation of reseach medication AZD 0530 (sarcatinib) in patients with FOPSaracatinib trial TO Prevent FOP - STOPFOP Fibrodyplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Saracatinib
Product Code: AZD0530
INN or Proposed INN: Saracatinib
Other descriptive name: SARACATINIB DIFUMARATE
VU University Medical CenterNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Netherlands
11EUCTR2016-005035-33-ES
(EUCTR)
17/01/201922/01/2019A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans ProgressivaA randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva - LUMINA-1 Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: REGN2477
Product Code: REGN2477
INN or Proposed INN: N/A
Other descriptive name: REGN2477
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;France;Mexico;Canada;Poland;Spain;Netherlands;Colombia;United Kingdom;Italy
12NCT04829773
(ClinicalTrials.gov)
January 3, 20191/4/2021Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult SubjectsAn Open-Label Study Evaluating the Effect of Food on the Pharmacokinetics of Palovarotene and the Effect of Palovarotene on the Pharmacokinetics of the CYP3A4 Substrate Midazolam in Two Cohorts of Healthy Adult SubjectsFibrodysplasia Ossificans ProgressivaDrug: Palovarotene;Drug: midazolamClementia Pharmaceuticals Inc.NULLCompleted18 Years55 YearsAll48Phase 1United States
13EUCTR2017-002541-29-NL
(EUCTR)
16/07/201806/02/2018 A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP) A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLNA Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden
14EUCTR2017-002541-29-IT
(EUCTR)
15/03/201826/01/2021A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: not applicable
Product Name: PALOVAROTENE
Product Code: not assigned
INN or Proposed INN: Palovarotene
Other descriptive name: PALOVAROTENE
Trade Name: not applicable
Product Name: PALOVAROTENE 1.5 mg
Product Code: [not applicable]
INN or Proposed INN: PALOVAROTENE
Other descriptive name: Palovarotene
Trade Name: not applicable
Product Name: PALOVAROTENE-2 mg
Product Code: [not assigned]
INN or Proposed INN: PALOVAROTENE
Other descriptive name: Palovarotene
Trade Name: not applicable
Product Name: PALOVAROTENE
Product Code: [not applicable]
INN or Proposed INN: PALOVAROTENE
Other descriptive name: palovarotene
Trade Name: not applicable
Product Name: PALOVAROTENE-3 mg
Product Code: [not applicable]
INN or Proposed INN: PALOVAROTENE
Other descriptive name: Palovarotene
Trade Name: not applicable
Product Name: PALOVAROTENE-4 mg
CLEMENTIA PHARMACEUTICALS INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
107Phase 3United States;Spain;Korea, Democratic People's Republic of;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden;Korea, Republic of
15EUCTR2016-005035-33-NL
(EUCTR)
05/03/201804/07/2017A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans ProgressivaA randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva - LUMINA-1 Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: REGN2477
Product Code: REGN2477
INN or Proposed INN: N/A
Other descriptive name: REGN2477
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
40Phase 2France;United States;Argentina;Spain;Brazil;Netherlands;United Kingdom;Italy
16NCT03188666
(ClinicalTrials.gov)
February 26, 201813/6/2017A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans ProgressivaA Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans ProgressivaFibrodysplasia Ossificans ProgressivaDrug: REGN2477;Drug: Matching placeboRegeneron PharmaceuticalsNULLCompleted18 Years60 YearsAll44Phase 2United States;Canada;France;Italy;Netherlands;Poland;Spain;United Kingdom;Colombia
17EUCTR2016-005035-33-IT
(EUCTR)
15/12/201708/03/2018A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans ProgressivaA randomized, placebo-controlled study to assess the safety, tolerability,pharmacokinetics, and effects on heterotopic bone formation of REGN2477in patients with Fibrodysplasia Ossificans Progressiva - LUMINA-1 Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: REGN2477
Product Code: REGN2477
Other descriptive name: REGN2477
REGENERON PHARMACEUTICALS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 2France;United States;Argentina;Brazil;Netherlands;United Kingdom;Italy
18EUCTR2017-002541-29-GB
(EUCTR)
14/12/201711/09/2017 A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP) A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
107 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;Sweden
19EUCTR2017-002541-29-SE
(EUCTR)
05/12/201720/09/2017A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
107Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;Sweden
20NCT03312634
(ClinicalTrials.gov)
November 28, 20179/10/2017An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)Fibrodysplasia Ossificans ProgressivaDrug: PalovaroteneClementia Pharmaceuticals Inc.NULLActive, not recruiting4 YearsN/AAll110Phase 3United States;Argentina;Australia;Brazil;Canada;France;Italy;Japan;Spain;Sweden;United Kingdom
21EUCTR2017-002541-29-ES
(EUCTR)
14/11/201728/09/2017A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
80Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden
22EUCTR2016-005035-33-GB
(EUCTR)
15/08/201722/05/2017A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans ProgressivaA randomized, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and effects on heterotopic bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva - LUMINA-1 Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: REGN2477
Product Code: REGN2477
INN or Proposed INN: N/A
Other descriptive name: REGN2477
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 2United States;France;Mexico;Canada;Spain;Poland;Netherlands;Colombia;Italy;United Kingdom
23NCT02979769
(ClinicalTrials.gov)
November 28, 201623/11/2016An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in People With Fibrodysplasia Ossificans Progressiva (FOP) in FranceA Phase 2, Open-Label Extension, Efficacy and Safety Study of a Retinoic Acid Receptor Gamma (RAR?)-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)Fibrodysplasia Ossificans ProgressivaDrug: PalovaroteneClementia Pharmaceuticals Inc.NULLActive, not recruiting6 Years65 YearsAll9Phase 2France
24NCT02521792
(ClinicalTrials.gov)
December 7, 20156/8/2015In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) SubjectsA Phase 2, In-Home, Safety and Efficacy Evaluation of Episodic Administration of Open-Label Palovarotene in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)Fibrodysplasia Ossificans ProgressivaDrug: PalovaroteneClementia Pharmaceuticals Inc.NULLTerminated6 Years65 YearsAll6Phase 2United States;France;United Kingdom
25EUCTR2014-002496-28-GB
(EUCTR)
13/11/201504/09/2015Extension Study of an Investigational Drug, Palovarotene, in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RAR?-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP) Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossification (HO) in muscles, tendons, and ligaments. Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. Prognosis is poor and median life expectancy is 40 years.
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
Product Code: Palovarotene
INN or Proposed INN: Palovarotene
Other descriptive name: Palovarotene
Clementia Pharmaceuticals Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2United States;Argentina;Australia;United Kingdom
26EUCTR2014-001453-17-GB
(EUCTR)
07/07/201525/02/2015The Study of an Investigational Drug, Palovarotene, in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva.A Phase 2 Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of a RAR?-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP). Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossification (HO) in muscles, tendons, and ligaments. Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. Prognosis is poor and median life expectancy is 40 years.
MedDRA version: 18.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
Product Code: Palovarotene
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: Palovarotene
Product Code: Palovarotene
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals Inc.NULLNot RecruitingFemale: yes
Male: yes
40Phase 2United States;Argentina;United Kingdom
27NCT02279095
(ClinicalTrials.gov)
October 27, 201426/10/2014An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)A Phase 2, Open-Label Extension, Efficacy and Safety Study of a Retinoic Acid Receptor Gamma (RAR?) Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)Fibrodysplasia Ossificans ProgressivaDrug: Palovarotene dose level 1;Drug: Palovarotene dose level 2;Drug: Palovarotene dose level 3;Drug: Palovarotene dose level 4Clementia Pharmaceuticals Inc.NULLActive, not recruiting6 Years65 YearsAll54Phase 2United States;Argentina;Australia;France;United Kingdom
28NCT02190747
(ClinicalTrials.gov)
July 201413/7/2014An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP SubjectsA Phase 2 Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of a RAR?-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)Fibrodysplasia Ossificans ProgressivaDrug: Palovarotene;Drug: PlaceboClementia Pharmaceuticals Inc.NULLCompleted6 YearsN/AAll40Phase 2United States;France;United Kingdom
29JPRN-UMIN000019348
2010/07/0114/10/2015Approved calcium channel blocker in the treatment of fibrodysplasia ossificans progressiva (FOP) fibrodysplasia ossificans progressivaoral administration of perhexiline maleate for one yearNagoya University Graduate School of MedicineNULLComplete: follow-up complete15years-old65years-oldMale and Female5Phase 1,2Japan
30EUCTR2017-002541-29-Outside-EU/EEA
(EUCTR)
15/07/2021A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: Palovarotene
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
Phase 3Australia;Brazil;Canada;Japan;United States;Argentina
31EUCTR2017-002541-29-DE
(EUCTR)
29/09/2017A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP)A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE - 1mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 1.5mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 2 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 2.5 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 3 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 4 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 5 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Product Name: PALOVAROTENE - 10 mg
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
80Phase 3United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden
32EUCTR2021-002286-17-FR
(EUCTR)
22/10/2021A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans ProgressivaA Phase 2, Randomized, Double-Blind, Placebo-Controlled Study toEvaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva Fibrodysplasia ossificans progressiva
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: INCB000928
Other descriptive name: INCB000928 fumarate dihydrate
Incyte CorporationNULLNAFemale: yes
Male: yes
60Phase 2United States;France;Canada;Brazil;Australia;Russian Federation;Netherlands;Germany;United Kingdom;Korea, Republic of
33EUCTR2017-002541-29-FR
(EUCTR)
20/10/2017 A clinical trial to evaluate the Efficacy and Safety of Oral Palovarotene as a treatment for Fibrodysplasia Ossificans Progressiva (FOP) A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) - MOVE Trial Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PALOVAROTENE
INN or Proposed INN: PALOVAROTENE
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals IncNULLNA Female: yes
Male: yes
80 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden
34EUCTR2014-002496-28-Outside-EU/EEA
(EUCTR)
15/07/2021Extension Study of an Investigational Drug, Palovarotene, in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RAR?-Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP). Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossification (HO) in muscles, tendons, and ligaments. Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. Prognosis is poor and median life expectancy is 40 years.
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
INN or Proposed INN: Palovarotene
Other descriptive name: PALOVAROTENE
Clementia Pharmaceuticals Inc.NULLNAFemale: yes
Male: yes
Phase 2Australia;United States;Argentina
35EUCTR2021-002244-70-SE
(EUCTR)
25/10/2021A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit from Palovarotene Therapy. - PIVOINE Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
Product Code: IPN60120
INN or Proposed INN: Palovarotene
Other descriptive name: PALOVAROTENE
Ipsen PharmaNULLNAFemale: yes
Male: yes
61Phase 3France;United States;Canada;Argentina;Spain;Brazil;Australia;United Kingdom;Italy;Sweden
36EUCTR2020-002858-24-DE
(EUCTR)
27/11/2020A study assessing the efficacy and safety of an investigational drug called IPN60130 for the treatment of fibrodysplasia ossificans progressiva in participants aged 5 years of age and olderA Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older fibrodysplasia ossificans progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: IPN60130 10 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Product Name: IPN60130 50 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Clementia Pharmaceuticals Inc, an Ipsen CompanyNULLNAFemale: yes
Male: yes
90Phase 2United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of
37EUCTR2021-002286-17-DE
(EUCTR)
04/11/2021A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans ProgressivaA Phase 2, Randomized, Double-Blind, Placebo-Controlled Study toEvaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva Fibrodysplasia ossificans progressiva
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: INCB000928
Other descriptive name: INCB000928 fumarate dihydrate
Incyte CorporationNULLNAFemale: yes
Male: yes
60Phase 2France;United States;Canada;Brazil;Australia;Russian Federation;Netherlands;Germany;United Kingdom;Korea, Republic of
38EUCTR2020-002858-24-NL
(EUCTR)
08/09/2021A study assessing the efficacy and safety of an investigational drug called IPN60130 for the treatment of fibrodysplasia ossificans progressiva in participants aged 5 years of age and olderA Phase 2, two-part, placebo-controlled, parallel-group, double-blind study to assess the efficacy and safety of 2 dosage regimens of oral IPN60130 for the treatment of fibrodysplasia ossificans progressiva in male and female participants 5 years of age and older fibrodysplasia ossificans progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: IPN60130 10 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Product Name: IPN60130 50 mg capsules
INN or Proposed INN: Not yet available
Other descriptive name: (R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4- yl)pyridine-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate
Clementia Pharmaceuticals Inc, an Ipsen CompanyNULLNAFemale: yes
Male: yes
90Phase 2United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of
39EUCTR2021-002244-70-FR
(EUCTR)
09/11/2021A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged =14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit from Palovarotene Therapy. - PIVOINE Fibrodysplasia Ossificans Progressiva (FOP)
MedDRA version: 20.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
Product Code: IPN60120
INN or Proposed INN: Palovarotene
Other descriptive name: PALOVAROTENE
Ipsen Pharma SASNULLNAFemale: yes
Male: yes
87Phase 3United States;France;Canada;Argentina;Spain;Brazil;Australia;United Kingdom;Italy;Sweden
40EUCTR2016-002526-36-FR
(EUCTR)
04/07/2016Study of an Investigational Drug, Palovarotene, in the prevention of Preosseous Flare-ups in Subjects with Fibrodysplasia Ossificans Progressiva (FOP)A Phase 2, Open-Label, Efficacy and Safety Study of an RAR?-Specific Agonist (Palovarotene) to Prevent Heterotopic Ossification in Subjects with Fibrodysplasia Ossificans Progressiva (FOP) Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and abnormal heterotopic ossification (HO) in muscles, tendons, and ligaments. Lesions begin in early childhood and lead to progressive ankyloses of major joints with resultant loss of movement. Prognosis is poor and median life expectancy is 40 years.
MedDRA version: 19.0;Level: PT;Classification code 10068715;Term: Fibrodysplasia ossificans progressiva;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Palovarotene
Product Code: Palovarotene
INN or Proposed INN: Palovarotene
Other descriptive name: Palovarotene
Clementia Pharmaceuticals Inc.NULLNot Recruiting Female: yes
Male: yes
17 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance