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
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05027802 (ClinicalTrials.gov) | March 15, 2022 | 25/8/2021 | A 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: Palovarotene | Ipsen | NULL | Not yet recruiting | 14 Years | N/A | All | 87 | Phase 3 | NULL |
| 2 | EUCTR2020-002858-24-SE (EUCTR) | 24/01/2022 | 27/11/2020 | A 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 older | 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 - 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 Company | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 2 | United States;Spain;Russian Federation;Colombia;Italy;United Kingdom;France;Mexico;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;China;Korea, Republic of;Sweden | ||
| 3 | NCT05090891 (ClinicalTrials.gov) | December 20, 2021 | 8/10/2021 | To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva | A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva | Fibrodysplasia Ossificans Progressiva (FOP) | Drug: INCB000928;Drug: placebo | Incyte Corporation | NULL | Not yet recruiting | 12 Years | 99 Years | All | 60 | Phase 2 | NULL |
| 4 | NCT05039515 (ClinicalTrials.gov) | December 1, 2021 | 2/9/2021 | Study 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 Progressiva | Drug: IPN60130;Drug: Placebo | Clementia Pharmaceuticals Inc. | Ipsen | Recruiting | 5 Years | N/A | All | 90 | Phase 2 | United States;France;Italy;Korea, Republic of;Spain;Sweden;United Kingdom |
| 5 | EUCTR2020-002858-24-ES (EUCTR) | 19/10/2021 | 27/07/2021 | A 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 older | 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 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 Company | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 2 | United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of | ||
| 6 | NCT04577820 (ClinicalTrials.gov) | October 13, 2021 | 30/9/2020 | Study 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 Progressiva | Fibrodyplasia Ossificans Progressiva (FOP);Heterotopic Ossification (HO) | Drug: garetosmab | Regeneron Pharmaceuticals | NULL | Withdrawn | 18 Years | 60 Years | All | 0 | Phase 3 | NULL |
| 7 | EUCTR2020-002858-24-IT (EUCTR) | 09/06/2021 | 04/06/2021 | A 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 older | 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 2 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Germany;Netherlands;China;Japan;Sweden;Korea, Republic of | ||
| 8 | NCT04818398 (ClinicalTrials.gov) | April 1, 2021 | 24/3/2021 | Study of Single-Ascending Doses of DS-6016a in Healthy Japanese Subjects | Single-Ascending Dose Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of DS-6016a After Subcutaneous Injection in Healthy Japanese Subjects | Fibrodysplasia Ossificans Progressiva | Drug: DS-6016a;Drug: Placebo | Daiichi Sankyo Co., Ltd. | NULL | Active, not recruiting | 20 Years | 45 Years | Male | 48 | Phase 1 | Japan |
| 9 | NCT04307953 (ClinicalTrials.gov) | August 5, 2020 | 11/3/2020 | Saracatinib Trial TO Prevent FOP | Saracatinib Trial TO Prevent FOP | Fibrodysplasia Ossificans Progressiva | Drug: AZD0530 Difumarate;Drug: Matching placebo | VU University Medical Center | Royal National Orthopaedic Hospital NHS Trust;Klinikum Garmisch-Patenkirchen;University of Oxford;Brigham and Women's Hospital;AstraZeneca;Innovative Medicines Initiative | Recruiting | 18 Years | 65 Years | All | 20 | Phase 2 | Germany;Netherlands;United Kingdom |
| 10 | EUCTR2019-003324-20-NL (EUCTR) | 08/01/2020 | 16/10/2019 | A clinical trial to judge the safety, tolerability and effects on abnormal bone formation of reseach medication AZD 0530 (sarcatinib) in patients with FOP | Saracatinib 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 Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Netherlands | ||
| 11 | EUCTR2016-005035-33-ES (EUCTR) | 17/01/2019 | 22/01/2019 | A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;France;Mexico;Canada;Poland;Spain;Netherlands;Colombia;United Kingdom;Italy | ||
| 12 | NCT04829773 (ClinicalTrials.gov) | January 3, 2019 | 1/4/2021 | 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 Subjects | An 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 Subjects | Fibrodysplasia Ossificans Progressiva | Drug: Palovarotene;Drug: midazolam | Clementia Pharmaceuticals Inc. | NULL | Completed | 18 Years | 55 Years | All | 48 | Phase 1 | United States |
| 13 | EUCTR2017-002541-29-NL (EUCTR) | 16/07/2018 | 06/02/2018 | 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 Inc | NULL | NA | Female: yes Male: yes | 80 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden | ||
| 14 | EUCTR2017-002541-29-IT (EUCTR) | 15/03/2018 | 26/01/2021 | 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] | 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 107 | Phase 3 | United 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 | ||
| 15 | EUCTR2016-005035-33-NL (EUCTR) | 05/03/2018 | 04/07/2017 | A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | France;United States;Argentina;Spain;Brazil;Netherlands;United Kingdom;Italy | ||
| 16 | NCT03188666 (ClinicalTrials.gov) | February 26, 2018 | 13/6/2017 | A Study to Examine the Safety, Tolerability and Effects on Abnormal Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva | A Randomized, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effects on Heterotopic Bone Formation of REGN2477 in Patients With Fibrodysplasia Ossificans Progressiva | Fibrodysplasia Ossificans Progressiva | Drug: REGN2477;Drug: Matching placebo | Regeneron Pharmaceuticals | NULL | Completed | 18 Years | 60 Years | All | 44 | Phase 2 | United States;Canada;France;Italy;Netherlands;Poland;Spain;United Kingdom;Colombia |
| 17 | EUCTR2016-005035-33-IT (EUCTR) | 15/12/2017 | 08/03/2018 | A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 2 | France;United States;Argentina;Brazil;Netherlands;United Kingdom;Italy | ||
| 18 | EUCTR2017-002541-29-GB (EUCTR) | 14/12/2017 | 11/09/2017 | 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 Inc | NULL | Authorised-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): no | United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;Sweden | ||
| 19 | EUCTR2017-002541-29-SE (EUCTR) | 05/12/2017 | 20/09/2017 | 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 Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 107 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Brazil;Australia;Germany;Netherlands;Japan;Sweden | ||
| 20 | NCT03312634 (ClinicalTrials.gov) | November 28, 2017 | 9/10/2017 | An 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 Progressiva | Drug: Palovarotene | Clementia Pharmaceuticals Inc. | NULL | Active, not recruiting | 4 Years | N/A | All | 110 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;France;Italy;Japan;Spain;Sweden;United Kingdom |
| 21 | EUCTR2017-002541-29-ES (EUCTR) | 14/11/2017 | 28/09/2017 | 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 Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 80 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden | ||
| 22 | EUCTR2016-005035-33-GB (EUCTR) | 15/08/2017 | 22/05/2017 | A study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 40 | Phase 2 | United States;France;Mexico;Canada;Spain;Poland;Netherlands;Colombia;Italy;United Kingdom | ||
| 23 | NCT02979769 (ClinicalTrials.gov) | November 28, 2016 | 23/11/2016 | An Open-Label Extension Study of Palovarotene to Prevent Heterotopic Ossification in People With Fibrodysplasia Ossificans Progressiva (FOP) in France | 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 Progressiva | Drug: Palovarotene | Clementia Pharmaceuticals Inc. | NULL | Active, not recruiting | 6 Years | 65 Years | All | 9 | Phase 2 | France |
| 24 | NCT02521792 (ClinicalTrials.gov) | December 7, 2015 | 6/8/2015 | In-Home Evaluation of Episodic Administration of Palovarotene in Fibrodysplasia Ossificans Progressiva (FOP) Subjects | A Phase 2, In-Home, Safety and Efficacy Evaluation of Episodic Administration of Open-Label Palovarotene in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) | Fibrodysplasia Ossificans Progressiva | Drug: Palovarotene | Clementia Pharmaceuticals Inc. | NULL | Terminated | 6 Years | 65 Years | All | 6 | Phase 2 | United States;France;United Kingdom |
| 25 | EUCTR2014-002496-28-GB (EUCTR) | 13/11/2015 | 04/09/2015 | Extension 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Argentina;Australia;United Kingdom | ||
| 26 | EUCTR2014-001453-17-GB (EUCTR) | 07/07/2015 | 25/02/2015 | The 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. | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2 | United States;Argentina;United Kingdom | ||
| 27 | NCT02279095 (ClinicalTrials.gov) | October 27, 2014 | 26/10/2014 | An 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 Progressiva | Drug: Palovarotene dose level 1;Drug: Palovarotene dose level 2;Drug: Palovarotene dose level 3;Drug: Palovarotene dose level 4 | Clementia Pharmaceuticals Inc. | NULL | Active, not recruiting | 6 Years | 65 Years | All | 54 | Phase 2 | United States;Argentina;Australia;France;United Kingdom |
| 28 | NCT02190747 (ClinicalTrials.gov) | July 2014 | 13/7/2014 | An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects | 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 | Drug: Palovarotene;Drug: Placebo | Clementia Pharmaceuticals Inc. | NULL | Completed | 6 Years | N/A | All | 40 | Phase 2 | United States;France;United Kingdom |
| 29 | JPRN-UMIN000019348 | 2010/07/01 | 14/10/2015 | Approved calcium channel blocker in the treatment of fibrodysplasia ossificans progressiva (FOP) | fibrodysplasia ossificans progressiva | oral administration of perhexiline maleate for one year | Nagoya University Graduate School of Medicine | NULL | Complete: follow-up complete | 15years-old | 65years-old | Male and Female | 5 | Phase 1,2 | Japan | |
| 30 | EUCTR2021-002244-70-SE (EUCTR) | 25/10/2021 | A 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 | NULL | NA | Female: yes Male: yes | 61 | Phase 3 | France;United States;Canada;Argentina;Spain;Brazil;Australia;United Kingdom;Italy;Sweden | |||
| 31 | EUCTR2021-002244-70-FR (EUCTR) | 09/11/2021 | A 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 SAS | NULL | NA | Female: yes Male: yes | 87 | Phase 3 | United States;France;Canada;Argentina;Spain;Brazil;Australia;United Kingdom;Italy;Sweden | |||
| 32 | EUCTR2021-002286-17-DE (EUCTR) | 04/11/2021 | A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva | A 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 Corporation | NULL | NA | Female: yes Male: yes | 60 | Phase 2 | France;United States;Canada;Brazil;Australia;Russian Federation;Netherlands;Germany;United Kingdom;Korea, Republic of | |||
| 33 | EUCTR2021-002286-17-FR (EUCTR) | 22/10/2021 | A Phase 2, Randomized Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva | A 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 Corporation | NULL | NA | Female: yes Male: yes | 60 | Phase 2 | United States;France;Canada;Brazil;Australia;Russian Federation;Netherlands;Germany;United Kingdom;Korea, Republic of | |||
| 34 | EUCTR2017-002541-29-Outside-EU/EEA (EUCTR) | 15/07/2021 | 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 Inc. | NULL | NA | Female: yes Male: yes | Phase 3 | Australia;Brazil;Canada;Japan;United States;Argentina | ||||
| 35 | EUCTR2016-002526-36-FR (EUCTR) | 04/07/2016 | Study 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. | NULL | Not 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): no | France | |||
| 36 | EUCTR2014-002496-28-Outside-EU/EEA (EUCTR) | 15/07/2021 | Extension 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. | NULL | NA | Female: yes Male: yes | Phase 2 | Australia;United States;Argentina | ||||
| 37 | EUCTR2020-002858-24-NL (EUCTR) | 08/09/2021 | A 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 older | 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 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 Company | NULL | NA | Female: yes Male: yes | 90 | Phase 2 | United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of | |||
| 38 | EUCTR2017-002541-29-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 Inc | NULL | NA | Female: yes Male: yes | 80 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden | |||
| 39 | EUCTR2020-002858-24-DE (EUCTR) | 27/11/2020 | A 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 older | 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 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 Company | NULL | NA | Female: yes Male: yes | 90 | Phase 2 | United States;Spain;Russian Federation;Colombia;United Kingdom;Italy;France;Mexico;Canada;Argentina;Brazil;Australia;Netherlands;Germany;China;Japan;Sweden;Korea, Republic of | |||
| 40 | EUCTR2017-002541-29-DE (EUCTR) | 29/09/2017 | 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 - 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 Inc | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | United States;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Brazil;Australia;South Africa;Netherlands;Germany;Japan;Sweden |