276. 軟骨無形成症 Achondroplasia Clinical trials / Disease details
臨床試験数 : 51 / 薬物数 : 34 - (DrugBank : 6) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 26
Showing 1 to 10 of 51 diseases
| 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 | NCT05598320 (ClinicalTrials.gov) | March 3, 2023 | 25/10/2022 | A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children W ... | A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children With Achondroplasia Followed by an Open Label Extension Period A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and ... | Achondroplasia | Drug: TransCon CNP;Drug: Placebo for TransCon CNP | Ascendis Pharma Growth Disorders A/S | NULL | Recruiting | 2 Years | 11 Years | All | 80 | Phase 2/Phase 3 | Canada;Denmark |
| 2 | EUCTR2022-002954-25-DK (EUCTR) | 24/01/2023 | 02/12/2022 | A phase 2b clinical trial to evaluate efficacy and safety of weekly doses of TransCon CNP compared with placebo in participants with achondroplasia aged 2 to 11 years of age A phase 2b clinical trial to evaluate efficacy and safety of weekly doses of TransCon CNP compared w ... | ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children with Achondroplasia followed by an Open Label Extension period - ApproaCH ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Eff ... | Achondroplasia (ACH) in prepubertal children MedDRA version: 25.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Achondroplasia(ACH) in prepubertal children MedDRA version: 25.0;Level: LLT;Classification code 1000 ... | Product Name: TransCon CNP 3.9 mg CNP-38/vial Product Code: TransCon CNP INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER Other descriptive name: C-type natriuretic peptide conjugated to a multi-arm polyethylene glycol carrier molecule through a cleavable linker Product Name: TransCon CNP3.9 mg CNP-38/vial Product Code: TransCon CNP INN or Proposed INN: C-TYPE NA ... | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 80 | Phase 2 | United States;Canada;Spain;Ireland;Australia;Denmark;United Kingdom;New Zealand | ||
| 3 | NCT05353192 (ClinicalTrials.gov) | September 23, 2022 | 25/4/2022 | A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Ach ... | A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of ... | Achondroplasia | Drug: Recombinant human growth hormone | GeneScience Pharmaceuticals Co., Ltd. | Children's Hospital of Fudan University;Tongji Hospital;Children's Hospital of Nanjing Medical University;Jiangxi Province Children's Hospital;Chengdu Women's and Children's Central Hospital;Shandong Provincial Hospital;Shengjing Hospital;Shanghai Children's Hospital;Children's Hospital of The Capital Institute of Pediatrics;West China Second University Hospital, Sichuan University Children's Hospital of Fudan University;Tongji Hospital;Children's Hospital of Nanjing Medical Unive ... | Recruiting | 2 Years | 10 Years | All | 38 | Phase 4 | China |
| 4 | EUCTR2021-003149-39-PT (EUCTR) | 25/05/2022 | 07/02/2022 | Phase 2 study of long-term safety, tolerability, PK and efficacy of recifercept in achondroplasia | A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS ... | Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;Sys ... | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (p ... | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | United States;Portugal;Spain;Belgium;Denmark;Australia;Japan;Italy | ||
| 5 | EUCTR2021-001855-15-ES (EUCTR) | 02/02/2022 | 15/09/2021 | A study to evaluate long-term Infigratinib in children with Achondroplasia | Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL OLE - PROPEL OLE Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosin ... | Achondroplasia in Children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Achondroplasiain Children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondr ... | Product Name: Infigratinib Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATINIB Product Name: Infigratinib Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATI ... | QED Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 280 | Phase 2 | France;United States;Canada;Spain;Australia;United Kingdom | ||
| 6 | NCT05246033 (ClinicalTrials.gov) | January 5, 2022 | 9/2/2022 | A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous D ... | ACcomplisH China: A Phase 2, Multicenter, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia ACcomplisH China: A Phase 2, Multicenter, Randomized, Placebo-controlled, Dose Escalation Trial Eval ... | Achondroplasia | Drug: TransCon CNP;Drug: Placebo for TransCon CNP | Ascendis Pharma A/S | NULL | Recruiting | 2 Years | 10 Years | All | 64 | Phase 2 | China |
| 7 | NCT05116046 (ClinicalTrials.gov) | December 24, 2021 | 18/10/2021 | Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept i ... | A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS ... | Achondroplasia | Biological: Recifercept | Pfizer | NULL | Active, not recruiting | 15 Months | 12 Years | All | 30 | Phase 2 | United States;Australia;Belgium;Denmark;Italy;Portugal;Spain |
| 8 | EUCTR2021-003149-39-ES (EUCTR) | 14/12/2021 | 17/09/2021 | A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS ... | A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS ... | Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;Sys ... | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (p ... | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Belgium;Spain;Denmark;Australia;Italy;Japan | ||
| 9 | NCT05145010 (ClinicalTrials.gov) | December 6, 2021 | 22/11/2021 | Extension Study of Infigratinib in Children With Achondroplasia (ACH) | Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosin ... | Achondroplasia | Drug: Infigratinib | QED Therapeutics, Inc. | NULL | Recruiting | 3 Years | 18 Years | All | 280 | Phase 2 | United States;Australia;France;Spain;United Kingdom |
| 10 | EUCTR2020-001189-13-PT (EUCTR) | 26/02/2021 | 29/09/2020 | Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasia | A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND E ... | Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;Sys ... | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (p ... | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | United States;Portugal;Spain;Belgium;Denmark;Australia;United Kingdom;Italy;Japan |