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
| 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 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 | 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 | 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 | 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] | 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 | 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 Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia | 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 | 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 | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | 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 | 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] | Product Name: Infigratinib Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATINIB | 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 | 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 | 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 | A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA | 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 AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | 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 | 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 | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | 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 | ||
| 11 | EUCTR2019-002754-22-PT (EUCTR) | 05/02/2021 | 18/06/2020 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - ACcomplisH | Achondroplasia (ACH) in prepubertal children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;Portugal;Canada;Spain;Ireland;Denmark;Austria;Australia;Germany;United Kingdom;Switzerland;New Zealand | ||
| 12 | EUCTR2020-001189-13-IT (EUCTR) | 05/02/2021 | 15/06/2021 | 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 - - | Achondroplasia MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 MedDRA version: 20.0;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: Recifercept Product Code: [PF-07256472] INN or Proposed INN: Recifercept | PFIZER INC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Spain;Belgium;Denmark;Australia;United Kingdom;Japan;Italy | ||
| 13 | EUCTR2020-001189-13-DK (EUCTR) | 22/12/2020 | 07/10/2020 | 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 | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | Pfizer Inc., 235 East 42nd Street, New York, NY 10017 | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Spain;Belgium;Australia;Denmark;Italy;Japan | ||
| 14 | NCT04638153 (ClinicalTrials.gov) | December 2, 2020 | 15/9/2020 | A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia | A PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA | Achondroplasia | Biological: Recifercept | Pfizer | NULL | Active, not recruiting | 3 Months | 10 Years | All | 61 | Phase 2 | United States;Australia;Belgium;Denmark;Italy;Japan;Portugal;Spain |
| 15 | NCT04554940 (ClinicalTrials.gov) | October 10, 2020 | 14/9/2020 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery | Achondroplasia | Biological: vosoritide | BioMarin Pharmaceutical | NULL | Active, not recruiting | 0 Months | 12 Months | All | 20 | Phase 2 | Australia;United Kingdom |
| 16 | EUCTR2020-001055-40-GB (EUCTR) | 29/07/2020 | 19/05/2020 | Open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia | A randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Australia;United Kingdom | ||
| 17 | EUCTR2019-002754-22-DE (EUCTR) | 28/07/2020 | 01/10/2019 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH | Achondroplasia (ACH) in prepubertal children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand | ||
| 18 | EUCTR2019-002754-22-DK (EUCTR) | 02/07/2020 | 12/05/2020 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - ACcomplisH | 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] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Canada;Spain;Ireland;Austria;Australia;Denmark;Germany;United Kingdom;Switzerland;New Zealand | ||
| 19 | NCT04085523 (ClinicalTrials.gov) | June 24, 2020 | 9/9/2019 | A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia | ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Prepubertal Children With Achondroplasia Followed by an Open-Label Extension Period | Achondroplasia | Drug: TransCon CNP;Drug: Placebo for TransCon CNP;Drug: sWfI Placebo for TransCon CNP | Ascendis Pharma A/S | NULL | Active, not recruiting | 2 Years | 10 Years | All | 57 | Phase 2 | Australia;Austria;Denmark;Germany;Ireland;New Zealand;Portugal;United States |
| 20 | EUCTR2019-002754-22-AT (EUCTR) | 09/06/2020 | 31/10/2019 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - ACcomplisH | 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] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Canada;Spain;Ireland;Denmark;Australia;Austria;Germany;United Kingdom;Switzerland;New Zealand | ||
| 21 | EUCTR2019-002954-21-FR (EUCTR) | 06/05/2020 | 20/01/2020 | A study to evaluate Infigratinib in children with Achondroplasia | Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2 | 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] | Product Name: Infigratinib phosphate Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATINIB | QED Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | United States;France;Canada;Spain;Australia;United Kingdom | ||
| 22 | EUCTR2019-002954-21-GB (EUCTR) | 31/03/2020 | 13/05/2020 | A study to evaluate Infigratinib in children with Achondroplasia | Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2 | 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] | Product Name: Infigratinib phosphate Product Code: BGJ398 (also known as BBP-831) INN or Proposed INN: INFIGRATINIB | QED Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 2 | United States;France;Canada;Spain;Australia;United Kingdom | ||
| 23 | NCT04265651 (ClinicalTrials.gov) | March 10, 2020 | 29/1/2020 | Study of Infigratinib in Children With Achondroplasia | Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2 | Achondroplasia | Drug: Infigratinib 0.016 mg/kg;Drug: Infigratinib 0.032 mg/kg;Drug: Infigratinib 0.064 mg/kg;Drug: Infigratinib 0.128 mg/kg;Drug: Infigratinib 0.25 mg/kg | QED Therapeutics, Inc. | NULL | Recruiting | 3 Years | 11 Years | All | 108 | Phase 2 | United States;Australia;Canada;France;Spain;United Kingdom |
| 24 | EUCTR2019-002754-22-IE (EUCTR) | 01/11/2019 | 03/09/2019 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period. - ACcomplisH | Achondroplasia (ACH) in prepubertal children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Canada;Spain;Ireland;Denmark;Austria;Australia;Germany;United Kingdom;Switzerland;New Zealand | ||
| 25 | EUCTR2018-004364-66-GB (EUCTR) | 22/10/2019 | 11/09/2019 | A Phase 2 open-label long-term study of BMN 111 in children with Achondroplasia | A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safetyand Efficacy of BMN 111 in Children with Achondroplasia | 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] | Product Name: Modified recombinant human c-type Product Code: Natriuretic peptide INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP Product Name: Modified recombinant human c-type Product Code: Natriuretic peptide INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | United States;Australia;Japan;United Kingdom | ||
| 26 | NCT03989947 (ClinicalTrials.gov) | June 12, 2019 | 22/5/2019 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | BioMarin Pharmaceutical | NULL | Active, not recruiting | 15 Months | N/A | All | 73 | Phase 2 | United States;Australia;Japan;United Kingdom |
| 27 | EUCTR2017-002404-28-DE (EUCTR) | 07/03/2019 | 13/12/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 28 | EUCTR2017-002404-28-ES (EUCTR) | 08/11/2018 | 23/11/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 29 | EUCTR2017-002404-28-GB (EUCTR) | 12/10/2018 | 15/06/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Phase 3 | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 30 | EUCTR2016-003826-18-GB (EUCTR) | 12/09/2018 | 30/05/2018 | A Phase 2 Study to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Australia;Japan;United Kingdom | ||
| 31 | JPRN-JapicCTI-184167 | 19/7/2018 | 23/10/2018 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3 Randomized, Double-Blind,Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia | Achondroplasia | Intervention name : modified recombinant human C-type natriuretic peptide INN of the intervention : Vosoritide Dosage And administration of the intervention : Subcutaneous injection of 15 micro g/kg of BMN 111 daily Control intervention name : Placebo INN of the control intervention : - Dosage And administration of the control intervention : Subcutaneous injection of placebo daily | BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.) | NULL | complete | 5 | 18 | BOTH | 6 | Phase 3 | Japan, North America, Europe, Oceania |
| 32 | NCT03583697 (ClinicalTrials.gov) | May 23, 2018 | 14/6/2018 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months | Achondroplasia | Drug: BMN 111;Drug: Placebo | BioMarin Pharmaceutical | NULL | Completed | N/A | 59 Months | All | 75 | Phase 2 | United States;Australia;Japan;United Kingdom |
| 33 | EUCTR2015-003836-11-DE (EUCTR) | 14/02/2018 | 21/08/2017 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Spain;Turkey;Australia;Germany;United Kingdom;Japan | ||
| 34 | NCT03424018 (ClinicalTrials.gov) | December 12, 2017 | 28/12/2017 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | NULL | Active, not recruiting | 6 Years | N/A | All | 119 | Phase 3 | United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom |
| 35 | EUCTR2017-000701-21-DE (EUCTR) | 19/06/2017 | 27/04/2017 | A study looking at growth measurements of children with achondroplasia | A Multicenter, Multinational Clinical Assessment Study for PediatricPatients with Achondroplasia | 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] | INN or Proposed INN: vorsoritide | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 500 | Phase 1 | France;United States;Spain;Turkey;Australia;Germany;United Kingdom;Japan | ||
| 36 | EUCTR2015-003836-11-ES (EUCTR) | 19/04/2017 | 10/03/2017 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. - | achondroplasia MedDRA version: 19.1;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vorsoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | France;United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 37 | EUCTR2015-003836-11-GB (EUCTR) | 20/01/2017 | 20/10/2016 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 38 | NCT03197766 (ClinicalTrials.gov) | December 12, 2016 | 23/5/2017 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111;Drug: Placebo | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 18 Years | All | 121 | Phase 3 | United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom |
| 39 | NCT02724228 (ClinicalTrials.gov) | January 26, 2016 | 12/2/2016 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | NULL | Active, not recruiting | 7 Years | N/A | All | 30 | Phase 2 | United States;Australia;France;United Kingdom |
| 40 | EUCTR2013-004137-32-GB (EUCTR) | 03/04/2014 | 17/02/2014 | A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia | achondroplasia MedDRA version: 19.1;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: Vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 36 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Australia;United Kingdom | ||
| 41 | NCT02055157 (ClinicalTrials.gov) | January 13, 2014 | 18/4/2013 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 14 Years | All | 35 | Phase 2 | United States;Australia;France;United Kingdom |
| 42 | NCT01435629 (ClinicalTrials.gov) | November 22, 2012 | 14/9/2011 | A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin | Open-label, Multicenter, Observational, Non-intervention Study to Retrospectively Evaluate the Efficacy of Norditropin® (Adult Height) in Patients With Achondroplasia/Hypochondroplasia Enrolled in the GH-1941 Study [Follow-up Survey] | Genetic Disorder;Achondroplasia | Drug: somatropin | Novo Nordisk A/S | NULL | Completed | N/A | N/A | All | 81 | N/A | Japan |
| 43 | NCT01590446 (ClinicalTrials.gov) | February 2012 | 14/3/2012 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | A Phase 1, Two-Part, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 111 Administered to Healthy Adult Volunteers | Achondroplasia | Drug: BMN 111;Drug: Normal Saline | BioMarin Pharmaceutical | NULL | Completed | 22 Years | 45 Years | Male | 74 | Phase 1 | United States |
| 44 | NCT01516229 (ClinicalTrials.gov) | May 1, 1997 | 19/1/2012 | Special Survey for Long Term Application | Specific Survey of Norditropin® in Achondroplasia: Survey for Long-term Application | Genetic Disorder;Achondroplasia | Drug: somatropin | Novo Nordisk A/S | NULL | Completed | 1 Year | 15 Years | All | 395 | N/A | Japan |
| 45 | EUCTR2015-004004-30-FR (EUCTR) | 10/06/2016 | A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | achondroplasia MedDRA version: 19.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 46 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Australia;United Kingdom | |||
| 46 | EUCTR2013-004137-32-FR (EUCTR) | 18/06/2015 | A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia | achondroplasia MedDRA version: 18.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: TBD Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 2 | United States;France;Australia;United Kingdom | |||
| 47 | EUCTR2021-003149-39-DK (EUCTR) | 10/03/2022 | 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 | Achondroplasia MedDRA version: 25.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | Pfizer Inc. | NULL | NA | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Belgium;Spain;Australia;Denmark;Italy;Japan | |||
| 48 | EUCTR2020-001189-13-BE (EUCTR) | 10/02/2021 | 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 | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | Pfizer Inc., 235 East 42nd Street, New York, NY 10017 | NULL | NA | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Spain;Belgium;Denmark;Australia;Italy;Japan | |||
| 49 | EUCTR2015-004004-30-GB (EUCTR) | 07/03/2016 | A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | 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] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 INN or Proposed INN: vosoritide Other descriptive name: MODIFIED RHCNP | BioMarin Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 30 | Phase 2 | United States;France;Australia;United Kingdom | |||
| 50 | EUCTR2021-003149-39-BE (EUCTR) | 07/03/2022 | 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 | 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] | Product Name: Recifercept (proposed INN) Product Code: PF-07256472 INN or Proposed INN: Recifercept (proposed INN) Other descriptive name: TA-46 | Pfizer Inc. | NULL | NA | Female: yes Male: yes | 63 | Phase 2 | Portugal;United States;Spain;Belgium;Denmark;Australia;Italy;Japan | |||
| 51 | EUCTR2019-002754-22-GB (EUCTR) | 09/09/2019 | A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia | ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH | Achondroplasia (ACH) in prepubertal children MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | 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 MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER | Ascendis Pharma Growth Disorders A/S | NULL | NA | Female: yes Male: yes | 60 | Phase 2 | Portugal;United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Australia;Denmark;Germany;New Zealand |