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 | 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 |
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 |
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | 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 |
| 12 | 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 |
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | ||
| 16 | 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 |
| 17 | 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 |
| 18 | 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 |