276. Achondroplasia
37 clinical trials, 29 drugs (DrugBank: 6 drugs), 4 drug target genes, 25 drug target pathways
Searched query = "Achondroplasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | 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;Denmark;Australia;Germany;New Zealand | ||
| 4 | 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 | 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 | ||
| 5 | 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 | 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 | ||
| 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 |
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 |
| 10 | 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 | ||
| 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 |
| 11 | 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 | ||
| 12 | 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 | |||
| 13 | EUCTR2019-002754-22-PT (EUCTR) | 18/06/2020 | 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;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand | |||
| 14 | 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 | |||
| 15 | 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 |