276. Achondroplasia Clinical trials / Disease details
Clinical trials : 51 / Drugs : 34 - (DrugBank : 6) / Drug target genes : 4 - Drug target pathways : 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 |