240. Phenylketonuria Clinical trials / Disease details
Clinical trials : 143 / Drugs : 90 - (DrugBank : 10) / Drug target gene : 1 - Drug target pathways : 5
| 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 | EUCTR2021-000474-29-NL (EUCTR) | 12/08/2022 | 14/04/2022 | A study to see if PTC923 reliefs phenylketonuria symptoms | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria - PTC923-MD-003-PKU | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 178 | Phase 3 | United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Netherlands;Germany | ||
| 2 | EUCTR2021-000474-29-DK (EUCTR) | 13/05/2022 | 10/03/2022 | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 178 | Phase 3 | Portugal;United States;France;Mexico;Canada;Spain;Brazil;Australia;Denmark;Netherlands;Germany;Italy | ||
| 3 | EUCTR2021-000474-29-ES (EUCTR) | 21/04/2022 | 19/10/2021 | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 178 | Phase 3 | Portugal;United States;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands | ||
| 4 | NCT05166161 (ClinicalTrials.gov) | February 14, 2022 | 8/12/2021 | A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria | A Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria | Phenylketonuria | Drug: PTC923 | PTC Therapeutics | NULL | Recruiting | N/A | N/A | All | 200 | Phase 3 | United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom |
| 5 | EUCTR2021-000474-29-PT (EUCTR) | 02/02/2022 | 29/11/2021 | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 178 | Phase 3 | United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands | ||
| 6 | NCT05099640 (ClinicalTrials.gov) | September 30, 2021 | 6/10/2021 | A Study of PTC923 in Participants With Phenylketonuria | A Phase 3 Study of PTC923 in Subjects With Phenylketonuria | Phenylketonuria | Drug: PTC923;Drug: Placebo | PTC Therapeutics | NULL | Recruiting | N/A | N/A | All | 178 | Phase 3 | United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portugal;Spain;Turkey;United Kingdom |
| 7 | EUCTR2021-000474-29-DE (EUCTR) | 13/09/2021 | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | A Phase 3 Study of PTC923 in Subjects with Phenylketonuria | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | NA | Female: yes Male: yes | 178 | Phase 3 | United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands | |||
| 8 | EUCTR2021-000497-28-DE (EUCTR) | 22/12/2021 | A Phase 3 Extension Study of PTC923 in Subjects with Phenylketonuria | Phase 3 Open-Label Extension Study of PTC923 in Phenylketonuria | Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one | PTC Therapeutics, Inc. | NULL | NA | Female: yes Male: yes | 200 | Phase 3 | United States;Portugal;Spain;Turkey;Italy;United Kingdom;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Netherlands;Germany |