240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 138 / 薬物数 : 106 - (DrugBank : 11) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
Showing 1 to 10 of 138 diseases
| 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 | NCT01465100 (ClinicalTrials.gov) | December 15, 2022 | 20/10/2011 | Liver Cell Transplant for Phenylketonuria | Hepatocyte Transplantation for Phenylketonuria | Phenylketonuria | Radiation: Preparative Radiation Therapy;Procedure: Hepatocyte Transplant;Drug: Immunosuppression;Other: Liver Evaluation;Behavioral: Neuro-psychological Assessment;Diagnostic Test: Whole body Phe oxidation testing;Procedure: Liver Biopsy Radiation: Preparative Radiation Therapy;Procedure: Hepatocyte Transplant;Drug: Immunosuppression;Ot ... | Ira Fox | NULL | Recruiting | 14 Years | 55 Years | All | 10 | Phase 1/Phase 2 | United States |
| 2 | NCT05222178 (ClinicalTrials.gov) | March 2022 | 21/12/2021 | Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKUDue ... | A Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH Deficiency A Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administ ... | Phenylketonurias;PAH Deficiency;Phenylketonuria | Drug: HMI-103 | Homology Medicines, Inc | NULL | Not yet recruiting | 18 Years | 55 Years | All | 9 | Phase 1 | United States |
| 3 | 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 United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portug ... |
| 4 | 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] Metabolic Disorders - Phenylketonuria MedDRA version: 20.0;Level: LLT;Classification code 10034873;T ... | 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 Product Code: PTC923 INN or Proposed INN: not available Other descriptive name: (S)-2-amino-6-(2-hydro ... | 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 United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denma ... | ||
| 5 | NCT05096988 (ClinicalTrials.gov) | November 23, 2021 | 14/10/2021 | A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU) A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKUS ... | A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU) A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKUS ... | Phenylketonurias | Dietary Supplement: PKU sphere liquid | Vitaflo International, Ltd | NULL | Recruiting | 3 Years | N/A | All | 15 | N/A | United Kingdom |
| 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 United States;Australia;Brazil;Canada;Denmark;France;Georgia;Germany;Italy;Mexico;Netherlands;Portug ... |
| 7 | NCT04898829 (ClinicalTrials.gov) | September 3, 2021 | 11/5/2021 | Evaluation of PKU Explore France | An Exploratory Study to Evaluate the Acceptability of PKU Explore, a Food for Special Medical Purposes, for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 3 Years of Age With Regard to Product-acceptability, Tolerance, Phe Levels and Growth An Exploratory Study to Evaluate the Acceptability of PKUExplore, a Food for Special Medical Purpose ... | Phenylketonurias | Dietary Supplement: PKU explore | Vitaflo International, Ltd | NULL | Recruiting | 6 Months | 3 Years | All | 10 | N/A | France |
| 8 | NCT05051657 (ClinicalTrials.gov) | June 14, 2021 | 10/9/2021 | Evaluation of PKU Express Plus | A Study To Evaluate The Acceptability Of PKU Express Plus, A Food For Special Medical Purposes (FSMP), In Children And Adults With Phenylketonuria (PKU) A Study To Evaluate The Acceptability Of PKUExpress Plus, A Food For Special Medical Purposes (FSMP) ... | Phenylketonurias;PKU | Dietary Supplement: PKU express plus | Vitaflo International, Ltd | NULL | Recruiting | 3 Years | N/A | All | 15 | N/A | United Kingdom |
| 9 | EUCTR2021-001174-29-NL (EUCTR) | 31/05/2021 | 31/03/2021 | Effectiveness of methylphenidate for treating ADHD in adultswith PKU | Effectiveness of methylphenidate in adults with phenylketonuria and attention-deficit/hyperactivity disorder: An N-of-1 series - MPH4PKU Effectiveness of methylphenidate in adults with phenylketonuriaand attention-deficit/hyperactivity d ... | Phenylketonuria, ADHD;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Phenylketonuria, ADHD;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases ... | Trade Name: Medikinet | Amsterdam UMC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 4 | Netherlands | ||
| 10 | NCT04879277 (ClinicalTrials.gov) | May 26, 2021 | 4/5/2021 | Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria | Etude de l'INflammation systémique de Bas GRade Chez Les Patients Adultes Atteints de PHénylcétonurie Etude de l'INflammation systémique de Bas GRade Chez Les Patients Adultes Atteints de PHénylcétonuri ... | Phenylketonuria | Biological: Blood samples | University Hospital, Tours | NULL | Completed | 18 Years | N/A | All | 40 | N/A | France |