240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
Showing 1 to 10 of 143 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) | July 2023 | 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 | NCT04272736 (ClinicalTrials.gov) | December 1, 2022 | 29/8/2018 | PKU Low Calorie Drink Study | Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute ... | Phenylketonurias;Hyperphenylalaninaemia | Dietary Supplement: Low calorie protein substitute | Nutricia UK Ltd | NULL | Not yet recruiting | 3 Years | N/A | All | 30 | N/A | NULL |
| 3 | NCT05579548 (ClinicalTrials.gov) | October 4, 2022 | 11/10/2022 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® ( ... | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® ( ... | Phenylketonuria, Maternal | Drug: Pegvaliase | BioMarin Pharmaceutical | Syneos Health | Recruiting | N/A | N/A | Female | 50 | United States | |
| 4 | 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] 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 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 | 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 United States;Portugal;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denma ... | ||
| 5 | NCT05270837 (ClinicalTrials.gov) | June 17, 2022 | 1/2/2022 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketon ... | A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegva ... | Phenylketonuria (PKU) | Drug: Pegvaliase;Other: Diet Only | BioMarin Pharmaceutical | NULL | Recruiting | 12 Years | 17 Years | All | 54 | Phase 3 | United States;Germany |
| 6 | NCT05222178 (ClinicalTrials.gov) | June 3, 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 | Recruiting | 18 Years | 55 Years | All | 9 | Phase 1 | United States |
| 7 | NCT05356377 (ClinicalTrials.gov) | May 25, 2022 | 27/4/2022 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With ... | A Preliminary Study of the Neurological and Neuropsychological Effects of Palynziq-Related Changes in Phenylalanine in Individuals With Phenylketonuria (PKU) A Preliminary Study of the Neurological and Neuropsychological Effects of Palynziq-Related Changes i ... | Phenylketonurias | Drug: Pegvaliase-Pqpz | University of Missouri-Columbia | BioMarin Pharmaceutical | Enrolling by invitation | 18 Years | 55 Years | All | 13 | United States | |
| 8 | NCT04898829 (ClinicalTrials.gov) | May 18, 2022 | 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 |
| 9 | 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] 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 | Portugal;United States;France;Mexico;Canada;Spain;Brazil;Australia;Denmark;Netherlands;Germany;Italy Portugal;United States;France;Mexico;Canada;Spain;Brazil;Australia;Denmark;Netherlands;Germany;Italy ... | ||
| 10 | 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] 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 | Portugal;United States;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denmark;Georgia;Germany;Netherlands Portugal;United States;Spain;Turkey;United Kingdom;Italy;France;Mexico;Canada;Brazil;Australia;Denma ... |