Phenylketonurias
Information
- Disease name
- Phenylketonurias
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04898829 | Active, not recruiting | N/A | Evaluation of PKU Explore France | May 18, 2022 | May 31, 2025 |
| NCT05174559 | Active, not recruiting | N/A | Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU) | September 1, 2023 | July 31, 2024 |
| NCT06332105 | Active, not recruiting | N/A | Evaluation of Low Phenylalanine Formulas | January 30, 2023 | April 2025 |
| NCT05971563 | Active, not recruiting | N/A | Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers | October 18, 2022 | August 31, 2023 |
| NCT02445521 | Completed | Testing of Four Home Phenylalanine Monitoring Prototype Devices | May 2015 | May 2015 | |
| NCT03167697 | Completed | N/A | Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia | May 1, 2017 | December 17, 2018 |
| NCT03419819 | Completed | N/A | Evaluation of PKU Sphere | May 1, 2018 | May 21, 2019 |
| NCT03771391 | Completed | N/A | A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU | December 6, 2018 | July 31, 2023 |
| NCT03777826 | Completed | N/A | Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU) | June 26, 2019 | May 27, 2021 |
| NCT03792451 | Completed | Nutrition Status of Adults Treated With Pegvaliase | January 17, 2019 | December 31, 2020 | |
| NCT03820804 | Completed | Nutritional Status in Phenylketonuria | January 7, 2019 | September 10, 2019 | |
| NCT03856203 | Completed | Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase | March 8, 2019 | October 20, 2023 | |
| NCT04076176 | Completed | N/A | The Effects of CGMP in Children and Adults With PKU | April 26, 2019 | January 31, 2023 |
| NCT00225615 | Completed | Phase 3 | A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels | November 2005 | June 2006 |
| NCT05096988 | Completed | N/A | Evaluation of PKU Sphere Liquid | November 23, 2021 | February 28, 2023 |
| NCT00272792 | Completed | Phase 3 | Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet | February 2006 | November 2006 |
| NCT01209819 | Completed | Bone Mineral Density in Adults With Hyperphenylalaninemia | September 2010 | October 2012 | |
| NCT04368624 | Completed | PKU Skin Stripping | October 12, 2015 | April 2021 | |
| NCT04375592 | Completed | Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketonuria | February 1, 2021 | May 10, 2021 | |
| NCT00104260 | Completed | Phase 2 | Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria | December 2004 | November 2005 |
| NCT04452513 | Completed | A Prospective Clinical Study of Phenylketonuria (PKU) | October 11, 2019 | June 2, 2022 | |
| NCT04679467 | Completed | N/A | Evaluation of PKU Sphere in Italy | January 15, 2020 | July 31, 2022 |
| NCT05249218 | Completed | N/A | Impact of Fruit and Vegetables in PKU | July 20, 2019 | June 30, 2021 |
| NCT04943393 | Completed | Remote Neurocognitive and Psychological Assessment in PKU | November 24, 2020 | October 2, 2021 | |
| NCT04248062 | Completed | Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism | August 1, 2019 | October 16, 2020 | |
| NCT00104247 | Completed | Phase 3 | Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels | March 2005 | February 2006 |
| NCT05497050 | Completed | N/A | The Effect of Nursing Empowerment Program | November 24, 2021 | June 22, 2023 |
| NCT04309331 | Completed | N/A | Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute | July 1, 2018 | September 19, 2018 |
| NCT04318509 | Completed | N/A | Market Research - Acceptability Study for a New PKU Protein Substitute | February 4, 2019 | May 19, 2019 |
| NCT03655223 | Enrolling by invitation | Early Check: Expanded Screening in Newborns | October 15, 2018 | December 31, 2025 | |
| NCT05356377 | Enrolling by invitation | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | May 25, 2022 | April 30, 2024 | |
| NCT06332807 | Not yet recruiting | Phase 1/Phase 2 | AAV Gene Therapy Clinical Study in Adult Classic PKU | November 22, 2024 | December 30, 2030 |
| NCT04433728 | Not yet recruiting | Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002. | September 2022 | September 2024 | |
| NCT04272736 | Not yet recruiting | N/A | PKU Low Calorie Drink Study | December 1, 2023 | December 31, 2023 |
| NCT06337864 | Not yet recruiting | N/A | Evaluating the Efficacy and Safety of Large Neutral Amino Acids in the Treatment of Classical Phenylketonuria | May 2024 | December 2027 |
| NCT04969809 | Not yet recruiting | N/A | Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients | September 1, 2021 | September 1, 2026 |
| NCT05827536 | Recruiting | N/A | Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU. | October 30, 2023 | July 31, 2024 |
| NCT04404530 | Recruiting | Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) | October 8, 2019 | November 2027 | |
| NCT05051657 | Recruiting | N/A | Evaluation of the Express Plus Range | June 14, 2021 | April 30, 2025 |
| NCT05128149 | Recruiting | Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference? | November 15, 2021 | December 31, 2024 | |
| NCT05487378 | Recruiting | N/A | Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute. | June 30, 2023 | June 2024 |
| NCT05998109 | Recruiting | PheCheck Feasibility Study | September 5, 2023 | March 15, 2024 | |
| NCT06061614 | Recruiting | Early Phase 1 | Safety and Efficacy Study of NGGT002 in PKU Adult Subjects | March 30, 2023 | December 30, 2028 |
| NCT04768348 | Terminated | Natural History Clinical Study in Adult PKU | April 20, 2021 | August 1, 2023 | |
| NCT04110496 | Terminated | Phase 1 | Safety and Tolerability of RTX-134 in Adults With Phenylketonuria | January 29, 2020 | December 1, 2022 |
| NCT04210206 | Terminated | N/A | Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria | December 1, 2019 | August 31, 2023 |
| NCT03952156 | Terminated | Phase 1/Phase 2 | Gene Therapy Clinical Study in Adult PKU | June 10, 2019 | August 1, 2023 |
| NCT05222178 | Terminated | Phase 1 | Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency | June 3, 2022 | September 14, 2023 |
| NCT05229549 | Terminated | N/A | Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria | May 6, 2022 | February 7, 2023 |
| NCT04251494 | Unknown status | A Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD). | February 23, 2019 | April 22, 2021 | |
| NCT04302194 | Unknown status | Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development. | May 1, 2020 | October 2021 | |
| NCT04256655 | Withdrawn | Phase 1 | Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients | December 1, 2020 | December 30, 2021 |
| NCT03333720 | Withdrawn | N/A | Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria | July 15, 2019 | December 31, 2019 |
- MeSH unique ID (MeSH (Medical Subject Headings))
- D010661