10 completed, 8 recruiting, 3 active not recruiting, 3 not yet recruiting. 5 Not applicable, 4 Phase 2, 4 Phase 3, 3 Phase 1, 1 Early Phase 1.
Follow Phenylketonuria (PKU): new trials by email Literature Review of Phenylketonuria (PKU) trials Research Report on Phenylketonuria (PKU)
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria GS1168 injection - Phenylketonuria (PKU) | Early Phase 1 | Not yet recruiting | Gritgen Therapeutics Co., Ltd. | 9 | 2027-12-31 | 2026-09-10 |
| PKU Microtablets Case Studies PKU Microtablet protein substitute - Phenylketonuria (PKU) | Not applicable | Not yet recruiting | Nutricia UK Ltd | 20 | 2026-10-01 | 2026-09-23 |
| The Impact of Frequency of Home Phenylalanine Measurements on Metabolic Control in a Population of Patients With Classic Phenylketonuria DBS Phe monitoring once weekly, DBS Phe monitoring once monthly - Phenylketonuria (PKU) | Not applicable | Recruiting | Michał Kania | 36 | 2026-09-11 | 2026-09-17 |
| A Study of MZE782 in Adults With PKU MZE782, Placebo - Phenylketonuria (PKU) | Phase 2 | Recruiting | Maze Therapeutics | 38 | 2026-06-23 | 2026-10-23 |
| Multiomics Approach in Adult Patients With Phenylketonuria Phenylketonuria (PKU) | Recruiting | University Hospital, Tours | 149 | 2026-03-23 | 2026-05-06 | |
| Prevalence and Determinants of Obesity in PKU Patients Phenylketonuria (PKU) | Enrolling by invitation | Christel Tran | 100 | 2026-03-01 | 2026-07-14 | |
| The Psychosocial Functioning of Adults With Phenylketonuria. Phenylketonuria (PKU) | Not yet recruiting | Central Hospital, Nancy, France | 30 | 2026-01-15 | 2026-02-12 | |
| Evaluation of BH4 Responsiveness in Our PKU Patients Sapropterin Dihydrochloride (BH4, tetrahydrobiopterin) - Phenylketonuria (PKU) | Phase 1 | Active not recruiting | Sohag University | 20 | 2025-09-01 | 2025-12-01 |
| A Long-Term Study of JNT-517 in Participants With Phenylketonuria JNT-517 - Phenylketonuria (PKU) | Phase 3 | Recruiting | Otsuka Pharmaceutical Development & Commercialization, Inc. | 240 | 2025-08-11 | 2026-05-01 |
| A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU) PL54 - Phenylketonuria (PKU) | Phase 1 | Recruiting | Chongqing Peg-Bio Biopharm Co., Ltd. | 48 | 2025-07-20 | 2026-07-01 |
| A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria JNT-517 Tablet, Placebo - Phenylketonuria (PKU) | Phase 2 | Completed | Otsuka Pharmaceutical Development & Commercialization, Inc. | 14 | 2025-07-16 | 2026-05-13 |
| A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability Phenylketonuria (PKU) | Enrolling by invitation | Egoo Health Aps | 20 | 2025-04-15 | 2025-04-23 | |
| PKU EASY Microtabs Plus - Acceptability and Tolerance PKU EASY Microtabs Plus - Phenylketonuria (PKU) | Not applicable | Completed | Galen Limited | 10 | 2025-03-15 | 2025-11-26 |
| Brain Aging in Phenylketonuria Phenylketonuria (PKU) | Recruiting | Insel Gruppe AG, University Hospital Bern | 90 | 2025-01-13 | 2025-05-13 | |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) Pegvaliase - Phenylketonuria (PKU) | Recruiting | BioMarin Pharmaceutical | 200 | 2024-01-20 | 2026-03-24 | |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Pegvaliase, Diet Only - Phenylketonuria (PKU) | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 55 | 2022-06-17 | 2026-07-21 |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) Pegvaliase - Phenylketonuria (PKU) | Recruiting | BioMarin Pharmaceutical | 450 | 2022-06-06 | 2026-03-25 | |
| AAV Gene Therapy Study for Subjects with PKU BMN 307 - Phenylketonuria (PKU) | Phase 1 / Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 100 | 2020-09-24 | 2024-12-12 |
| Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study iron supplementation, control group - Phenylketonuria (PKU) | Phase 2 | Completed | ASST Santi Paolo e Carlo | 84 | 2020-01-07 | 2025-01-24 |
| PANDA: PKU Amino Acid Evaluation Phenylketonuria (PKU) | Completed | Nutricia Research | 37 | 2019-09-10 | 2023-06-02 |
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Phenylketonurias (28) · Body Weight (1) · Obesity (1) · Rare Diseases (1)
Amino Acid Metabolism, Inborn Errors (28) · Brain Diseases (28) · Brain Diseases, Metabolic (28) · Brain Diseases, Metabolic, Inborn (28) · Central Nervous System Diseases (28) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (28) · Genetic Diseases, Inborn (28) · Metabolic Diseases (28) · Metabolism, Inborn Errors (28) · Nervous System Diseases (28) · Nutritional and Metabolic Diseases (28) · Pathological Conditions, Signs and Symptoms (3) · Signs and Symptoms (2) · Body Weight (1) · Disease Attributes (1)
pegvaliase (5) · sapropterin (3) · Control Groups (1) · Dextrans (1) · Diet (1)
BioMarin Pharmaceutical (7) · Otsuka Pharmaceutical Development & Commercialization, Inc. (2) · University of Glasgow (2) · ASST Santi Paolo e Carlo (1) · Central Hospital, Nancy, France (1) · Chongqing Peg-Bio Biopharm Co., Ltd. (1) · Christel Tran (1) · Dr. Linda Randolph (1) · Egoo Health Aps (1) · Galen Limited (1) · Gritgen Therapeutics Co., Ltd. (1) · Insel Gruppe AG, University Hospital Bern (1) · Maze Therapeutics (1) · Michał Kania (1) · Nutricia Research (1)
United States (12) · United Kingdom (9) · France (3) · Germany (3) · Italy (3) · Australia (2) · Canada (2) · China (2) · Switzerland (2) · Belgium (1) · Egypt (1) · Poland (1) · Turkey (Türkiye) (1)
Anita MacDonald (1) · Anita MacDonald, Ph.D. (1) · Barbara Cochrane, BSc,MSc (1) · Dalia Malkova, BSc,MSc,PhD (1) · Dalia Malkova, PhD (1) · Dr. P. Verloo (1) · Gary Hubbard, Dr (1) · Konstantinos Gerasimidis, PhD,MSc,APHNutr (1) · Linda M. Randolph, MD (1) · Michal Kania (1) · Peter Galloway, Med,MB CHB,DCH,FRCP,FRCPath (1) · Raed Selmi, MD-PhD (1)
PMID 40411344, PMID 12141594, PMID 14640675, PMID 15026283, PMID 15110326, PMID 18566668, PMID 21813309, PMID 26797689, PMID 27513937, PMID 28318688, PMID 30864096, PMID 32461102, PMID 34826353, PMID 39179054, PMID 41989430, PMID 503651, PMID 5447890, PMID 8444222
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