Phenylketonuria Clinical Trials

124 clinical trials for Phenylketonuria across international registries, including 24 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function — RECRUITING · PHASE1
• A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria — RECRUITING · PHASE1
• Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU — RECRUITING
• A Study of Sepiapterin in Participants With Phenylketonuria (PKU) — RECRUITING · PHASE3
• A Study of MZE782 in Adults With PKU — RECRUITING · PHASE2
• Prevalence and Determinants of Obesity in PKU Patients — RECRUITING
• Eating Disorders in Patients With Phenylketonuria — RECRUITING
• A Study of JNT-517 in Participants With Phenylketonuria (PKU) — RECRUITING · PHASE3
• GenSci144 Tablets Phase I Clinical Trial — RECRUITING · PHASE1
• A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU) — RECRUITING · PHASE1
• Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience. — RECRUITING
• Immune Modulation During Palynziq® Treatment in Adults (IMPALA) — RECRUITING · PHASE4
• Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria — RECRUITING · NA
• Multiomics Approach in Adult Patients With Phenylketonuria — RECRUITING
• A Long-Term Study of JNT-517 in Participants With Phenylketonuria — RECRUITING · PHASE3
• Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises — RECRUITING · NA
• A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) — RECRUITING
• A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) — RECRUITING
• GMP Powdered Substitutes in PKU and TYR — RECRUITING · NA
• A PHASE 3, DOUBLE-BLIND, RANDOMIZED, TWO-PERIOD, MULTICENTER, PLACEBO CONTROLLED, EFFICACY AND SAFETY STUDY OF JNT-517 FOR THE TREATMENT OF PARTICIPANTS WITH PHENYLKETONURIA — RECRUITING · PHASE3
• Brain Aging in Phenylketonuria — RECRUITING
• 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 — RECRUITING
• A PHASE 3, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED WITHDRAWAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SYNB1934 IN PATIENTS WITH PKU (SYNPHENY-3) — RECRUITING · PHASE3
• Long-Term Follow Up Study of Subjects Previously Administered HMI 102 — RECRUITING
• A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects with Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 — COMPLETED · PHASE3
• A clinical study, carried out in different centers and using a placebo, to assess the effect of a medicinal product, containing sapropterin as the active substance, on mental abilities in young adults suffering from phenylketonuria, a genetic disease in which an enzyme defect leads to accumulation of a waste product (phenylketone) which can be detected in urine. — TERMINATED · PHASE2
• A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria — OTHER · PHASE2
• Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria — ACTIVE_NOT_RECRUITING · PHASE3
• First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria — COMPLETED · PHASE1
• PKU Microtablets Case Studies — NOT_YET_RECRUITING · NA
• A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria — ACTIVE_NOT_RECRUITING · PHASE3
• A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin (EPIPHENY) — TERMINATED · PHASE3
• Evaluation of AG-181 in Subjects 18 to 69 Years of Age With Phenylketonuria — TERMINATED · PHASE1
• A phase 1 first-in-human study of a phenylalanine-binding polymer (phelimin) in healthy volunteers and adult, classical PKU patients — NOT_YET_RECRUITING · PHASE1
• 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 — TERMINATED · PHASE3
• A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria — COMPLETED · PHASE2
• Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq — ACTIVE_NOT_RECRUITING · PHASE4
• A Phase 3, Double-Blind, Randomized, Two-Period, Multicenter, Placebo-Controlled, Efficacy and Safety Study of JNT-517 for the Treatment of Participants with Phenylketonuria — TERMINATED · PHASE3
• Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria — COMPLETED · PHASE1
• The Psychosocial Functioning of Adults With Phenylketonuria. — NOT_YET_RECRUITING
• To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria — NOT_YET_RECRUITING · PHASE1
• Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria — COMPLETED · NA
• A Phase 3, Randomized, Crossover, Open-Label, Active-Controlled Study of Sepiapterin versus Sapropterin in Participants With Phenylketonuria ≥2 years of Age — OTHER · PHASE3
• Evaluation of BH4 Responsiveness in Our PKU Patients — ACTIVE_NOT_RECRUITING · PHASE1
• PKU EASY Microtabs Plus - Acceptability and Tolerance — COMPLETED · NA
• A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria — ACTIVE_NOT_RECRUITING · PHASE1
• Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria — ACTIVE_NOT_RECRUITING · PHASE4
• Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study — COMPLETED · PHASE2
• AAV Gene Therapy Study for Subjects with PKU — ACTIVE_NOT_RECRUITING · PHASE1
• A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria — WITHDRAWN · PHASE1

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