Phase 1 Phenylketonuria Clinical Trials
8 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–8 of 8 trials
Recruiting
Phase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
Agios Pharmaceuticals, Inc.20 enrolled1 locationNCT07241234
Recruiting
Phase 1Phase 2
AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Phenylketonurias
NGGT INC.12 enrolled5 locationsNCT06332807
Not Yet Recruiting
Phase 1
A Trial to Examine the Amount of JNT-517 Entering the Bloodstream and the Effect of Food on Different Formulations of JNT-517
Phenylketonuria
CTI Clinical Trial and Consulting Services Australia Pty Ltd24 enrolled1 locationACTRN12625000641493
Withdrawn
Phase 1
A Phase 1 Randomized, Double-blind, Placebo-controlled Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of MZE782 in Healthy Adults
Chronic Kidney DiseasePhenylketonuria
Maze Therapeutics24 enrolled1 locationACTRN12625000488404
Recruiting
Phase 1Phase 2
Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Phenylketonurias
NGGT (Suzhou) Biotechnology Co., Ltd.18 enrolled2 locationsNCT06687733
Completed
Phase 1
A Phase 1, Three-Part Open-Label Drug-Drug Interaction Study in Healthy participants to Determine the Effects of Itraconazole on the Pharmacokinetics of JNT-517 (Victim) and the Effects of JNT-517 (Perpetrator) on the Pharmacokinetics of Midazolam and Pravastatin
Phenylketonuria
CTI Clinical Trial and Consulting Services Australia Pty Ltd36 enrolled1 locationACTRN12624000718549
Completed
Phase 1
First in human clinical study of a novel drug JNT-517 to assess its safety and tolerability in healthy volunteers
Phenylketonuria
CTI Clinical Trial and Consulting Services Australia Pty Ltd.91 enrolled1 locationACTRN12622001222730
Completed
Phase 1
A Phase 1, Randomized, Open-Label, Crossover Study to Evaluate the Relative Oral Bioavailability of 2 Formulations of PTC923 and the Food Effect on the Phase 3 Formulation When Administered as a Single Dose to Healthy Subjects
Phenylketonuria (PKU)
PTC Therapeutics Inc.32 enrolled1 locationACTRN12622000523707