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Home › Medications › sapropterin

sapropterin

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials of sapropterin

12 trials tracked of sapropterin: 4 in phase 3 or 4.

12Trials tracked
4Phase 3 & 4
0Recruiting
0With published results
Phase distribution
Phase 3 4 Phase 2 5 Other / NA 3
  1. Phase 3 Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU Completed
  2. Phase 3 Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients Completed
  3. Phase 3 Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet Completed
  4. Phase 3 Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 Completed
  5. Phase 2 A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction Completed
  6. Phase 2 Study of Kuvan Treatment in Adults With GTPCH Deficiency Completed
Show 6 more trials
  1. Phase 2 A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease Completed
  2. Phase 2 Sapropterin as a Treatment for Autistic Disorder Completed
  3. Phase 2 A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease Completed
  4. N/A Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders Completed
  5. N/A Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy Completed
  6. N/A Impact of KUVAN® on Gastric Relaxation in Women With Diabetic Gastroparesis. Completed

Showing the 12 most-cited and recently-updated of 12 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Sepiapterin reduces blood phenylalanine more than sapropterin in PKU patients
Drug Pipeline RCT
Sepiapterin reduces blood phenylalanine more than sapropterin in PKU patients Sepiapterin beats sapropterin for lowering blood levels
This Phase 3, randomized, crossover trial in 82 children and adults with phenylketonuria compared sepiapterin to sapropterin.
New research shows a specific vitamin treatment works much better than current options for a rare genetic condition.
May 1, 2026