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Clinical trial

A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With Hyperphenylalaninemia

NCT03519711 is registered in ClinicalTrials.gov with PTC Therapeutics as lead sponsor.

NCT NCT03519711
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With Hyperphenylalaninemia
Lead sponsor
PTC Therapeutics
Phase
Phase 1/2
Overall status
Completed
Start date
January 3, 2019
Completion date
October 2, 2020
Enrollment
8 (actual)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

bh4 deficiency, hyperphenylalaninemia