BH4 Deficiency

Clinical trial pipeline · Data from ClinicalTrials.gov

See which BH4 Deficiency trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT04896281

Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU

Hyperphenylalaninemia is not only present in patients with congenital phenylketonuria. In adults with critical illness, hyperphenylalaninemia is noted in some p…

Taiwan100 participantsStarted 2021-07-30

Treatment: Phenylalanine-free milk

All recent trials (14 shown)

By InvitationNot Applicable

NCT03655223

Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…

United States30,000 participantsStarted 2018-10-15

Treatment: Confirmatory Testing

CompletedPhase 1/2

NCT01425528

Study of Kuvan Treatment in Adults With GTPCH Deficiency

The purpose of this study is to identify the dose of Kuvan needed to normalize the levels of tetrahydrobiopterin (BH4) in patients with a Guanosine Triphosphate…

United States12 participantsStarted 2011-08

Treatment: Sapropterin

BH4 Deficiency

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU

All recent trials (14 shown)

Early Check: Expanded Screening in Newborns

Study of Kuvan Treatment in Adults With GTPCH Deficiency

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU

All recent trials (14 shown)

Early Check: Expanded Screening in Newborns

Study of Kuvan Treatment in Adults With GTPCH Deficiency

Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of Phenylketo…

O2 Transport and Utilization in Health and Lung Disease

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phen…

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

BH4 Responsiveness in PAH Deficiency PKU Patients

Retrospective Observational Safety Effectiveness With Kuvan in hpA

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

Sapropterin Expanded Access Program

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety