Clinical trials: Phenylketonuria
Phenylketonuria
Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors
To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation.
- Trial name
- NCT00728676
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Shoji Yano
Alcohol Dependence
Stress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in Alcoholism
This proposal is part of the INIA Stress Consortium. This study will 1. explore the contributions of lifetime trauma, recent stress, and alcohol use on stress-hormone axis disruption in treatment seeking, one-month abstinent…
- Trial name
- NCT00744588
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- University of Texas Southwestern Medical Center
Phenylketonuria
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.
- Trial name
- NCT00332189
- Testing
- sapropterin dihydrochloride
- Where
- 14 sites in United States
- Volunteers
- 111 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonurias
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
- Trial name
- NCT00225615
- Testing
- sapropterin dihydrochloride
- Where
- 14 sites in United States
- Volunteers
- 100 people
- Sponsor
- BioMarin Pharmaceutical
Hypertension
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
The purpose of this study is to determine whether 6R-BH4 (sapropterin dihydrochloride) is safe and effective in the treatment of poorly controlled hypertension in the presence or absence of type 2 diabetes.
- Trial name
- NCT00325962
- Testing
- 6R-BH4 (sapropterin dihydrochloride)
- Where
- 24 sites in United States
- Volunteers
- 84 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria
Sapropterin Expanded Access Program
The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded…
- Trial name
- NCT00484991
- Testing
- Sapropterin dihydrochloride
- Where
- 36 sites in United States
- Sponsor
- BioMarin Pharmaceutical
Phenylalanine Hydroxylase Deficiencies
Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive to BH4.
- Trial name
- NCT00432822
- Testing
- tetrahydrobiopterin (BH4)
- Volunteers
- 50 people
- Sponsor
- Orphanetics Pharma Entwicklungs GmbH
Phenylketonurias
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU)…
- Trial name
- NCT00104260
- Testing
- sapropterin dihydrochloride
- Where
- 13 sites in United States
- Volunteers
- 700 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonuria
Study of BH4, a New and Simple Treatment of Mild PKU
The main purpose is to test whether treatment with BH4-tablets can replace the protein restrictive diet in patients with mild PKU caused by a certain frequent mutation.
- Trial name
- NCT00260000
- Testing
- 5,6,7,8-tetrahydrobiopterin
- Where
- Denmark
- Volunteers
- 15 people
- Sponsor
- The Kennedy Institute-National Eye Clinic
Phenylketonuria
Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria
OBJECTIVES: I. Assess the impact of a phenylalanine restricted diet during pregnancy on symptoms in offspring of patients with phenylketonuria.
- Trial name
- NCT00006142
- Testing
- phenylalanine restricted diet
- Sponsor
- National Center for Research Resources (NCRR)
Phenylketonuria
Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)
Phenylketonuria (PKU) is a rare genetic condition. If not treated, PKU can cause severe mental retardation. Women with PKU are advised to eat a special diet when pregnant to prevent mental retardation in their children.
- Trial name
- NCT00065299
- Testing
- Restricted phenylalanine diet
- Where
- 3 sites in United States
- Volunteers
- 572 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)