Clinical Trials for Phenylketonuria
Clinical trials are research studies conducted in an effort to improve overall patient health and care.
Each trial involves running supervised tests to determine the effectiveness and safety of new drugs, procedures and/or devices with the aim of answering scientific questions about a disease or condition.
Risks and side-effects are also evaluated during these trials with results ranging from being unpleasant, with side effects such as headaches, to more serious or even life-threatening risks.
A clinical trial may be separated into phases, or steps, with each step designed to answer a separate research question. This maybe reflected in the trial name.
A brief description of each phase:
- Phase I: Usually designed to evaluate safety, determine a safe dosage range, and identify side effects on a small group of patients.
- Phase II: If Phase I is successful, the trial is then repeated with a larger group to further evaluate its effect and safety.
- Phase III: Trials are conducted on an ever larger group and are compared with the best current treatment while gathering more information on effect and safety.
- Phase IV: These studies monitor long-term side effects after the treatment has been marketed.
Clinical Trials data for Phenylketonuria is grouped into the following categories:-
The format for each of the following is:
- The name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
- followed by a list of drug/s, if any, used in the trial
ClinicalTrials.gov has listed the following trials for Phenylketonuria:
- Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria - This study has been completed (Current: 23 Nov 2006)
- The Early History of Universal Screening for Metabolic Disorders - This study is currently recruiting patients (Current: 23 Nov 2006)
- Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels - This study is no longer recruiting patients (Current: 23 Nov 2006) - sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
- Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects With Phenylketonuria - This study is no longer recruiting patients (Current: 23 Nov 2006) - sapropterin dihydrochloride
- A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels - This study is no longer recruiting patients (Current: 23 Nov 2006) - sapropterin dihydrochloride
- A Phase 3, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-Restricted Diet - This study is currently recruiting patients (Current: 23 Nov 2006) - sapropterin dihydrochloride
- Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU) - This study has been completed (Current: 23 Nov 2006)
- Response to Phenylketonuria to Tetrahydrobiopterin (BH4) - This study is currently recruiting patients (Current: 23 Nov 2006) - tetrahydrobiopterin (BH4)
- Study of BH4, a New and Simple Treatment of Mild PKU - This study has been completed (Current: 23 Nov 2006) - 5,6,7,8-tetrahydrobiopterin
- A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - This study is not yet open for patient recruitment (Current: 23 Nov 2006) - sapropterin dihydrochloride
- Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency - This study is currently recruiting patients (Current: 23 Nov 2006) - Phenoptin (drug)
The format for each of the following is:
- The drug,
- followed by the name of the trial,
- followed by whether the trial is recruiting patients,
- The trial may not have started and not yet recruiting, or the trial may have started and do not need any more recruitements, or the trial may be completed.
- Note**: Please follow the link to determine the current trial status.
ClinicalTrials.gov has listed the following drugs in trials for Phenylketonuria:
- 5,6,7,8-tetrahydrobiopterin - Study of BH4, a New and Simple Treatment of Mild PKU - This study has been completed (Current: 23 Nov 2006)
- Phenoptin (drug) - Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency - This study is currently recruiting patients (Current: 23 Nov 2006)
- Sapropterin Dihydrochloride, 6R-Bh4, Tetrahydrobiopterin - Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels - This study is no longer recruiting patients (Current: 23 Nov 2006)
- Sapropterin Dihydrochloride - A Phase 3, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-Restricted Diet - This study is currently recruiting patients (Current: 23 Nov 2006)
- Sapropterin Dihydrochloride - A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels - This study is no longer recruiting patients (Current: 23 Nov 2006)
- Sapropterin Dihydrochloride - A Phase 3b, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 - This study is not yet open for patient recruitment (Current: 23 Nov 2006)
- Sapropterin Dihydrochloride - Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin Treatment in Subjects With Phenylketonuria - This study is no longer recruiting patients (Current: 23 Nov 2006)
- Tetrahydrobiopterin (Bh4) - Response to Phenylketonuria to Tetrahydrobiopterin (BH4) - This study is currently recruiting patients (Current: 23 Nov 2006)
The trial data on this page has been sourced from
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