PhenylketonuriasPhase 3NCT00225615
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
A Phase 3 study testing sapropterin dihydrochloride in people with Phenylketonurias, run by BioMarin Pharmaceutical at 14 sites in United States.
Trial summary
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- sapropterin dihydrochloride
- How many people
- 100 participants
- Where
- 14 sitesUnited States
- Study dates
- Nov 2005to Jun 2006
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 8 years of age and older
- Prior successful participation in Study PKU-003
- Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian
- For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those measures while participating in the study
- Willing and able to comply with study procedures
- Willing to continue current diet unchanged while participating in the study
Who cannot join
- Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
- Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level
- Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments
- Pregnant or breastfeeding, or planning pregnancy
- Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)
- Requirement for concomitant treatment with any drug known to inhibit folate synthesis (e.g., methotrexate)
- Concurrent use of levodopa
Trial locations and contacts
14 sites in 1 country.
14 sites in 1 country
United States14 sites
Study site
Atlanta, Georgia
Study site
Chicago, Illinois
Study site
Dallas, Texas
Study site
Los Angeles, California
Study site
Madison, Wisconsin
Study site
Minneapolis, Minnesota
Study site
New Haven, Connecticut
Study site
New York, New York
Study site
Oakland, California
Study site
Portland, Oregon
Study site
Sacramento, California
Study site
Salt Lake City, Utah
Study site
San Jose, California
Study site
St Louis, Missouri
About this study
Official title: A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria 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.
- Conditions
- Phenylketonurias
- Being tested
- sapropterin dihydrochloride
- Keywords
- Phenylalanine Hydroxylase
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sapropterin dihydrochlorideDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary objective:
- To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Secondary outcomes (6)
Secondary objectives:
To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
To evaluate the population pharmacokinetics of Phenoptin.
To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2005 (estimated)
- Study startNov 2005
- Study completionJun 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Alex Dorenbaum, MD, Study chair, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00225615
- Sponsor's protocol ID
- PKU-004
- Status last verified
- Aug 2009
- First submitted
- 22 Sep 2005
- Last updated
- 20 Aug 2009
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
- ClinicalTrials.gov ID
- NCT00225615
- Acronym / sponsor ID
- PKU-004
- Conditions
- Phenylketonurias
- Keywords
- Phenylalanine Hydroxylase
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- Nov 2005
- Study completion
- Jun 2006 (actual)
- First posted
- 26 Sep 2005 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 8 years of age and older
- Prior successful participation in Study PKU-003
- Willing and able to provide written informed consent or assent and written informed consent (if required) by a parent or legal guardian
- For females of child-bearing potential only: Negative urine pregnancy test within 24 hours prior to enrollment. Women using acceptable birth control measures must agree to continue to use those measures while participating in the study
- Willing and able to comply with study procedures
Key exclusion criteria (7 listed)
- Perceived to be unreliable or unavailable for study participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unavailable
- Withdrew from, or otherwise did not successfully complete, study PKU-003, except for subjects who were removed from the study because their blood Phe exceeded the alert level
- Expected to require any investigational agent or vaccine prior to completion of all scheduled study assessments
- Pregnant or breastfeeding, or planning pregnancy
- Concurrent disease or condition that would interfere with study participation or safety (e.g., seizure disorder, oral steroid-dependent asthma or other condition requiring oral or parenteral corticosteroid administration, or insulin-dependent diabetes)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sapropterin dihydrochloride | Drug | – |
Primary outcome
Primary objective:
- To evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
6 secondary outcomes
Secondary objectives:
To compare the safety and tolerability of three different doses of Phenoptin treatment in subjects with PKU.
To determine the effect of various doses of Phenoptin on blood phenylalanine (Phe) levels.
To evaluate the population pharmacokinetics of Phenoptin.
To evaluate the ability of Phenoptin to reduce phenylalanine (Phe) levels over a 24-hour period.
To evaluate the persistence of benefit of Phenoptin treatment in the subject population as evidenced by long-term control of blood Phe levels.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2009.
Related trials for Phenylketonurias
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RecruitingPhase 3
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Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alex Dorenbaum, MD study chair
BioMarin Pharmaceutical
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing sapropterin dihydrochloride in people with Phenylketonurias, run by BioMarin Pharmaceutical at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Aug 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary objective:. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Aug 2009.