PhenylketonuriaPhase 2NCT00925054

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

A Phase 2 study testing rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg and rAvPAL-PEG 0.01 mg/kg in people with Phenylketonuria, run by BioMarin Pharmaceutical at 11 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether repeated administration is safe. Read more about this study

Who is it for?: Ages 16 to 55 years
Conditions the study is for: Phenylketonuria
What is being tested?: rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg and rAvPAL-PEG 0.01 mg/kg
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 40 participants
Where: 11 sitesUnited States
Study dates: Sep 2009to Jun 2015
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 16 to 55 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

For subjects who did not participate in PAL-001, diagnosis of PKU with both of the following: Current blood Phe concentration of ≥ 600 mmol/L at Screening and average blood Phe concentration of ≥ 600 µmol/L over the past 3 years, using available data.
For subjects who did not participate in PAL-001, evidence that the subject is a non-responder to Kuvan® treatment (ie, 4 weeks of treatment with 20 mg/kg/day of Kuvan®, insufficient response per investigator determination, and treatment end date ≥ 14 days prior to Day 1 [ie, first dose]). Subjects who have had a previous response to Kuvan® treatment but are not currently taking Kuvan® because of noncompliance and have been off treatment for ≥ 6 months prior to Screening are eligible for participation.
Willing and able to provide written, signed informed consent, or, in the case of participants under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained, and prior to any research-related procedures.
Between the ages of 16 and 55 years, inclusive.
Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.
Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.

Show all 9Show fewer

Maintained a stable diet with no significant modifications during the 4 weeks preceding the administration of study drug.
In generally good health as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) at Screening.
Willing and able to comply with study procedures.

Who cannot join

Use of any investigational product (with the exception of rAvPAL-PEG) or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
Use of any medication that is intended to treat PKU within 14 days prior to the administration of study drug.
Use or planned use of any injectable drugs containing PEG (other than rAvPAL-PEG), including Depo-Provera, within 3 months prior to Screening and during study participation.
A prior reaction that included systemic symptoms (eg, respiratory or gastrointestinal problems, hypotension, angioedema, anaphylaxis) to rAvPAL-PEG or a PEG containing product. Subjects with a prior systemic reaction of generalized rash may be eligible for participation per the discretion of the Principal Investigator in consultation with the Sponsor's Medical Officer.
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study.
Concurrent disease or condition that would interfere with study participation or safety (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease).

Show all 9Show fewer

Any condition that, in the view of the PI, places the subject at high risk of poor treatment compliance or of not completing the study.
Alanine aminotransferase (ALT) concentration > 2 times the upper limit of normal.
Creatinine > 1.5 times the upper limit of normal.

Trial locations and contacts

11 sites in 1 country.

City, postcode or hospital

11 sites in 1 country

United States11 sites

Akron Children's Hospital
Akron, Ohio, 44308
Albany Medical Center
Albany, New York, 12208
The Children's Hospital
Aurora, Colorado, 80045
Children's Memorial Hospital
Chicago, Illinois, 60614
Emory Universty
Decatur, Georgia, 30033
University of Minnesota Medical Center-Fairview
Minneapolis, Minnesota, 55455
Mount Sinai School of Medicine
New York, New York, 10029
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Oregon Health & Science University
Portland, Oregon, 97239
University of Utah Hospital
Salt Lake City, Utah, 84132
Washington University Center for Applied Research Sciences
St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU

Read the detailed description

This is a 2 part, Phase 2, open-label dose-finding study in approximately 35 subjects with PKU. Seven dose cohorts are planned, each consisting of 5 subjects. In Part 1, the planned starting dose levels are those tested in PAL 001 (0.001, 0.003, 0.01, 0.03, 0.1, 0.3, and 1.0 mg/kg), provided no dose limiting toxicity was observed in PAL 001. In Parts 1 and 2, study drug will be administered by clinic staff.

Subjects who completed participation in PAL 001 will receive priority to participate in PAL 002. rAvPAL PEG naïve subjects will be enrolled to fill any dose cohort vacancies resulting from subjects who did not complete PAL 001 or who chose not to continue into PAL 002. In addition, if the number of dose cohorts determined in PAL 001 is less than 7, additional naïve subjects may be added to the existing dose cohorts to provide a total of approximately 35 subjects entering Part 1 of PAL 002. Furthermore, if serial dosing of cohorts in Part 1 of PAL 002 is stopped, additional subjects (either naïve subjects or PAL 001 subjects) may be added to the existing cohorts so that total study enrollment is approximately 35 subjects. In any of these cases, additional subjects will be enrolled sequentially from lowest to highest dose cohort.

Diet will not be altered during the course of this study, except as necessary for safety.

Subjects will be evaluated for safety and for blood Phe concentrations throughout the study. Toxicity will be measured according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 3.

A Data Monitoring Committee will monitor the study.

Conditions: Phenylketonuria
Being tested: rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
Keywords: PKU, PEG PAL, BioMarin
Medical subject headings: Phenylketonurias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

rAvPAL-PEG 0.001 mg/kgDrug
In Part 1, the planned starting dose levels is 0.001 mg/kg
rAvPAL-PEG 0.003 mg/kgDrug
In Part 1, the planned starting dose levels is 0.003 mg/kg
rAvPAL-PEG 0.01 mg/kgDrug
In Part 1, the planned starting dose levels is 0.01 mg/kg
rAvPAL-PEG 0.03 mg/kgDrug
In Part 1, the planned starting dose levels is 0.03 mg/kg
rAvPAL-PEG 0.1 mg/kgDrug
In Part 1, the planned starting dose levels is 0.1 mg/kg

Study groups in NCT00925054
Study group	Role	What they receive
rAvPAL-PEG 0.001 mg/kg	Experimental	Subjects will start on rAvPAL-PEG 0.001 mg/kg Receives: Drug: rAvPAL-PEG 0.001 mg/kg
rAvPAL-PEG 0.003 mg/kg	Experimental	Subjects will start on rAvPAL-PEG 0.003 mg/kg Receives: Drug: rAvPAL-PEG 0.003 mg/kg
rAvPAL-PEG 0.01 mg/kg	Experimental	Subjects will start on rAvPAL-PEG 0.01 mg/kg Receives: Drug: rAvPAL-PEG 0.01 mg/kg
rAvPAL-PEG 0.03 mg/kg	Experimental	Subjects will start on rAvPAL-PEG 0.03 mg/kg Receives: Drug: rAvPAL-PEG 0.03 mg/kg
rAvPAL-PEG 0.1 mg/kg	Experimental	Subjects will start on rAvPAL-PEG 0.1 mg/kg Receives: Drug: rAvPAL-PEG 0.1 mg/kg

What is being measured

The primary outcome is the main question the study is designed to answer.

Blood Phenylalanine Concentrations
Measured: Baseline, Week 1/Day 5, Week 7, Week 16/Day 106

Secondary outcomes (8)

Study Drug Related Adverse Events
Measured: Screening, Weeks 1-22
Number of Participants With Positive PAL IgG Antibody
Antibody against PAL (phenylalanine ammonia lyase) measured over time
Measured: Baseline, Week 12
Number of Participants With Positive PAL IgM Antibody
Antibody positivity over time
Measured: Baseline, Week 12
Number of Participants With Positive PEG IgM Antibody
Antibody positivity over time
Measured: Baseline, Week 16
Number of Participants With Positive PEG IgG Antibody
Antibody positivity over time
Measured: Baseline, Week 16
Percentage of Participants With Positive Neutralizing Antibodies [NAb]
Antibody positivity over time
Measured: Baseline, Week 12
Percentage of Participants With Positive PAL IgE Antibody
Antibody positivity over time
Measured: Baselline, Week 12
Percentage of Participants With Positive Anti-PAL-PEG IgE Antibodies
Antibody positivity over time
Measured: Baseline, Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2009Jun 2015

First posted19 Jun 2009 (estimated)
Study startSep 2009
Primary completionJun 2015
Study completionJun 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for PhenylketonuriaStudies 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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 40 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00925054
Sponsor's protocol ID: PAL-002
Status last verified: Feb 2019
First submitted: 17 Jun 2009
Last updated: 26 Feb 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Publications

Burton BK, Longo N, Vockley J, Grange DK, Harding CO, Decker C, Li M, Lau K, Rosen O, Larimore K, Thomas J; PAL-002 and PAL-004 Investigators. Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up. Mol Genet Metab. 2020 Aug;130(4):239-246. doi: 10.1016/j.ymgme.2020.06.006. Epub 2020 Jun 16.
PubMed 32593547
Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S. Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial. Lancet. 2014 Jul 5;384(9937):37-44. doi: 10.1016/S0140-6736(13)61841-3. Epub 2014 Apr 14.
PubMed 24743000

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 26 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU
ClinicalTrials.gov ID: NCT00925054
Acronym / sponsor ID: PAL-002
Conditions: Phenylketonuria
Keywords: PKU, PEG PAL, BioMarin
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 40 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: Sep 2009
Primary completion: Jun 2015 (actual)
Study completion: Jun 2015 (actual)
First posted: 19 Jun 2009 (estimated)
Results: Posted 26 Feb 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Diet will not be altered during the course of this study, except as necessary for safety.

A Data Monitoring Committee will monitor the study.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 5

Arm	Type	Receives
rAvPAL-PEG 0.001 mg/kg	Experimental	Drug: rAvPAL-PEG 0.001 mg/kgSubjects will start on rAvPAL-PEG 0.001 mg/kg
rAvPAL-PEG 0.003 mg/kg	Experimental	Drug: rAvPAL-PEG 0.003 mg/kgSubjects will start on rAvPAL-PEG 0.003 mg/kg
rAvPAL-PEG 0.01 mg/kg	Experimental	Drug: rAvPAL-PEG 0.01 mg/kgSubjects will start on rAvPAL-PEG 0.01 mg/kg
rAvPAL-PEG 0.03 mg/kg	Experimental	Drug: rAvPAL-PEG 0.03 mg/kgSubjects will start on rAvPAL-PEG 0.03 mg/kg
rAvPAL-PEG 0.1 mg/kg	Experimental	Drug: rAvPAL-PEG 0.1 mg/kgSubjects will start on rAvPAL-PEG 0.1 mg/kg

Who may qualify

Ages: 16 to 55 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (9 listed)

For subjects who did not participate in PAL-001, diagnosis of PKU with both of the following: Current blood Phe concentration of ≥ 600 mmol/L at Screening and average blood Phe concentration of ≥ 600 µmol/L over the past 3 years, using available data.
For subjects who did not participate in PAL-001, evidence that the subject is a non-responder to Kuvan® treatment (ie, 4 weeks of treatment with 20 mg/kg/day of Kuvan®, insufficient response per investigator determination, and treatment end date ≥ 14 days prior to Day 1 [ie, first dose]). Subjects who have had a previous response to Kuvan® treatment but are not currently taking Kuvan® because of noncompliance and have been off treatment for ≥ 6 months prior to Screening are eligible for participation.
Willing and able to provide written, signed informed consent, or, in the case of participants under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian, after the nature of the study has been explained, and prior to any research-related procedures.
Between the ages of 16 and 55 years, inclusive.
Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.

Key exclusion criteria (9 listed)

Use of any investigational product (with the exception of rAvPAL-PEG) or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
Use of any medication that is intended to treat PKU within 14 days prior to the administration of study drug.
Use or planned use of any injectable drugs containing PEG (other than rAvPAL-PEG), including Depo-Provera, within 3 months prior to Screening and during study participation.
A prior reaction that included systemic symptoms (eg, respiratory or gastrointestinal problems, hypotension, angioedema, anaphylaxis) to rAvPAL-PEG or a PEG containing product. Subjects with a prior systemic reaction of generalized rash may be eligible for participation per the discretion of the Principal Investigator in consultation with the Sponsor's Medical Officer.
Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study.

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
rAvPAL-PEG 0.001 mg/kgIn Part 1, the planned starting dose levels is 0.001 mg/kg	Drug	rAvPAL-PEG 0.001 mg/kg
rAvPAL-PEG 0.003 mg/kgIn Part 1, the planned starting dose levels is 0.003 mg/kg	Drug	rAvPAL-PEG 0.003 mg/kg
rAvPAL-PEG 0.01 mg/kgIn Part 1, the planned starting dose levels is 0.01 mg/kg	Drug	rAvPAL-PEG 0.01 mg/kg
rAvPAL-PEG 0.03 mg/kgIn Part 1, the planned starting dose levels is 0.03 mg/kg	Drug	rAvPAL-PEG 0.03 mg/kg
rAvPAL-PEG 0.1 mg/kgIn Part 1, the planned starting dose levels is 0.1 mg/kg	Drug	rAvPAL-PEG 0.1 mg/kg

Primary outcome

Blood Phenylalanine Concentrations
Time frame: Baseline, Week 1/Day 5, Week 7, Week 16/Day 106

8 secondary outcomes

Study Drug Related Adverse Events
Time frame: Screening, Weeks 1-22
Number of Participants With Positive PAL IgG Antibody
Time frame: Baseline, Week 12
Number of Participants With Positive PAL IgM Antibody
Time frame: Baseline, Week 12
Number of Participants With Positive PEG IgM Antibody
Time frame: Baseline, Week 16
Number of Participants With Positive PEG IgG Antibody
Time frame: Baseline, Week 16
Percentage of Participants With Positive Neutralizing Antibodies [NAb]
Time frame: Baseline, Week 12
Percentage of Participants With Positive PAL IgE Antibody
Time frame: Baselline, Week 12
Percentage of Participants With Positive Anti-PAL-PEG IgE Antibodies
Time frame: Baseline, Week 12

Results and safety

Results posted to the registry on 26 Feb 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Starting Dose=0.001 mg/kg	7	7
Starting Dose=0.003 mg/kg	9	8
Starting Dose=0.01 mg/kg	13	12
Starting Dose=0.03 mg/kg	1	0
Starting Dose=0.1 mg/kg	10	10

Primary outcome: Blood Phenylalanine Concentrations

Time frame: Baseline, Week 1/Day 5, Week 7, Week 16/Day 106. Mean (Standard Deviation). Unit: umol/L

Measure	Starting Dose=0.001 mg/kg n = 7	Starting Dose=0.003 mg/kg n = 9	Starting Dose=0.01 mg/kg n = 13	Starting Dose=0.03 mg/kg n = 1	Starting Dose =0.1 mg/kg n = 10	Total n = 40
Baseline	1382.7 (191.40)	1336.1 (344.87)	1290.5 (459.43)	846.0	1310.3 (319.01)	1310.8 (353.86)
Week 1 Day 5	1405.3 (190.87)	1276.3 (410.22)	1216.5 (434.53)	682.0	863.7 (307.59)	1158.5 (403.63)
Week 7	1436.0 (274.08)	1324.8 (304.64)	1108.7 (510.74)	1849.0	1321.6 (350.78)	1284.5 (405.75)
week 16 Day 106	1284.0 (221.73)	1049.4 (378.00)	1093.5 (445.02)	–	930.7 (331.36)	1088.5 (367.67)

Population analysed: Efficacy Population

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Screening- Week 22. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Starting Dose=0.001 mg/kg	0 of 7 (0%)	7 of 7 (100%)	0 of 7
Starting Dose=0.003 mg/kg	0 of 9 (0%)	9 of 9 (100%)	0 of 9
Starting Dose=0.01 mg/kg	1 of 13 (7.7%)	13 of 13 (100%)	0 of 13
Starting Dose=0.03 mg/kg	0 of 1 (0%)	1 of 1 (100%)	0 of 1
Starting Dose=0.1 mg/kg	1 of 10 (10%)	10 of 10 (100%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32593547Burton BK, Longo N, Vockley J, Grange DK, Harding CO, Decker C, Li M, Lau K, Rosen O, Larimore K, Thomas J; PAL-002 and PAL-004 Investigators. Pegvaliase for the treatment of phenylketonuria: Results of the phase 2 dose-finding studies with long-term follow-up. Mol Genet Metab. 2020 Aug;130(4):239-246. doi: 10.1016/j.ymgme.2020.06.006. Epub 2020 Jun 16.
Derived from this study: PubMed 24743000Longo N, Harding CO, Burton BK, Grange DK, Vockley J, Wasserstein M, Rice GM, Dorenbaum A, Neuenburg JK, Musson DG, Gu Z, Sile S. Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial. Lancet. 2014 Jul 5;384(9937):37-44. doi: 10.1016/S0140-6736(13)61841-3. Epub 2014 Apr 14.
Individual participant dataSharing planned: No

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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.

Ari Gershman, MD study director

BioMarin Pharmaceutical

11 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg and rAvPAL-PEG 0.01 mg/kg in people with Phenylketonuria, run by BioMarin Pharmaceutical at 11 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 9 inclusion and 9 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 26 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 26 Feb 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Blood Phenylalanine Concentrations (time frame: Baseline, Week 1/Day 5, Week 7, Week 16/Day 106). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00925054, which also lists 2 linked publications.

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 26 Feb 2019.