BioavailabilityPhase 1NCT06481709
Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects
A Phase 1 study testing RLF-OD032 and Kuvan in people with Bioavailability, run by APR Applied Pharma Research s.a. at 1 site in Canada.
Trial summary
This is a single center, Phase 1, randomized, open-label, single-dose, 4 treatment, 4-period, 4-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin from the Test and Reference products, and to evaluate the effect of food and the effect of water administration on the bioavailability… Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Bioavailability
- What is being tested?
- RLF-OD032 and Kuvan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteCanada
- Study dates
- 30 Jul 2024to 31 Aug 2024
- Phase
- Phase 1
- 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 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Male or female, light smoker (no more than 10 cigarettes daily) or non smoker, ≥18 and ≤50 years of age, with body mass index (BMI) ≥18.5 and ≤30.0 kg/m2 and body weight ≥50.0 kg for males and ≥45.0 kg for females.
- Healthy as defined by:
- the absence of clinically significant illness and surgery within 30 days prior to dosing.
- the absence of clinically significant history of neurological, endocrine, cardiovascular, respiratory, hematological, immunological, psychiatric, gastrointestinal, renal, hepatic, and metabolic disease.
- Female subjects of non-childbearing potential must be:
- post-menopausal (no menstrual period at least 12 consecutive months without any other medical cause and FSH and LH values consistent with being menopausal); or
Show all 10Show fewer
- surgically sterile (bilateral oophorectomy, bilateral salpingectomy, hysterectomy or tubal ligation) at least 3 months prior to dosing.
- Sexually active female subjects of childbearing potential must be willing to use an acceptable contraceptive method throughout the study as detailed in the protocol.
- Willing to take off dentures or mouth piercing at the time of dosing.
- Able to understand the study procedures and provide signed informed consent to participate in the study.
Who cannot join
- Any clinically significant abnormal finding at physical examination at screening.
- Clinically significant abnormal laboratory test results (may be repeated up to two times) or positive serology test results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV)-1 and HIV-2 antibodies at screening.
- Positive pregnancy test or lactating female subject.
- Positive urine drug screen.
- Known allergic reactions to sapropterin dihydrochloride or other related drugs, or to any excipient in the formulation.
- Clinically significant ECG abnormalities or vital signs abnormalities (systolic blood pressure lower than 90 or over 150 mmHg, diastolic blood pressure lower than 50 or over 90 mmHg, or pulse rate less than 50 or over 100 bpm) at screening. ECG and vitals signs may be repeated up to two times, to determine if the values are significantly abnormal.
Show all 19Show fewer
- Recreational use of soft drugs (such as marijuana) within 1 month or hard drugs (such as cocaine, phencyclidine [PCP], crack, opioid derivatives including heroin, and amphetamine derivatives) within 3 months prior to screening.
- History of alcohol addiction requiring treatment.
- History of abuse of medicinal product or drugs within the last 3 years.
- History or presence of alcoholism within the last 3 years. (>40 g ethanol/day or more than 10 units per week [1 unit =150 mL of wine, or 360 mL of beer, or 45 mL of 45% alcohol]).
- Use of medications within the timeframes specified in the protocol
- Participation in a clinical research study involving the administration of an investigational or marketed drug or device within 30 days prior to dosing, administration of a biological product in the context of a clinical research study within 90 days prior to dosing, or concomitant participation in an investigational study involving no drug or device administration.
- Known predisposition to seizures.
- Donation of plasma within 7 days prior to dosing or donation or loss of 500 mL or more of whole blood within 8 weeks prior to dosing.
- Presence of orthodontic braces or orthodontic retention wires, or any physical findings in the mouth or tongue that would be likely to interfere with successful completion of the dosing procedure.
- Females who:
- Have discontinued or changed the use of implanted, intrauterine, intravaginal, or injected hormonal contraceptives within 6 months prior to study treatment administration.
- Have discontinued or changed the use of oral or patch hormonal contraceptives within 1 month prior to study treatment administration.
- Any reason which, in the opinion of the Investigator, would prevent the subject from participating in the study.
Trial locations and contacts
1 site in 1 country.
Canada1 site
Pharma Medica Research Inc. 4770 Sheppard Ave E,
Toronto, Ontario, M1S3V6
About this study
Official title: A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension (Product Code: RLF-OD032) (Test) With Kuvan® (Sapropterin Dihydrochloride) 100 mg Powder for Oral Solution (Reference) and to Evaluate the Effect of Food and the Effect of Water on the Bioavailability of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension in Healthy Subjects
This is a single center, Phase 1, randomized, open-label, single-dose, 4 treatment, 4-period, 4-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin from the Test and Reference products, and to evaluate the effect of food and the effect of water administration on the bioavailability of sapropterin from the Test product in healthy subjects.
.
Read the detailed description
In each period, subjects will receive a single 10 mg/kg dose of sapropterin dihydrochloride on Day 1, under fasting or fed conditions, and with or without water, followed by 24 hours of PK sampling. The study will include a screening visit from Day -30 to Day -1. In each period, eligible subjects will be admitted to the clinical site on Day -1 and will be confined until completion of the assessments on Day 2. There will be a washout period of at least 7 days between doses.
- Conditions
- Bioavailability
- Being tested
- RLF-OD032, Kuvan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RLF-OD032Drug
Also called Sapropterin dihydrochloride
100 mg/mL oral suspension
KuvanDrug
Also called Sapropterin dihydrochloride
100 mg powder for oral solution
| Study group | Role | What they receive |
|---|---|---|
| Sequence DCAB | Other | Where the Subject receives in this order
D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions
C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions.
A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions.
B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. Receives: Drug: RLF-OD032; Drug: Kuvan |
| Sequence ADBC | Other | Where the Subject receives in this order
A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions.
D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions
B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions.
C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. Receives: Drug: RLF-OD032; Drug: Kuvan |
| Sequence BACD | Other | Where the Subject receives in this order
B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions.
A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions.
C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions.
D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions Receives: Drug: RLF-OD032; Drug: Kuvan |
| Sequence CBDA | Other | Where the Subject receives in this order
C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions.
B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions.
D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions
A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. Receives: Drug: RLF-OD032; Drug: Kuvan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Uncorrected Sapropterin AUC0-t
Area under the concentration-time curve from time zero until the last observed concentration, as calculated by the linear up/log down variant of the trapezoidal method
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Baseline-corrected Sapropterin AUC0-t
Area under the concentration-time curve from time zero until the last observed concentration, as calculated by the linear up/log down variant of the trapezoidal method
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Uncorrected Sapropterin Cmax
Maximum observed concentration
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Baseline-corrected Sapropterin Cmax
Maximum observed concentration
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Uncorrected Sapropterin AUC0-inf
Area under the concentration-time curve from time zero to infinity (extrapolated)
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Baseline-corrected Sapropterin AUC0-inf
Area under the concentration-time curve from time zero to infinity (extrapolated)
Measured: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2024
- Study start30 Jul 2024
- Primary completion31 Aug 2024
- Study completion31 Aug 2024
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.
- Other trials for BioavailabilityStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- In each period, subjects will receive one of the following treatments, according to the randomization scheme: Treatment A: 1 x 10 mg/kg dose\* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. Treatment B: 1 x 10 mg/kg dose\* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. Treatment C: 1 x 10 mg/kg dose\* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. Treatment D: 1 x 10 mg/kg dose\* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- APR Applied Pharma Research s.a.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06481709
- Sponsor's protocol ID
- 2023-5439
- Status last verified
- Nov 2023
- First submitted
- 6 Jun 2024
- Last updated
- 12 Jun 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot, Phase 1, Randomized, Open-Label, Single-Dose, Four-Way Crossover Study to Compare the Pharmacokinetics of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension (Product Code: RLF-OD032) (Test) With Kuvan® (Sapropterin Dihydrochloride) 100 mg Powder for Oral Solution (Reference) and to Evaluate the Effect of Food and the Effect of Water on the Bioavailability of Sapropterin Dihydrochloride 100 mg/mL Oral Suspension in Healthy Subjects
- ClinicalTrials.gov ID
- NCT06481709
- Acronym / sponsor ID
- 2023-5439
- Conditions
- Bioavailability
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- APR Applied Pharma Research s.a.University, hospital or other
- Study start
- 30 Jul 2024 (actual)
- Primary completion
- 31 Aug 2024 (actual)
- Study completion
- 31 Aug 2024 (actual)
- First posted
- 1 Jul 2024 (actual)
- Results
- Posted 12 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single center, Phase 1, randomized, open-label, single-dose, 4 treatment, 4-period, 4-sequence, crossover study designed to compare the pharmacokinetics (PK) of sapropterin from the Test and Reference products, and to evaluate the effect of food and the effect of water administration on the bioavailability of sapropterin from the Test product in healthy subjects.
.
Detailed description
In each period, subjects will receive a single 10 mg/kg dose of sapropterin dihydrochloride on Day 1, under fasting or fed conditions, and with or without water, followed by 24 hours of PK sampling. The study will include a screening visit from Day -30 to Day -1. In each period, eligible subjects will be admitted to the clinical site on Day -1 and will be confined until completion of the assessments on Day 2. There will be a washout period of at least 7 days between doses.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- In each period, subjects will receive one of the following treatments, according to the randomization scheme: Treatment A: 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. Treatment B: 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. Treatment C: 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. Treatment D: 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions
| Arm | Type | Receives |
|---|---|---|
| Sequence DCAB | Other | Drug: RLF-OD032, Drug: KuvanWhere the Subject receives in this order D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. |
| Sequence ADBC | Other | Drug: RLF-OD032, Drug: KuvanWhere the Subject receives in this order A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. |
| Sequence BACD | Other | Drug: RLF-OD032, Drug: KuvanWhere the Subject receives in this order B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions |
| Sequence CBDA | Other | Drug: RLF-OD032, Drug: KuvanWhere the Subject receives in this order C is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered with water under fed conditions. B is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fed conditions. D is 1 x 10 mg/kg dose* of Kuvan (sapropterin dihydrochloride) 100 mg powder for oral solution dissolved in water and administered under fed conditions A is 1 x 10 mg/kg dose* of sapropterin dihydrochloride oral suspension 100 mg/mL administered without water under fasting conditions. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Male or female, light smoker (no more than 10 cigarettes daily) or non smoker, ≥18 and ≤50 years of age, with body mass index (BMI) ≥18.5 and ≤30.0 kg/m2 and body weight ≥50.0 kg for males and ≥45.0 kg for females.
- Healthy as defined by:
- Female subjects of non-childbearing potential must be:
- Sexually active female subjects of childbearing potential must be willing to use an acceptable contraceptive method throughout the study as detailed in the protocol.
- Willing to take off dentures or mouth piercing at the time of dosing.
Key exclusion criteria (19 listed)
- Any clinically significant abnormal finding at physical examination at screening.
- Clinically significant abnormal laboratory test results (may be repeated up to two times) or positive serology test results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV)-1 and HIV-2 antibodies at screening.
- Positive pregnancy test or lactating female subject.
- Positive urine drug screen.
- Known allergic reactions to sapropterin dihydrochloride or other related drugs, or to any excipient in the formulation.
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 |
|---|---|---|
| RLF-OD032Also: Sapropterin dihydrochloride100 mg/mL oral suspension | Drug | Sequence ADBC, Sequence BACD, Sequence CBDA, Sequence DCAB |
| KuvanAlso: Sapropterin dihydrochloride100 mg powder for oral solution | Drug | Sequence ADBC, Sequence BACD, Sequence CBDA, Sequence DCAB |
Primary outcome
Uncorrected Sapropterin AUC0-t
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Area under the concentration-time curve from time zero until the last observed concentration, as calculated by the linear up/log down variant of the trapezoidal method
Baseline-corrected Sapropterin AUC0-t
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Area under the concentration-time curve from time zero until the last observed concentration, as calculated by the linear up/log down variant of the trapezoidal method
Uncorrected Sapropterin Cmax
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Maximum observed concentration
Baseline-corrected Sapropterin Cmax
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Maximum observed concentration
Uncorrected Sapropterin AUC0-inf
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Area under the concentration-time curve from time zero to infinity (extrapolated)
Baseline-corrected Sapropterin AUC0-inf
Time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.
Area under the concentration-time curve from time zero to infinity (extrapolated)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Bioavailability
Not yet recruitingPhase 1
Comparing the Extent to Which Maridebart Cafraglutide (AMG 133) is Made Available in the Body When Administered Using Two Subcutaneous Presentations After Multiple Doses
Obesity, Overweight
Not yet recruitingPhase 1
Pharmacokinetics and Relative Bioavailability of Intranasal vs Intramuscular Olanzapine in Healthy Adult Participants
Healthy Adult Participants
Active, not recruitingPhase 1
A Comparative Study to Investigate the Difference in Blood Levels of Two Different Formulations of GSK5784283
Asthma
Not yet recruitingPhase 1
A Study of Imlunestrant (LY3484356) in Healthy Female Participants
Healthy
Active, not recruitingPhase 4
Tacrolimus C:D Ratio Measured in Renal Transplant Recipients Treated With Once-daily Prolonged-release Drugs
Immunosuppression
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RLF-OD032 and Kuvan in people with Bioavailability, run by APR Applied Pharma Research s.a. at 1 site in Canada.
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 10 inclusion and 19 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 12 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 12 Jun 2025). 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?
Uncorrected Sapropterin AUC0-t (time frame: Time points 1, 0.5, pre-dose (0-hour), and 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 12, 16, and 24 hours post-dose.). The study lists 6 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 12 Jun 2025.