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New treatment option for Phenylketonuria
Official title A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
ClinicalTrials.gov ID: NCT06302348
What this study is testing
What is Sepiapterin?
Sepiapterin is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for phenylketonuria.
Also referred to as PTC923, Sephience.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 9
You may be able to join if
- Key For all participants:
- Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective...
- Willing to maintain prescribed daily protein/Phe during Screening and Part 1. For participants ≥1 month of age at Screening:
- Established diagnosis of PKU with hyperphenylalaninemia (HPA) evidenced by at least 2 blood Phe measurement ≥600 micromoles (μmol)/liter (L) as...
- A minimum of 1 documented blood Phe measurement \<480 μmol/L within 1 month prior to Screening.
You likely can't join if
- History of allergies or adverse reactions to any of the ingredients or excipients of synthetic tetrahydrobiopterin (BH4) or sepiapterin.
- Serious neuropsychiatric illness (for example, major depression) not currently under medical control or other concurrent disease or condition that...
- Treatment with BH4 supplementation (sapropterin, KUVAN®) within 3 months prior to Screening.
- Current participation in another investigational drug study or use of any investigational agent within 30 days prior to Screening.
- Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive...
- Any clinically significant laboratory abnormality as determined by the investigator.
See the full eligibility criteria
- Key For all participants:
- Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least...
- Willing to maintain prescribed daily protein/Phe during Screening and Part 1. For participants ≥1 month of age at Screening:
- Established diagnosis of PKU with hyperphenylalaninemia (HPA) evidenced by at least 2 blood Phe measurement ≥600 micromoles (μmol)/liter (L) as documented in the medical history.
- A minimum of 1 documented blood Phe measurement \<480 μmol/L within 1 month prior to Screening.
- Two screening blood Phe concentration values must be in the range ≥120 to ≤480 μmol/L. For participants \<1 month of age at the time of informed consent/assent only:
- Blood Phe at newborn screening ≥600 μmol/L. For participants ≥30 months to \<10 years of age:
- Baseline FSIQ score ≥80. Key
- History of allergies or adverse reactions to any of the ingredients or excipients of synthetic tetrahydrobiopterin (BH4) or sepiapterin.
- Serious neuropsychiatric illness (for example, major depression) not currently under medical control or other concurrent disease or condition that, in the opinion of the investigator or sponsor, would interfere with the...
- Treatment with BH4 supplementation (sapropterin, KUVAN®) within 3 months prior to Screening.
- Current participation in another investigational drug study or use of any investigational agent within 30 days prior to Screening.
- Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive Guanosine-5'-triphosphate (GTP) cyclohydrolase I, sepiapterin reductase...
- Any clinically significant laboratory abnormality as determined by the investigator.
- Any past medical history of an abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated (Bedside Schwartz Equation) glomerular filtration rate (GFR)...
- Major surgery within 90 days prior to Screening visit.
- Previous treatment for \>6 weeks with sepiapterin (that is, Sephience). Note: Other protocol-defined inclusion and exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- Indianapolis, Indiana, United States
- North Adelaide, Australia
- Parkville, Australia
- Lille, France
- Paris, France
- Tours, France
- Dublin, Ireland
- Szczecin, Poland
- Warsaw, Poland
- Birmingham, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 9 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Indianapolis, Indiana, United States; North Adelaide, Australia; Parkville, Australia; Lille, France; Paris, France; Tours, France and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.