Indianapolis, INNCT06302348Now EnrollingIRB Ready

Phenylketonuria Clinical Trial in Indianapolis, IN

Access cutting-edge phenylketonuria treatment through this clinical trial at a research site in Indianapolis. Study-provided care at no cost to qualified participants.

Sponsored by PTC Therapeutics

Quick Self-Assessment

See if you qualify for this Indianapolis location

Preparing your pre-screening questions…

Expert Care in Indianapolis

Access phenylketonuria specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related phenylketonuria treatment provided free

Apply for This Indianapolis Location

Check if you qualify for this phenylketonuria clinical trial in Indianapolis, IN

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Indianapolis

    Convenient for IN residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Indianapolis site if eligible
  4. 4Begin participation

About This Phenylketonuria Study in Indianapolis

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.

Sponsor: PTC Therapeutics

Who Can Participate

Inclusion Criteria

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 90 days after the last dose of the study drug.
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

Exclusion Criteria

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 participant's ability to participate in the study or increase the risk of participation for that participant.
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, quinoid dihydropteridine reductase, or pterin 4-alphacarbinolamine dehydratase genes.
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) \<60 milliliters (mL)/minute (min)/1.73 square meter (m\^2).
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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Indianapolis?

Yes, this clinical trial (NCT06302348) has an active research site in Indianapolis, IN that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Phenylketonuria Treatment Options in Indianapolis, IN

If you're searching for phenylketonuria treatment options in Indianapolis, IN, this clinical trial (NCT06302348) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Indianapolis research site is actively enrolling participants for this clinical trial. You'll receive care from experienced phenylketonuria specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all phenylketonuria clinical trials near you to find additional studies recruiting in your area.

More Essential Tremor Trials in Indianapolis, IN

See all essential tremor clinical trials recruiting in Indianapolis — not just this study.

Browse Essential Tremor Trials in Indianapolis →

Browse More Trials by Condition

Essential Tremor TrialsPulmonary Hypertension TrialsPKU TrialsAll Conditions →

Ready to Join in Indianapolis?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · Indianapolis, IN