Is NCT06332807 recruiting?

Yes, NCT06332807 is currently recruiting. This pku study is enrolling participants at 5 locations.

What is the status of NCT06332807?

NCT06332807 is currently recruiting. This is a Clinical Trial trial for pku sponsored by NGGT INC.. Status verified Dec 2, 2025 via ClinicalTrials.gov.

Where is NCT06332807 enrolling participants?

NCT06332807 has 5 active sites including Orange, California; Minneapolis, Minnesota; Morristown, New Jersey and 2 more locations.

Who is eligible for NCT06332807?

This pku trial is targeting 12 participants. Detailed eligibility criteria including age requirements and medical history are listed on this page.

Who can participate in this PKU study?

Eligibility varies by study, but generally includes adults diagnosed with PKU who meet specific medical criteria. Complete the interest form above to learn if you may qualify.

Is the study available in Minneapolis, Minnesota?

Yes! This study has active research sites in Minneapolis, Morristown, Pittsburgh. When you express interest, you'll be connected with the site nearest to you.

What are the benefits of joining a clinical trial for PKU?

Participants often receive: access to new treatments before they're widely available, close monitoring by medical professionals, study-related care at no cost, and the opportunity to contribute to medical research.

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NCT06332807 RECRUITINGIRB Ready

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

Express Your Interest Today

Interested in learning more? Complete the form below.

Why Participate?

No-Cost Study Care
Advance Medical Science
Contribute to groundbreaking research
Cutting-Edge Treatment
Access to innovative therapies
Expert Medical Care
Close monitoring by specialists
Possible Compensation*
For time and travel (varies by study)
Help Others
Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

1Submit this form
2Phone screening
3In-person assessment if eligible
4Begin participation

About This Study

This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene. Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.

Who Can Participate

Inclusion Criteria

Is willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; a legally authorized representative may provide written consent and assent may be requested. 2. Male and female subjects with diagnosis of classic PKU, a condition characterized by severe PAH deficiency with confirmed PAH mutations predicted with no residual enzyme activity. A list of PAH mutations for classic PKU based on in vitro PAH activity (Himmelreich et al., 2018) and the genotype-phenotype correlation (Garbade et al., 2019) can be found in BIOPKU genotypes database (http://www.biopku.org/pah). 3. Adults aged 18-55 at the time of informed consent 4. Subjects intolerant or unresponsive to available medical therapies, such as Kuvan, Playnzip, etc. 5. Subjects who have been on medications, such as Kuvan, Palynziq, etc but have come off for medical reasons or the patient's decision at least 28 days prio

❓ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

📋

Study Type

INTERVENTIONAL

👥

Target Enrollment

12 participants

🎂

Age Range

18 Years - 55 Years

⚧

Gender

ALL

🏢

Sponsor

Texas

Location available

View Dallas location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

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

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

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

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

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Find More PKU Trials by City

Browse all pku clinical trials in these cities — not just this study.

Minneapolis, Minnesota Morristown, New Jersey Pittsburgh, Pennsylvania Dallas, Texas

Browse More Trials by Condition

PKU Trials All Conditions →

Looking for PKU Treatment in Minneapolis?

Join others in Minnesota exploring innovative treatment options through clinical research

PKU Treatment Options in Minneapolis, Minnesota

If you're searching for PKU treatment in Minneapolis, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Minneapolis, Morristown, Pittsburgh and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with PKU. All study-related care is provided at no cost to participants.

Local Sites

3 locations in Minnesota

Now Enrolling

Up to 12 participants

Quick Start

Screening available now

Why Consider a Clinical Trial for PKU?

Potential Benefits

✓Access to new treatment approaches before public availability
✓Close monitoring by experienced medical professionals
✓Study-related care provided at no cost
✓Contribute to medical research for PKU

What to Expect

→Initial screening to determine eligibility
→Regular check-ups and monitoring visits
→Possible compensation for time and travel
→You can withdraw at any time

Frequently Asked Questions About This PKU Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06332807. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.