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Virus Therapy

Gene Therapy for Phenylketonuria

Phase 1 & 2

Recruiting

Research Sponsored by NGGT INC.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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)

Adults aged 18-55 at the time of informed consent

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 52 and during year 1 to 5

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment called NGGT002 for adults with classic Phenylketonuria (PKU). NGGT002 is a gene therapy that carries a functional copy of a specific gene

Who is the study for?

Adults aged 18-55 with classic Phenylketonuria (PKU), severe PAH deficiency, and specific genetic mutations. Participants must have had high phenylalanine levels despite a restricted diet and not be well-controlled on existing PKU medications like Kuvan or Palynziq. They should agree to follow dietary guidelines and use effective contraception.

What is being tested?

The trial is testing NGGT002, a gene therapy using an rAAV8 vector to deliver a functional human PAH gene to adults with classic PKU. It's designed to see if this one-time treatment can safely improve the body's ability to process phenylalanine over five years.

What are the potential side effects?

As it’s an early-phase trial for NGGT002, potential side effects are being studied; they may include immune reactions to the viral vector, liver enzyme changes, injection site reactions, and other unforeseen issues related to gene therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have classic PKU with confirmed PAH mutations and no enzyme activity.

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I am between 18 and 55 years old.

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I stopped taking medications like Kuvan or Palynziq for my condition over 28 days ago.

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I have undergone a vasectomy or permanent sterilization.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 52 and during year 1 to 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 52 and during year 1 to 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 52 and during year 1 to 5 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in 12-lead electrocardiograms (ECGs), vital signsand physical examinations

Change from baseline in Plasma Phe Concentration

Change from baseline in clinical laboratory values

+1 more

Secondary study objectives

Change from baseline in total protein intake at at Week 28, Week 52 and during Year 1 to 5 post dose

Change in Phenylketonuria Quality of Life Questionnaire (PKU-QOL)

Incidence of sustained plasma Phe concentration of ≤360 μmol/L (6 mg/dL) at Week 12, Week 28, Week 52 and during Year 1 to 5 post dose

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: NGGT002Experimental Treatment1 Intervention

Low dose and high dose group: Six to twelve patients will be enrolled into two cohorts at two dose levels. The safety of this study can be ensured by selecting the highest dose under the No Observed Adverse Effect Level (NOAEL) doses observed in preclinical toxicology studies.

0 / 4Eligibility criteria met