AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
ConditionPhenylketonurias
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-55
SponsorNGGT INC.
About this trial
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.
Eligibility criteria
Qualifiers
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.
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
Subjects intolerant or unresponsive to available medical therapies, such as Kuvan, Playnzip, etc.
Disqualifiers
Subjects with PKU that is not due to PAH mutation
Presence of anti-AAV8 neutralizing antibodies
Alanine aminotransferase (ALT) >1.5×ULN and/or aspartate aminotransferase (AST) >1.5×ULN
Alkaline phosphatase (ALP) >1.5×ULN
Trial design
Treatments tested in this trial
- NGGT002
Treatment groups
12 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov