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Ketohexokinase Inhibition in Hereditary Fructose Intolerance

NCT06089265 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2024-01-24

No results posted yet for this study

Summary

Hereditary fructose intolerance (HFI) is a rare inborn error of metabolism. Patients with HFI develop acute abdominal pain, nausea, vomiting, hypoglycemia and proximal tubular dysfunction upon consumption of a fructose containing food product. In rare cases, (prolonged) fructose consumption can even lead to liver and kidney failure. Patients with HFI are therefore treated with a lifelong fructose-restricted diet. Animal studies have shown that the clinical manifestations of HFI are abrogated upon inhibition of ketohexokinase (KHK), the enzyme that catalyses the first step in fructose metabolism.

Recently, PF-06835919, a KHK inhibitor (KHKi), was developed as a new treatment for non-alcoholic fatty liver disease. The compound was well tolerated in several phase II clinical trials.

It is hypothesized that PF-06835919 is also effective in patients with HFI.

Conditions

  • HFI

Interventions

DRUG

PF-06801591

Active ketohexokinase inhibitor

Sponsors & Collaborators

  • Pfizer

    collaborator INDUSTRY

  • Maastricht University Medical Center

    lead OTHER

Principal Investigators

  • Patrick Schrauwen, PhD · Maastricht University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2023-06-15
Primary Completion
2023-11-30
Completion
2023-11-30

Countries

  • Netherlands

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06089265 on ClinicalTrials.gov