NCT06089265|CompletedPhase 2
Ketohexokinase Inhibition in Hereditary Fructose Intolerance
KHKi in HFI
Short-term Safety and Efficacy of Ketohexokinase Inhibition in Patients With Hereditary Fructose Intolerance
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KHKi in HFI
Short-term Safety and Efficacy of Ketohexokinase Inhibition in Patients With Hereditary Fructose Intolerance
Sponsor study identifier
interventional
8
1 country
1
Study Start
June 15, 2023
Actual (reported)Primary Completion
November 30, 2023
Actual (reported)Study Completion
November 30, 2023
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
November 30, 2023
Actual (reported)Click on a node to explore related trials.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
May 15, 2023
Actual (reported)June 15, 2023
Actual (reported)October 18, 2023
Actual (reported)November 30, 2023
Actual (reported)November 30, 2023
Actual (reported)Intestinal Fructose tolerance,
a visual analog scale from 1-10 for abdominal pain will be used. Additional every 5 minutes the participant will be asked if he/she is nauseous, and more, less or similar nauseous as 5 minutes before.
9 days
Intestinal Fructose tolerance,
Every 5 minutes the participant will be asked if he/she is nauseous, and more, less or similar nauseous as 5 minutes before.
9 days
Renal Fructose tolerance
Urinary pH
9 days
Renal Fructose tolerance
Glucose content, mmol/L
9 days
Renal Fructose tolerance
Phosphate content mmol/L
9 days
Renal Fructose tolerance
Amino acid content mmol/L
9 days
Hepatic fructose tolerance
Serum glucose levels, mmol/L
9 days
Hepatic fructose tolerance
Serum phosphate levels, mmol/L
9 days
Intrahepatic lipid content
9 days
Blood pressure
9 days
Glycosylated transferrin
9 days
HFI participants will receive PF-06835919 for 9 days. Dosage; once daily 300 mg PF-06835919 in the form of 3 tablets, oral.
Healthy controls will receive no intervention, but a single fructose tolerance test.
Maastricht University Medical centre
Maastricht, Limburg, 6202AZ, Netherlands
Patrick Schrauwen, PhD
Maastricht University
8 participants (actual enrollment reported by the registry).
First Submitted
May 15, 2023
First Posted
October 18, 2023
Study Start
June 15, 2023
Actual (reported)Contact the study team to confirm eligibility.
Primary Completion
November 30, 2023
Actual (reported)Study Completion
November 30, 2023
Actual (reported)Last Updated
January 24, 2024
Record last verified: 2024-01
Data can be obtained with the PI on request
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Source: ClinicalTrials.gov.
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