Short-term safety and efficacy of ketohexokinase inhibition in patients with hereditary fructose intolerance

Phase 2

Recruiting

Conditions: hereditary fructose intolerance
fructosemia
10021605

Registration Number: NL-OMON53368

Lead Sponsor: Medisch Universitair Ziekenhuis Maastricht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 8

Inclusion Criteria

• Participants are able to provide signed and dated written informed consent
prior to any study specific procedures
• Use of effective contraception (only applicable to premenopausal women)
• Aged >= 18 years

Exclusion Criteria

• Type 2 diabetes
• Patients with congestive heart failure and/or severe renal and/or liver
insufficiency
• Uncontrolled hypertension
• Previous enrolment in a clinical study with an investigational product during
the last 3 months or as judged by the investigator which would possibly hamper
our study results
• Use of drugs that inhibit organic anion transporting polypeptide (OATP)
transporters (e.g. rifampicin, gemfibrozil, cyclosporine, erythromcyin and
clarithromycin)*
• Subjects who do not want to be informed about unexpected medical findings

* Exclusion criterion for HFI patients only.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>- Intrahepatic lipid content assessed by proton magnetic resonance spectroscopy<br /><br>(at baseline and completion in HFI patients only),<br /><br>- Intestinal fructose tolerance, i.e. onset of abdominal pain and/or nausea<br /><br>after oral fructose challenge (in comparison to oral glucose challenge)<br /><br>- Hepatic fructose tolerance, i.e. serum glucose and phosphate after oral<br /><br>fructose challenge (in comparison to oral fructose challenge in healthy<br /><br>individuals)<br /><br>- Renal fructose tolerance, i.e. urinary glucose, phosphate, pH and amino acids<br /><br>after oral fructose challenge (in comparison to oral fructose challenge in<br /><br>healthy individuals). </p><br>

Secondary Outcome Measures