Liver drug study for rare disease pulled before it started
NCT ID NCT07123363
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study aimed to see if the drug bitopertin is safe for people with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who also have early signs of liver disease. Researchers planned to check for side effects and measure changes in protoporphyrin levels in blood, bile, liver, and stool. However, the study was withdrawn before any participants were enrolled.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Expected to start
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Jan 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2035
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Diagnosis of EEP or XLP that has been biochemically and genetically verified prior diagnosis of EPP or XLP * Age 18-75 years * Increased liver stiffness at baseline, defined as stiffness measurement \[E value\] by: Fibroscan \>7 kPa; MRE that is \>3.0 kPa; Velacur/Sonicincyte that is \>6.0 kPa; and/or elevated liver enzymes: serum ALT \> 2 X upper limit of normal (ULN), but not greater than 5 X ULN and/or serum AP \> 2 X ULN, but not greater than 5 X ULN * INR \< 1.4 * Compensated liver disease at baseline, defined as lack of clinically evident ascites, encephalopathy, hepatocellular carcinoma, clinically evident icterus or jaundice, peripheral edema. * Willingness and ability to volunteer and provide informed consent for a long-term study that will include liver biopsies and collection of bile from the second portion of the duodenum, at baseline and at the end of study. In addition, follow-up visits will be planned every 26 weeks throughout the study, with plans for repeat routine/safety labs at each visit and for measures of liver stiffness and markers of hepatic status and fibrosis (Fib-4, APRI, ELF, Fibrotest) performed once every 6 months. Exclusion Criteria: * Chronic hepatitis B, C, D, or E; * Human immunodeficiency (HIV) infection; * Alcohol use \> 14 units/week for men or \> 7 units/week for women; * Pregnancy or breast feeding among women; * Any known active malignancy other than small and localized squamous or basal cell carcinoma of the skin; * Advanced or decompensated heart, lung, kidney, liver, or neuro-psychiatric disease; * History of diagnosed depression or suicidality; * History of diagnosed substance abuse or poor impulse control; * Any other conditions that, in the opinion of the Investigator, renders the individual unfit to participate
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.