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NCT06910943PHASE2, PHASE3Recruiting

Study of Choline Chloride for Injection in Adolescent and Adult Patients With Intestinal Failure Receiving Long Term Parenteral Support

Protara Therapeutics

Start Date

12/10/2025

Completion Date

6/1/2028

Summary

TARA-001-301 is a Phase 2b/3 randomized Open-Label Dose-Selection study with an Open-Label Extension and randomized Double-Blind, Placebo-Controlled Study with Open-Label Extension to investigate the safety and efficacy of Choline Chloride for Injection (Low Dose and High Dose) versus Placebo in adolescents (ages 12 to \< 18 years of age) and adults (≥ 18 years of age) with intestinal failure receiving long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated. Participants will be enrolled in one of 2 parts, each part will be followed by an open-label extension period of approximately a year. Part 1: Open-Label Dose-Selection Phase Part 2: Double-Blind, Placebo-Controlled Phase The purpose of the Open-Label Dose-Selection Phase is to evaluate the safety, tolerability, how Choline Chloride for Injection (study drug) is distributed in the body, and to select 2 of 3 doses for testing in the Double-Blind, Placebo-Controlled Phase. The purpose of the Double-Blind, Placebo-Controlled Phase is to assess the safety of the study drug and how well the study drug works at the 2 selected dose levels.

Eligibility Criteria

Age Range: 12 years to No maximum

Key Inclusion Criteria: * Male or female 12 years of age or older at the time of signing the informed consent * Individuals who have voluntarily given written informed consent after the nature of the study has been explained according to applicable requirements, prior to study entry * Individuals with intestinal failure receiving long-term PS when oral or enteral nutrition is not possible, insufficient, or contraindicated who are receiving stable PS at time of screening and for the duration of the study; Note: Long-Term PS = Participant must have been receiving PS for at least 6 months prior to screening and requiring PS at least 3 times per week * Females of childbearing potential must have a negative urine pregnancy test at screening Key Exclusion Criteria: * Patients taking steatogenic medications for ≥ 12 weeks in the past 12 months; those taking any medicine that could affect the measurement of hepatic steatosis within 12 weeks prior to study entry * Evidence of systemic active infection at the time of dosing * Participants intending to take non-study drug choline supplements or choline-containing multivitamins during the course of the study * Participants unwilling to limit alcohol intake to no more than 20/g a day for 24 hours prior to their screening visit and for the duration of the study * Active malignancy (excluding basal cell skin tumor, low or very low risk prostate cancer, cervical carcinoma in situ and local resected cervical cancer) * Clinically significant renal disease * Low B12 or low serum folic acid levels that are less than the normal range * Fulminant liver failure, with active bleeding and/or encephalopathy

Interventions

DRUG

Choline Chloride for Injection

DRUG

Placebo

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Conditions

Choline DeficiencyLiver Injury

Locations

University of Colorado School of Medicine

Aurora, Colorado 80045

United States

Floridian Clinical Research

Miami Lakes, Florida 33016

United States

Nebraska Medicine

Omaha, Nebraska 68105

United States

Columbia University Medical Center/ New York Presbyterian Hospital

New York, New York 10032

United States

Duke Clinic - Abdominal Transplant Research Office

Durham, North Carolina 27710

United States

Cleveland Clinic

Cleveland, Ohio 44195

United States

Pinnacle Clinical Research- San Antonio

San Antonio, Texas 78229

United States

University Hospitals Leuven, Campus Gasthuisberg

Leuven, 3000

Belgium

Aalborg University Hospital, Department of Medical Gastroenterology

Aalborg, 9100

Denmark

Rigshospitalet - University Hospital Copenhagen

Copenhagen, 2100

Denmark

Beaujon Hospital - APHP

Clichy, 92110

France

Rennes University Hospital Center - Pontchaillou Site

Rennes, 35000

France

CHRU Nancy - Barbois Hospital

Vandœuvre-lès-Nancy, 54511

France

Charite - University Hospital Berlin

Berlin, 13353

Germany

University Duisburg-Essen, University Hospital Essen

Essen, 45147

Germany

M. Pirogow Provincial Specialized Hospital in Lodz, Nutritional Treatment Center

Lodz, 90-532

Poland

Czerniakowski Hospital Sp. z o.o. (LCC)

Warsaw, 00-739

Poland