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

NCT06910943 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: TARA-001-301
• Study Type: interventional
• Target: 129
• Locations: 6 countries
• Sites: 14

Brief 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.

Conditions

Choline Deficiency; Liver Injury

Keywords

Intestinal Failure; Parenteral Nutrition; Parenteral Support; Choline Chloride; choline deficiency

Outcome Measures

Primary Outcomes (10)

• Open-Label Dose-Selection Phase: PK of plasma free choline (Cmax) during Week 1 and Week 8 Visits

  Cmax = maximum concentration

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Open-Label Dose-Selection Phase: PK of plasma free choline (Tmax) during Week 1 and Week 8 Visits

  Tmax = time of maximum concentration

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: PK of plasma free choline (AUC(0-TAU)) during Week 1 and Week 8 Visits

  AUC = area under the curve, AUC(0-TAU) = AUC at end of dosing

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Change from Baseline in plasma free choline concentrations at Week 8

  Week 1 to Week 8

• Open-Label Dose-Selection Phase and Double-Blind, Placebo-Controlled Phase, Open-Label Extension Phase: Incidence and severity of TEAEs Incidence of TESAEs

  TEAE = treatment emergent adverse event, TESAE = treatment emergent serious adverse event

  Week 1 to Week 64

• Double-Blind, Placebo-Controlled Phase: Change from Baseline in peak plasma free choline concentrations (Cmax) at Week 8 in participants receiving Choline Chloride for Injection versus Placebo

  Tmax = time of maximum concentration

  Week 1 to Week 8

• Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase: Percentage of participants with plasma free choline concentrations of ≥ 9.5 nmol/mL at Week 64

  Week 64

• Open-Label Extension Phase: Percentage of participants maintaining plasma free choline concentrations of ≥ 9.5 nmol/mL at Week 8 and Week 64 (ie, both timepoints)

  Week 8 to Week 64

• Open-Label Extension Phase: Participants from Double-Blind, Placebo-Controlled Phase: % with plasma free choline concentrations of ≥9.5 nmol/mL at Week 64

  Week 1 to Week 64

• Open-Label Extension Phase: Participants from Double-Blind Placebo-Controlled Phase: % maintaining plasma free choline concentrations of ≥ 9.5 nmol/mL at Week 8, Week 24 and Week 64 for participants who previously received Choline Chloride for Injection

  Week 8 to Week 64

Secondary Outcomes (32)

• Open-Label Dose-Selection Phase: Change from Baseline to Week 8 in ALP, AST, ALT, GGT, VLDL, total bilirubin, direct bilirubin levels, CPK, homocysteine and albumin levels

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Triplicate QTc measurements collected during Week 1 and Week 8, and changes from pre-infusion QTc at Week 1 to all post-baseline timepoints

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Percentage of participants achieving plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Percentage of participants maintaining plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8

  Week 1 to Week 8

• Open-Label Dose-Selection Phase: Change from Baseline to Week 8 in height, weight and BMI

  Week 1 to Week 8

Study Arms (8)

Open-Label, Dose-Selection: Dose 1

EXPERIMENTAL

Drug: Choline Chloride for Injection

Open-Label, Dose-Selection: Dose 2

EXPERIMENTAL

Drug: Choline Chloride for Injection

Open-Label, Dose-Selection: Dose 3

EXPERIMENTAL

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: High Dose

EXPERIMENTAL

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: Low Dose

EXPERIMENTAL

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: Placebo

PLACEBO COMPARATOR

Drug: Placebo

Open Label Extension: High Dose

EXPERIMENTAL

Drug: Choline Chloride for Injection

Open Label Extension: Low Dose

EXPERIMENTAL

Drug: Choline Chloride for Injection

Interventions

Choline Chloride for Injection DRUG

Intravenous use

Double-Blind, Placebo-Controlled: High Dose; Double-Blind, Placebo-Controlled: Low Dose; Open Label Extension: High Dose; Open Label Extension: Low Dose; Open-Label, Dose-Selection: Dose 1; Open-Label, Dose-Selection: Dose 2; Open-Label, Dose-Selection: Dose 3

Placebo DRUG

Intravenous use

Double-Blind, Placebo-Controlled: Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Protara Therapeutics: lead

Study Sites (14)

University of Colorado School of Medicine

Aurora, Colorado, 80045, United States

RECRUITING

Columbia University Medical Center/ New York Presbyterian Hospital

New York, New York, 10032, United States

NOT YET RECRUITING

Duke Clinic - Abdominal Transplant Research Office

Durham, North Carolina, 27710, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

University Hospitals Leuven, Campus Gasthuisberg

Leuven, 3000, Belgium

NOT YET RECRUITING

Aalborg University Hospital, Department of Medical Gastroenterology

Aalborg, 9100, Denmark

NOT YET RECRUITING

Rigshospitalet - University Hospital Copenhagen

Copenhagen, 2100, Denmark

RECRUITING

Beaujon Hospital - APHP

Clichy, 92110, France

NOT YET RECRUITING

Rennes University Hospital Center - Pontchaillou Site

Rennes, 35000, France

NOT YET RECRUITING

CHRU Nancy - Barbois Hospital

Vandœuvre-lès-Nancy, 54511, France

NOT YET RECRUITING

Charite - University Hospital Berlin

Berlin, 13353, Germany

NOT YET RECRUITING

University Duisburg-Essen, University Hospital Essen

Essen, 45147, Germany

NOT YET RECRUITING

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

Lodz, 90-532, Poland

NOT YET RECRUITING

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

Warsaw, 00-739, Poland

RECRUITING

MeSH Terms

Conditions

Choline Deficiency; Intestinal Failure; Hyperphagia

Interventions

Choline; Injections

Condition Hierarchy (Ancestors)

Vitamin B Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Trimethyl Ammonium Compounds; Quaternary Ammonium Compounds; Onium Compounds; Drug Administration Routes; Drug Therapy; Therapeutics

Study Officials

• Chief Scientific Operations Officer

  Protara Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Chief Scientific Operations Officer

CONTACT

16468440337; clinicaltrials@protaratx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Masking Details: The Open-Label Dose Selection Phase will be open label, and the Double-Blind, Placebo-Controlled Phase will be double blind. The Open Label Extension Phase will be open label.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 18, 2025
• First Posted: April 4, 2025
• Study Start: December 10, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2028
• Last Updated: January 27, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3); DK (2); US (4); BE (1); DE (2); PL (2)