Recruiting
Phase 2
Phase 3

Choline Chloride

Sponsor:

Protara Therapeutics

Code:

NCT06910943

Conditions

Choline Deficiency

Liver Injury

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

Choline Chloride for Injection

Placebo

Study Details

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

Study ID

NCT06910943

Start date

Dec 10, 2025

Status verified date

Oct, 2025

Completion date

Jan, 2029

Anticipated

Primary completion date

Mar, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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:

  • Participants taking steatogenic medications or any medicine that could affect the measurement of hepatic steatosis within 12 weeks prior to signing consent
  • 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
  • Participants with severe hepatic impairment, defined as Child-Turcotte-Pugh (CTP) Class C (score ≥10) at screening.

Study Design

Enrollment

129 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Open-Label, Dose-Selection: Dose 1

experimental: Open-Label, Dose-Selection: Dose 2

experimental: Open-Label, Dose-Selection: Dose 3

experimental: Double-Blind, Placebo-Controlled: High Dose

experimental: Double-Blind, Placebo-Controlled: Low Dose

placebo comparator: Double-Blind, Placebo-Controlled: Placebo

experimental: Open Label Extension: High Dose

experimental: Open Label Extension: Low Dose

Interventions

Choline Chloride for Injection

Intravenous use

Placebo

Intravenous use

Primary outcome measure

  • Open-Label Dose-Selection Phase: PK of plasma free choline (Cmax) during Week 1 and Week 8 Visits [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Week 1 to Week 8 ]
  • Open-Label Dose-Selection Phase: Change from Baseline in plasma free choline concentrations at Week 8 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Week 8 to Week 64 ]

Central Contacts and Locations

Central contacts

Chief Scientific Operations Officer

16468440337clinicaltrials@protaratx.com

Locations

University of Colorado School of Medicine

Recruiting

Aurora, Colorado, United States, 80045

Floridian Clinical Research

Recruiting

Miami Lakes, Florida, United States, 33016

Ann & Robert H. Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Duke Clinic - Abdominal Transplant Research Office

Recruiting

Durham, North Carolina, United States, 27710

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

Pinnacle Clinical Research- San Antonio

Recruiting

San Antonio, Texas, United States, 78229

More Information

Sponsor

Protara Therapeutics

Last update posted

Aug 24, 2026

Last verified

Oct, 2025

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Protara Therapeutics on 2026-08-24.