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.
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
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
Parallel
Choline Chloride for Injection
DrugIntravenous use
Placebo
DrugIntravenous use
8
Treatment groupsSee each treatment group below.
Open-Label Dose-Selection Phase: PK of plasma free choline (Cmax) during Week 1 and Week 8 Visits
Cmax = maximum concentration
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
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
Open-Label Dose-Selection Phase: Change from Baseline in plasma free choline concentrations at Week 8
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
ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase
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
QTc = QT corrected for heart rate
Open-Label Dose-Selection Phase: Percentage of participants achieving plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8
Cmax = maximum concentration
Open-Label Dose-Selection Phase: Percentage of participants maintaining plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8
Cmax = maximum concentration
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