Study of Choline Chloride for Injection in Adolescent and Adult Patients With Intestinal Failure Receiving Long Term Parenteral Support (THRIVE-3)

Protara Therapeutics

Status and phase: Not yet enrolling; Phase 3; Phase 2

Conditions: Liver Injury; Choline Deficiency

Treatments: Drug: Placebo; Drug: Choline Chloride for Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT06910943

TARA-001-301

Details and patient eligibility

About

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.

Enrollment: 144 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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144 participants in 8 patient groups, including a placebo group

Open-Label, Dose-Selection: Dose 1

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Open-Label, Dose-Selection: Dose 2

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Open-Label, Dose-Selection: Dose 3

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: High Dose

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: Low Dose

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Double-Blind, Placebo-Controlled: Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

Open Label Extension: High Dose

Experimental group

Treatment:

Drug: Choline Chloride for Injection

Open Label Extension: Low Dose

Experimental group

Treatment:

Drug: Choline Chloride for Injection