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NCT Number: NCT06910943

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

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.

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Key information

Conditions

Choline Deficiency Avitaminosis Deficiency Diseases Digestive System Diseases Gastrointestinal Diseases Hyperphagia Intestinal Diseases Intestinal Failure Liver Injury Malnutrition Nutrition Disorders Nutritional and Metabolic Diseases Pathological Conditions, Signs and Symptoms Signs and Symptoms Signs and Symptoms, Digestive Vitamin B Deficiency

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

University Hospitals Leuven, Campus Gasthuisberg, Leuven, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Choline Chloride for Injection

Drug

Intravenous use

Placebo

Drug

Intravenous use

Primary outcomes

  1. 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

    Cmax = maximum concentration

  2. 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

    Tmax = time of maximum concentration

  3. 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

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

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

    Time frame: Week 1 to Week 8

  5. 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

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

  6. 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

    Tmax = time of maximum concentration

  7. 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

  8. 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

  9. 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

  10. 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

Secondary outcomes

  1. 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

    Time frame: Week 1 to Week 8

    ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase

  2. 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

    Time frame: Week 1 to Week 8

    QTc = QT corrected for heart rate

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

    Time frame: Week 1 to Week 8

    Cmax = maximum concentration

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

    Time frame: Week 1 to Week 8

    Cmax = maximum concentration

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

    Time frame: Week 1 to Week 8

    BMI = body mass index Weight and height will be combined to report BMI in kg/m^2

  6. Open-Label Dose-Selection Phase: Percentage of participants with no worsening of steatosis from Baseline to Week 8 as measured by MRI-PDFF

    Time frame: Week 1 to Week 8

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  7. Open-Label Dose-Selection Phase: Percentage of participants with any improvement of steatosis from Baseline to Week 8 as measured by MRI-PDFF

    Time frame: Week 1 to Week 8

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  8. Double-Blind, Placebo-Controlled Phase: Change from Baseline and Week 8 in peak plasma free choline concentrations (Cmax) at Week 24 in participants receiving Choline Chloride for Injection versus Placebo

    Time frame: Week 1 to Week 24

    Tmax = time of maximum concentration

  9. Double-Blind, Placebo-Controlled Phase: Percentage of participants achieving plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8

    Time frame: Week 1 to Week 8

    Cmax = maximum concentration

  10. Double-Blind, Placebo-Controlled Phase: Percentage of participants maintaining plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8 and Week 24 (ie, through Week 24)

    Time frame: Week 1 to Week 24

    Cmax = maximum concentration

  11. Double-Blind, Placebo-Controlled Phase: Change from Baseline to Week 8 and Week 24 in height, weight and BMI

    Time frame: Week 1 to Week 24

    BMI = body mass index Weight and height will be combined to report BMI in kg/m^2

  12. Double-Blind, Placebo-Controlled Phase: Change from Baseline to Week 8 and Week 24 in ALP, AST, ALT, GGT, VLDL, total bilirubin, direct bilirubin levels, CPK, homocysteine and albumin levels

    Time frame: Week 1 to Week 24

    ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase

  13. Double-Blind, Placebo-Controlled Phase: Percentage of participants with no worsening of steatosis from Baseline to Week 24 as measured by MRI-PDFF

    Time frame: Week 1 to Week 24

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  14. Double-Blind, Placebo-Controlled Phase: Percentage of participants with any improvement of steatosis from Baseline on MRI-PDFF at Week 24

    Time frame: Week 1 to Week 24

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  15. Double-Blind, Placebo-Controlled Phase: Percentage of participants with no worsening in fibrosis grade from Baseline to Week 24, as measured by MRE and ELF test

    Time frame: Week 1 to Week 24

    MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis

  16. Double-Blind, Placebo-Controlled Phase: Percentage of participants with improvement in fibrosis grade from Baseline to Week 24, as measured by MRE and ELF test

    Time frame: Week 1 to Week 24

    MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis

  17. Double-Blind, Placebo-Controlled Phase: Percentage of participants with improvement of steatosis from Baseline on MRI-PDFF with improvement of ALP from Baseline to Week 24

    Time frame: Week 1 to Week 24

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction, ALP = alkaline phosphatase

  18. Double-Blind, Placebo-Controlled Phase: Percentage of participants with improvement of steatosis from Baseline on MRI-PDFF with improvement of ALT or AST from Baseline to Week 24

    Time frame: Week 1 to Week 24

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction, ALT = alanine transaminase, AST = aspartate aminotransferase

  19. Double-Blind, Placebo-Controlled Phase: Assessment of Quality of Life based on CLDQ at Baseline and Week 24

    Time frame: Week 1 to Week 24

    CLDQ = chronic liver disease questionnaire

  20. Double-Blind, Placebo-Controlled Phase: Assessment of Quality of Life based on PGIS at Baseline and PGIC at Week 24

    Time frame: Week 1 to Week 24

    PGIC = patient global impression of change

  21. Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase: Change from Baseline (Week 1) to Week 64 in ALP, AST, ALT, GGT, VLDL, total bilirubin, direct bilirubin levels, CPK, homocysteine and albumin levels

    Time frame: Week 1 to Week 64

    ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase

  22. Open-Label Extension Phase: Double-Blind, Placebo-Controlled Phase: Change from W1 to W64 in Choline Chloride for Injection group, and change from W24 to W64 in Placebo group in ALP, AST, ALT, GGT, VLDL, TBIL, DBil levels, CPK, homocysteine and albumin

    Time frame: Week 1 to Week 64

    ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase, TBIL = total bilirubin, DBil = direct bilirubin

  23. Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase Change from Baseline (Week 1) to Week 64 in height, weight and BMI

    Time frame: Week 1 to Week 64

    BMI = body mass index Weight and height will be combined to report BMI in kg/m^2

  24. Open-Label Extension Phase: Participants from Double-Blind, Placebo-Controlled Phase: Change from W1 to W64 for participants in Choline Chloride for Injection group, and change from W24 to W64 for participants in Placebo group in height, weight and BMI

    Time frame: Week 1 to Week 64

    BMI = body mass index Weight and height will be combined to report BMI in kg/m^2

  25. Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase: Percentage of participants with no worsening of steatosis as measured by MRI-PDFF from Baseline (Week 1) to Week 64

    Time frame: Week 1 to Week 64

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  26. Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase: Percentage of participants with improvement of steatosis as measured by MRI-PDFF from Baseline (Week 1) to Week 64

    Time frame: Week 1 to Week 64

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  27. Open-Label Extension Phase: Participants from Double-Blind, Placebo-Controlled Phase: % of participants with no worsening of steatosis measured by MRI-PDFF from W1 to W64 in Choline Chloride for Injection group, and from W24 to W64 in Placebo group

    Time frame: Week 1 to Week 64

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  28. Open-Label Extension Phase: Participants from Double-Blind, Placebo-Controlled Phase: % of participants with improvement of steatosis measured by MRI-PDFF from W1 to W64 in Choline Chloride for Injection group, and from W24 to W64 in Placebo group

    Time frame: Week 1 to Week 64

    MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction

  29. Open-Label Extension Phase: Double-Blind, Placebo-Controlled Phase: % of participants with no worsening in fibrosis grade measured by MRE and ELF test from W1 to W64 in Choline Chloride for Injection group, and from W24 to W64 in Placebo group

    Time frame: Week 1 to Week 64

    MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis

  30. Open-Label Extension Phase: Participants from Double-Blind, Placebo-Controlled Phase: % of participants with improvement in fibrosis measured by MRE and ELF test from W1 to W64 in Choline Chloride for Injection group, and W24 to W64 in Placebo group

    Time frame: Week 1 to Week 64

    MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis

  31. Open-Label Extension Phase: Participants from Double-Blind Placebo-Controlled Phase: Assessment of QOL based on CLDQ at Week 64

    Time frame: Week 64

    QOL = quality of life, CLDQ = chronic liver disease questionnaire

  32. Open-Label Extension Phase: Participants from Double-Blind Placebo-Controlled Phase: Assessment of QOL based on PGIC at Week 64

    Time frame: Week 64

    QOL = quality of life, PGIC = patient global impression of change

Study contacts

Contact information is provided by the study sponsor or research team.

Chief Scientific Operations Officer

CONTACT

[email protected]

16468440337

Sponsors and collaborators

Lead sponsor

Protara Therapeutics

Industry

Registry information

Official study title

A Phase 2b/3 Randomized Open-Label Dose-Selection Study With Open-Label Extension and Randomized Double-Blind, Placebo-Controlled Study With Open-Label Extension to Evaluate the Safety and Efficacy of Choline Chloride for Injection (Low Dose and High Dose) Versus Placebo in Adolescents and Adults With Intestinal Failure Receiving Long-Term Parenteral Support

Acronym: THRIVE-3

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Apr 4, 2025
Registry last updated
Jun 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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