Choline DeficiencyPhase 2/3THRIVE-3NCT06910943
Study of Choline Chloride for Injection in Adolescent and Adult Patients With Intestinal Failure Receiving Long Term Parenteral Support
A Phase 2/3 study testing Choline Chloride for Injection in people with Choline Deficiency and Liver Injury, run by Protara Therapeutics at 18 sites in 6 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Choline Deficiency, Liver Injury
- What is being tested?
- Choline Chloride for Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 129 participants
- Where
- 18 sitesin 6 countries
- Study dates
- 10 Dec 2025to Jan 2029
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
18 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 6 countries
United States8 sites, 8 recruiting
- Recruiting
University of Colorado School of Medicine
Aurora, Colorado, 80045
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- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
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- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44195
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- Recruiting
Duke Clinic - Abdominal Transplant Research Office
Durham, North Carolina, 27710
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- Recruiting
Floridian Clinical Research
Miami Lakes, Florida, 33016
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- Not yet recruiting
Columbia University Medical Center/ New York Presbyterian Hospital
New York, New York, 10032
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- Not yet recruiting
Nebraska Medicine
Omaha, Nebraska, 68105
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- Recruiting
Pinnacle Clinical Research- San Antonio
San Antonio, Texas, 78229
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France3 sites, 2 recruiting
- Recruiting
Beaujon Hospital - APHP
Clichy, 92110
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- Withdrawn
Rennes University Hospital Center - Pontchaillou Site
Rennes, 35000
- Not yet recruiting
CHRU Nancy - Barbois Hospital
Vandœuvre-lès-Nancy, 54511
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Denmark2 sites, 2 recruiting
- Recruiting
Aalborg University Hospital, Department of Medical Gastroenterology
Aalborg, 9100
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- Recruiting
Rigshospitalet - University Hospital Copenhagen
Copenhagen, 2100
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Germany2 sites, 2 recruiting
- Recruiting
Charite - University Hospital Berlin
Berlin, 13353
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- Recruiting
University Duisburg-Essen, University Hospital Essen
Essen, 45147
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Poland2 sites, 2 recruiting
- Recruiting
M. Pirogow Provincial Specialized Hospital in Lodz, Nutritional Treatment Center
Lodz, 90-532
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- Recruiting
Czerniakowski Hospital Sp. z o.o. (LCC)
Warsaw, 00-739
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Belgium1 site, 1 recruiting
- Recruiting
University Hospitals Leuven, Campus Gasthuisberg
Leuven, 3000
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About this study
Official 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
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.
Read more
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
- Being tested
- Choline Chloride for Injection, Placebo
- Keywords
- Intestinal Failure, Parenteral Nutrition, Parenteral Support, Choline Chloride, choline deficiency
- Medical subject headings
- Choline Deficiency, Intestinal Failure, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Choline Chloride for InjectionDrug
Intravenous use
PlaceboDrug
Intravenous use
| Study group | Role | What they receive |
|---|---|---|
| Open-Label, Dose-Selection: Dose 1 | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Open-Label, Dose-Selection: Dose 2 | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Open-Label, Dose-Selection: Dose 3 | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Double-Blind, Placebo-Controlled: High Dose | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Double-Blind, Placebo-Controlled: Low Dose | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Double-Blind, Placebo-Controlled: Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| Open Label Extension: High Dose | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
| Open Label Extension: Low Dose | Experimental | Not described Receives: Drug: Choline Chloride for Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Open-Label Dose-Selection Phase: PK of plasma free choline (Cmax) during Week 1 and Week 8 Visits
Cmax = maximum concentration
Measured: 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
Measured: 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
Measured: Week 1 to Week 8
Open-Label Dose-Selection Phase: Change from Baseline in plasma free choline concentrations at Week 8
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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
Measured: 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
Measured: 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
ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase
Measured: 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
QTc = QT corrected for heart rate
Measured: 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
Cmax = maximum concentration
Measured: 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
Cmax = maximum concentration
Measured: Week 1 to Week 8
Open-Label Dose-Selection Phase: Change from Baseline to Week 8 in height, weight and BMI
BMI = body mass index Weight and height will be combined to report BMI in kg/m^2
Measured: Week 1 to Week 8
Open-Label Dose-Selection Phase: Percentage of participants with no worsening of steatosis from Baseline to Week 8 as measured by MRI-PDFF
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 8
Open-Label Dose-Selection Phase: Percentage of participants with any improvement of steatosis from Baseline to Week 8 as measured by MRI-PDFF
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 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
Tmax = time of maximum concentration
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Percentage of participants achieving plasma free choline concentration Cmax ≥ 9.5 nmol/mL at Week 8
Cmax = maximum concentration
Measured: Week 1 to Week 8
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)
Cmax = maximum concentration
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Change from Baseline to Week 8 and Week 24 in height, weight and BMI
BMI = body mass index Weight and height will be combined to report BMI in kg/m^2
Measured: Week 1 to Week 24
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
ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Percentage of participants with no worsening of steatosis from Baseline to Week 24 as measured by MRI-PDFF
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Percentage of participants with any improvement of steatosis from Baseline on MRI-PDFF at Week 24
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 24
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
MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis
Measured: Week 1 to Week 24
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
MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis
Measured: Week 1 to Week 24
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction, ALP = alkaline phosphatase
Measured: Week 1 to Week 24
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction, ALT = alanine transaminase, AST = aspartate aminotransferase
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Assessment of Quality of Life based on CLDQ at Baseline and Week 24
CLDQ = chronic liver disease questionnaire
Measured: Week 1 to Week 24
Double-Blind, Placebo-Controlled Phase: Assessment of Quality of Life based on PGIS at Baseline and PGIC at Week 24
PGIC = patient global impression of change
Measured: Week 1 to Week 24
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
ALP = alkaline phosphatase, AST = aspartate aminotransferase, ALT = alanine transaminase, GGT = gamma-glutamyl transpeptidase, VLDL = very low-density lipoprotein, CPK = creatine phosphokinase
Measured: Week 1 to Week 64
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
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
Measured: Week 1 to Week 64
Open-Label Extension Phase: Participants from Open-Label Dose-Selection Phase Change from Baseline (Week 1) to Week 64 in height, weight and BMI
BMI = body mass index Weight and height will be combined to report BMI in kg/m^2
Measured: Week 1 to Week 64
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
BMI = body mass index Weight and height will be combined to report BMI in kg/m^2
Measured: Week 1 to Week 64
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 64
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 64
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 64
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
MRI-PDFF = magnetic resonance imaging-estimated proton density fat fraction
Measured: Week 1 to Week 64
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
MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis
Measured: Week 1 to Week 64
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
MRE = magnetic resonance elastography, ELF = enhanced liver fibrosis
Measured: Week 1 to Week 64
Open-Label Extension Phase: Participants from Double-Blind Placebo-Controlled Phase: Assessment of QOL based on CLDQ at Week 64
QOL = quality of life, CLDQ = chronic liver disease questionnaire
Measured: Week 64
Open-Label Extension Phase: Participants from Double-Blind Placebo-Controlled Phase: Assessment of QOL based on PGIC at Week 64
QOL = quality of life, PGIC = patient global impression of change
Measured: Week 64
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2025
- Study start10 Dec 2025
- Primary completionMar 2028 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Choline DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 129 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Protara Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Scientific Operations Officer, Study director, Protara Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06910943
- Sponsor's protocol ID
- TARA-001-301
- Acronym
- THRIVE-3
- Status last verified
- Oct 2025
- First submitted
- 18 Mar 2025
- Last updated
- 24 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No