End-stage Liver DiseasePhase 4NCT00656266
Trial of Calcineurin Inhibitor-Sparing Immunosuppression Regimen in Pediatric Liver Transplantation
A Phase 4 study testing mycophenolate mofetil in people with End-stage Liver Disease and Renal Insufficiency, run by Baylor College of Medicine at 1 site in United States.
Trial summary
The objective of this study is to compare the effects of two liver transplant immunosuppression regimens on renal function. Patients receiving the standard combination of prednisone and high-dose tacrolimus, a drug with known nephrotoxicity (Arm A) will be compared to patients receiving prednisone, low-dose tacrolimus… Read more about this study
- Who is it for?
- Ages Up to 16 years
- Conditions the study is for
- End-stage Liver Disease, Renal Insufficiency, Renal Failure
- What is being tested?
- mycophenolate mofetil
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2004to Nov 2005
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 Up to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- End-stage liver disease or acute fulminant hepatic failure recalcitrant to conventional medical or surgical therapy.
- Listed as candidate for pediatric liver transplantation listed with United Network for Organ Sharing (UNOS).
- Patients must be 18 years of age or younger.
Who cannot join
- History of autoimmune disease or primary sclerosing cholangitis.
- History of end-stage renal disease, dialysis treatment or acute renal failure (not including hepatorenal syndrome).
- Patients with pretransplant renal insufficiency as determined by a glomerular filtration rate (GFR) of <80 mL/min/1.73m2 (see below).
- Patients with renal agenesis or hypoplasia, polycystic kidney disease, or hydroureter seen on pretransplant renal ultrasound.
- Patients with malignancy or previous malignancy.
- Patients with active bacterial, viral, or fungal infections.
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- Patients with a pretransplant diagnosis of diabetes mellitus.
- Patients with history of previous transplant or multi-organ recipients.
- Patients with serological evidence of HIV, HBSAg or HCV.
- Patients with hereditary syndrome that causes genetic deficiency of hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) such as Lesch-Nyhan or Kelley-Seegmiller syndrome.
- Patients with history of phenylketonuria.
- Females that are pregnant or breastfeeding.
- Sexually active females who are not: a) post-menopausal, or b) surgically sterile, and c) using an acceptable method of contraception (oral contraceptive, implanted devices, injection, and barrier devices are acceptable; condoms used alone are not acceptable).
- Patients with alcohol abuse, substance abuse or smoking within the previous 6 months.
- Patients or caretakers of patients with psychogenic factors that preclude therapeutic compliance.
- Inability to reach participating hospital within 2 hours of notification.
- Any conditions or any circumstance that makes it unsafe to undergo a liver transplant.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective Randomized Trial of Prednisone and Tacrolimus Versus Prednisone, Tacrolimus and Mycophenolate Mofetil in Pediatric Liver Transplantation
The objective of this study is to compare the effects of two liver transplant immunosuppression regimens on renal function. Patients receiving the standard combination of prednisone and high-dose tacrolimus, a drug with known nephrotoxicity (Arm A) will be compared to patients receiving prednisone, low-dose tacrolimus and mycophenolate mofetil (MMF) (Arm B). MMF is an immunosuppression agent that has no associated nephrotoxicity. The primary end point of the study will be renal function as measured by glomerular filtration rate (GFR). Thirty pediatric liver transplant recipients will be randomized to these two arms in a 1:1 ratio (i.e. 15 patients in each group). Secondary end points will measure patient and graft outcome and incidence of immunosuppression-related complications, including: neurotoxicity, diabetes mellitus, growth retardation, vomiting, diarrhea, gastrointestinal hemorrhage, thrombocytopenia, anemia, leukopenia, acute or chronic liver graft rejection, posttransplant lymphoproliferative disease (PTLD), viral infections, fungal infections and bacterial infections.
Read the detailed description
The objective of this study is to compare the effects of two liver transplant immunosuppression regimens on renal function. Patients receiving the standard combination of prednisone and high-dose tacrolimus, a drug with known nephrotoxicity (Arm A) will be compared to patients receiving prednisone, low-dose tacrolimus and mycophenolate mofetil (MMF) (Arm B). MMF is an immunosuppression agent that has no associated nephrotoxicity. The primary end point of the study will be renal function as measured by glomerular filtration rate (GFR). Thirty pediatric liver transplant recipients will be randomized to these two arms in a 1:1 ratio (i.e. 15 patients in each group). Secondary end points will measure patient and graft outcome and incidence of immunosuppression-related complications, including: neurotoxicity, diabetes mellitus, growth retardation, vomiting, diarrhea, gastrointestinal hemorrhage, thrombocytopenia, anemia, leukopenia, acute or chronic liver graft rejection, posttransplant lymphoproliferative disease (PTLD), viral infections, fungal infections and bacterial infections.
- Conditions
- End-stage Liver Disease, Renal Insufficiency, Renal Failure
- Being tested
- mycophenolate mofetil, placebo medication
- Keywords
- renal insufficiency or failure; liver transplantation
- Medical subject headings
- End Stage Liver Disease, Renal Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Also called CellCept, MMF
immunosuppresion medication called mycophenolate mofetil, also known as MMF or CellCept
placebo medicationOther
Also called placebo pill
medication that looks like mycophenolate mofetil
| Study group | Role | What they receive |
|---|---|---|
| tacrolimus & corticosteroids | Placebo comparator | Standard post-transplant immunosuppression medications: tacrolimus and corticosteroids Receives: Other: placebo medication |
| low-dose tacrolimus + steroids + MMF | Experimental | Comparison arm: low-dose tacrolimus + steroids + MMF Receives: Drug: mycophenolate mofetil |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in glomerular filtration rate (GFR) two years after liver transplantation as calculated by the Schwartz formula
change in glomerular filtration rate (GFR) two years after liver transplantation as calculated by the Schwartz formula
Measured: two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2008 (estimated)
- Study startNov 2004
- Primary completionNov 2005
- Study completionNov 2005
Why it stopped: insufficient funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for End-stage Liver DiseaseStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, John Goss, Baylor College of Medicine
- Investigators
- John Goss, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00656266
- Sponsor's protocol ID
- H-15138
- Status last verified
- Mar 2020
- First submitted
- 7 Apr 2008
- Last updated
- 13 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Randomized Trial of Prednisone and Tacrolimus Versus Prednisone, Tacrolimus and Mycophenolate Mofetil in Pediatric Liver Transplantation
- ClinicalTrials.gov ID
- NCT00656266
- Acronym / sponsor ID
- H-15138
- Conditions
- End-stage Liver Disease, Renal Insufficiency, Renal Failure
- Keywords
- renal insufficiency or failure; liver transplantation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: insufficient funding
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Study start
- Nov 2004
- Primary completion
- Nov 2005 (actual)
- Study completion
- Nov 2005 (actual)
- First posted
- 11 Apr 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to compare the effects of two liver transplant immunosuppression regimens on renal function. Patients receiving the standard combination of prednisone and high-dose tacrolimus, a drug with known nephrotoxicity (Arm A) will be compared to patients receiving prednisone, low-dose tacrolimus and mycophenolate mofetil (MMF) (Arm B). MMF is an immunosuppression agent that has no associated nephrotoxicity. The primary end point of the study will be renal function as measured by glomerular filtration rate (GFR). Thirty pediatric liver transplant recipients will be randomized to these two arms in a 1:1 ratio (i.e. 15 patients in each group). Secondary end points will measure patient and graft outcome and incidence of immunosuppression-related complications, including: neurotoxicity, diabetes mellitus, growth retardation, vomiting, diarrhea, gastrointestinal hemorrhage, thrombocytopenia, anemia, leukopenia, acute or chronic liver graft rejection, posttransplant lymphoproliferative disease (PTLD), viral infections, fungal infections and bacterial infections.
Detailed description
The objective of this study is to compare the effects of two liver transplant immunosuppression regimens on renal function. Patients receiving the standard combination of prednisone and high-dose tacrolimus, a drug with known nephrotoxicity (Arm A) will be compared to patients receiving prednisone, low-dose tacrolimus and mycophenolate mofetil (MMF) (Arm B). MMF is an immunosuppression agent that has no associated nephrotoxicity. The primary end point of the study will be renal function as measured by glomerular filtration rate (GFR). Thirty pediatric liver transplant recipients will be randomized to these two arms in a 1:1 ratio (i.e. 15 patients in each group). Secondary end points will measure patient and graft outcome and incidence of immunosuppression-related complications, including: neurotoxicity, diabetes mellitus, growth retardation, vomiting, diarrhea, gastrointestinal hemorrhage, thrombocytopenia, anemia, leukopenia, acute or chronic liver graft rejection, posttransplant lymphoproliferative disease (PTLD), viral infections, fungal infections and bacterial infections.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| tacrolimus & corticosteroids | Placebo comparator | Other: placebo medicationStandard post-transplant immunosuppression medications: tacrolimus and corticosteroids |
| low-dose tacrolimus + steroids + MMF | Experimental | Drug: mycophenolate mofetilComparison arm: low-dose tacrolimus + steroids + MMF |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- End-stage liver disease or acute fulminant hepatic failure recalcitrant to conventional medical or surgical therapy.
- Listed as candidate for pediatric liver transplantation listed with United Network for Organ Sharing (UNOS).
- Patients must be 18 years of age or younger.
Key exclusion criteria (17 listed)
- History of autoimmune disease or primary sclerosing cholangitis.
- History of end-stage renal disease, dialysis treatment or acute renal failure (not including hepatorenal syndrome).
- Patients with pretransplant renal insufficiency as determined by a glomerular filtration rate (GFR) of <80 mL/min/1.73m2 (see below).
- Patients with renal agenesis or hypoplasia, polycystic kidney disease, or hydroureter seen on pretransplant renal ultrasound.
- Patients with malignancy or previous malignancy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mycophenolate mofetilAlso: CellCept, MMFimmunosuppresion medication called mycophenolate mofetil, also known as MMF or CellCept | Drug | low-dose tacrolimus + steroids + MMF |
| placebo medicationAlso: placebo pillmedication that looks like mycophenolate mofetil | Other | tacrolimus & corticosteroids |
Primary outcome
change in glomerular filtration rate (GFR) two years after liver transplantation as calculated by the Schwartz formula
Time frame: two years
change in glomerular filtration rate (GFR) two years after liver transplantation as calculated by the Schwartz formula
Results and safety
The study was stopped early (reason given: insufficient funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Goss, MD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing mycophenolate mofetil in people with End-stage Liver Disease and Renal Insufficiency, run by Baylor College of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: insufficient funding). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change in glomerular filtration rate (GFR) two years after liver transplantation as calculated by the Schwartz formula (time frame: two years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2020.