Hematopoietic Stem Cell Transplant (HSCT)Phase 1NCT05524246
Pravastatin as a Prophylactic to Reduce Endothelial Injury in Pediatric Patients With Elevated Body Mass Index
A Phase 1 study testing Pravastatin in people with Hematopoietic Stem Cell Transplant (HSCT) and Endothelial Injury, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
Chemotherapy and radiation used in patients undergoing bone marrow transplant (BMT) disrupts the endothelial lining (a thin layer of cells inside the blood vessels) which is found all throughout the body including the kidney, heart, lungs, and intestines. Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Hematopoietic Stem Cell Transplant (HSCT), Endothelial Injury
- What is being tested?
- Pravastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Jan 2023to 5 Mar 2025
- Phase
- Phase 1
- 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 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Scheduled for allogeneic stem cell transplant
- Ages ≥ 2 - ≤ 25 years old
- Elevated BMI defined by the Center for Disease Control and Prevention definitions. Both overweight (BMI between 85th-94th percentile) and obese (BMI >95th percentile) patients are eligible
- All diagnoses are eligible
Who cannot join
- Patients with documented anaphylaxis to pravastatin
- Patients will be ineligible if they are unable to take medication orally or enterally (i.e. intestinal failure)
- Patients with elevations in ALT/AST levels 3x ULN at time of enrollment
- Patients with renal impairment as clinically measured (GFR <50 ml/min/1.73m2) at time of enrollment
- Patients with known neuromuscular and metabolic disorders associated with an increased risk of rhabdomyolysis (ie metabolic muscle disorders, mitochondrial disorders, and muscular dystrophies)
- Patients taking any drugs that are known substrates for OATP1B1 and OATP1B3 transporters
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Pilot Trial of Pravastatin as a Novel Prophylactic Medicine to Reduce Endothelial Injury in Pediatric Patients With Elevated Body Mass Index
Chemotherapy and radiation used in patients undergoing bone marrow transplant (BMT) disrupts the endothelial lining (a thin layer of cells inside the blood vessels) which is found all throughout the body including the kidney, heart, lungs, and intestines. Disruption of this endothelial lining can lead to complications such as graft-vs-host disease (GVHD), thrombotic microangiopathy (TMA) and veno-occlusive disease (VOD). The purpose of this research study is to help investigators see if pravastatin is safe and well tolerated in patients undergoing BMT to see if it will reduce endothelial injury after BMT.
The investigator hypothesizes that prophylactic pravastatin in pediatric allogeneic hematopoietic stem cell transplant recipients with elevated BMI is safe and feasible.
- Being tested
- Pravastatin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PravastatinDrug
Participants will receive pravastatin orally once daily through the first 35 days after bone marrow transplant.
| Study group | Role | What they receive |
|---|---|---|
| Pravastatin Prophylactic Treatment | Experimental | Not described Receives: Drug: Pravastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of participants who developed clinically significant transaminitis, renal dysfunction and rhabdomyolysis
Every enrolled patient will have routine liver function test (LFT) and renal function profile monitoring as part of their standardized transplant care. Transaminitis is expected and often multifactorial in the HSCT population, (i.e. preparative regimen, azole antifungal prophylaxis, or post-transplant complications such as infection and GVHD), therefore, we have determined modified parameters to hold or modify the dose of pravastatin. We will modify and/or hold pravastatin dosing at the time of transaminase elevations > 3X upper limit of normal unless a clear-cut alternative explanation can be provided. Additionally, we will also check creatinine kinase (CK) levels on admission and once weekly while the patient is actively taking pravastatin to monitor for rhabdomyolysis.
Measured: Until day 35 after bone marrow transplant when pravastatin is discontinued
Percentage of participants who adhered to the medication plan
Participants will be considered adhered to the medication plan if they took the study drug at least 70% of the time. Nursing documentation of pravastatin administration and drug diaries will be used as documentation of compliance.
Measured: Until day 35 after bone marrow transplant when pravastatin is discontinued
Secondary outcomes (8)
Number of participants with overall survival
Measured: 100 days after bone marrow transplant
Number of participants with graft failure
Graft failure is defined as follows:
Primary graft failure is defined as no evidence of engraftment or hematological recovery of donor cells, within the first month after transplant, without evidence of disease relapse.
Secondary graft failure refers to the loss of a previously functioning graft, resulting in cytopenia involving at least two blood cell lineages.
Measured: 100 days after bone marrow transplant
Number of participants with primary disease relapse
Primary disease relapse is defined as: The return of a disease or the signs and symptoms of a disease after a period of improvement.
Measured: 100 days after bone marrow transplant
Median number of days until neutrophil engraftment
Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm3 (0.5 x 10^9/L) or greater.
Measured: Through study completion, an average of up to 100 days
Median number of days until platelet engraftment
Platelet engraftment is usually defined as independence from platelet transfusion for at least 7 days with a platelet count of more than >20 × 10^9/L
Measured: Through study completion, an average of up to 100 days
Assessment of SLCO1B1 genotyping
The SLCO1B1 gene encodes for making the protein organic anion transporting polypeptide 1B1 (OATP1B1), which is the transporter for drugs, including statins, into the liver. Patients enrolled on study will have their SLCO1B1 genotyping performed from whole blood samples at baseline to determine how this affects their overall statin clearance.
Measured: Baseline
Measurement of sphingosine-1-phosphate (S1P) levels in plasma
S1P levels will be measured on weekly intervals using mass spectrometry
Measured: Weekly from admission until day 35 from bone marrow transplant
Number of participants who adhered to the medication plan
The lipid profile is a blood test that includes measurements of cholesterol, triglycerides, High-density lipoprotein (HDL) and Low-density lipoprotein (LDL). It is a routine test that monitors cholesterol levels. Pravastatin reduces cholesterol production. The lipid profile will be measured prior to the first dose of pravastatin. After the last dose of pravastatin the lipid profile will be measured. Any reduction in total cholesterol and LDL levels will be used as a surrogate marker for medication adherence.
Measured: Through study completion, an average of 35 days after bone marrow transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2022
- Study start27 Jan 2023
- Primary completion31 Dec 2024
- Study completion5 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Stem Cell Transplant (HSCT)Studies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jane Koo, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05524246
- Sponsor's protocol ID
- 2022-0308
- Status last verified
- Apr 2025
- First submitted
- 24 Aug 2022
- Last updated
- 18 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Trial of Pravastatin as a Novel Prophylactic Medicine to Reduce Endothelial Injury in Pediatric Patients With Elevated Body Mass Index
- ClinicalTrials.gov ID
- NCT05524246
- Acronym / sponsor ID
- 2022-0308
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 27 Jan 2023 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 5 Mar 2025 (actual)
- First posted
- 1 Sep 2022 (actual)
- 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.
Chemotherapy and radiation used in patients undergoing bone marrow transplant (BMT) disrupts the endothelial lining (a thin layer of cells inside the blood vessels) which is found all throughout the body including the kidney, heart, lungs, and intestines. Disruption of this endothelial lining can lead to complications such as graft-vs-host disease (GVHD), thrombotic microangiopathy (TMA) and veno-occlusive disease (VOD). The purpose of this research study is to help investigators see if pravastatin is safe and well tolerated in patients undergoing BMT to see if it will reduce endothelial injury after BMT.
The investigator hypothesizes that prophylactic pravastatin in pediatric allogeneic hematopoietic stem cell transplant recipients with elevated BMI is safe and feasible.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pravastatin Prophylactic Treatment | Experimental | Drug: Pravastatin |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Scheduled for allogeneic stem cell transplant
- Ages ≥ 2 - ≤ 25 years old
- Elevated BMI defined by the Center for Disease Control and Prevention definitions. Both overweight (BMI between 85th-94th percentile) and obese (BMI >95th percentile) patients are eligible
- All diagnoses are eligible
Key exclusion criteria (6 listed)
- Patients with documented anaphylaxis to pravastatin
- Patients will be ineligible if they are unable to take medication orally or enterally (i.e. intestinal failure)
- Patients with elevations in ALT/AST levels 3x ULN at time of enrollment
- Patients with renal impairment as clinically measured (GFR <50 ml/min/1.73m2) at time of enrollment
- Patients with known neuromuscular and metabolic disorders associated with an increased risk of rhabdomyolysis (ie metabolic muscle disorders, mitochondrial disorders, and muscular dystrophies)
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 |
|---|---|---|
| PravastatinParticipants will receive pravastatin orally once daily through the first 35 days after bone marrow transplant. | Drug | Pravastatin Prophylactic Treatment |
Primary outcome
Percentage of participants who developed clinically significant transaminitis, renal dysfunction and rhabdomyolysis
Time frame: Until day 35 after bone marrow transplant when pravastatin is discontinued
Every enrolled patient will have routine liver function test (LFT) and renal function profile monitoring as part of their standardized transplant care. Transaminitis is expected and often multifactorial in the HSCT population, (i.e. preparative regimen, azole antifungal prophylaxis, or post-transplant complications such as infection and GVHD), therefore, we have determined modified parameters to hold or modify the dose of pravastatin. We will modify and/or hold pravastatin dosing at the time of transaminase elevations > 3X upper limit of normal unless a clear-cut alternative explanation can be provided. Additionally, we will also check creatinine kinase (CK) levels on admission and once weekly while the patient is actively taking pravastatin to monitor for rhabdomyolysis.
Percentage of participants who adhered to the medication plan
Time frame: Until day 35 after bone marrow transplant when pravastatin is discontinued
Participants will be considered adhered to the medication plan if they took the study drug at least 70% of the time. Nursing documentation of pravastatin administration and drug diaries will be used as documentation of compliance.
8 secondary outcomes
Number of participants with overall survival
Time frame: 100 days after bone marrow transplant
Number of participants with graft failure
Time frame: 100 days after bone marrow transplant
Number of participants with primary disease relapse
Time frame: 100 days after bone marrow transplant
Median number of days until neutrophil engraftment
Time frame: Through study completion, an average of up to 100 days
Median number of days until platelet engraftment
Time frame: Through study completion, an average of up to 100 days
Assessment of SLCO1B1 genotyping
Time frame: Baseline
Measurement of sphingosine-1-phosphate (S1P) levels in plasma
Time frame: Weekly from admission until day 35 from bone marrow transplant
Number of participants who adhered to the medication plan
Time frame: Through study completion, an average of 35 days after bone marrow transplant
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2025.
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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.
Jane Koo, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Pravastatin in people with Hematopoietic Stem Cell Transplant (HSCT) and Endothelial Injury, run by Children's Hospital Medical Center, Cincinnati 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 4 inclusion and 6 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: finished, as of the sponsor's last update on 18 Apr 2025. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Percentage of participants who developed clinically significant transaminitis, renal dysfunction and rhabdomyolysis (time frame: Until day 35 after bone marrow transplant when pravastatin is discontinued). The study lists 2 primary outcomes in all. 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 18 Apr 2025.