Pravastatin as a Prophylactic to Reduce Endothelial Injury in Pediatric Patients With Elevated Body Mass Index

Cincinnati Children's Hospital Medical Center

Status and phase: Completed; Phase 1

Conditions: Hematopoietic Stem Cell Transplant (HSCT); Endothelial Injury

Treatments: Drug: Pravastatin

Study type: Interventional

Funder types: Other

Identifiers

NCT05524246

2022-0308

Details and patient eligibility

About

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.

Enrollment: 25 patients

Sex: All

Ages: 2 to 25 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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 design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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25 participants in 1 patient group

Pravastatin Prophylactic Treatment

Experimental group

Treatment:

Drug: Pravastatin