Pravastatin for Endothelial Injury

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Cincinnati Children's Hospital Medical Center, Cincinnati, OH
Endothelial Injury+2 More
Pravastatin - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study 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. 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.

Eligible Conditions

  • Endothelial Injury
  • Hematopoietic Stem Cell Transplantation (SCT)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

2 Primary · 8 Secondary · Reporting Duration: Baseline

Day 100
Number of participants with graft failure
Number of participants with overall survival
Number of participants with primary disease relapse
Baseline
Assessment of SLCO1B1 genotyping
Day 35
Number of participants who adhered to the medication plan
Day 100
Median number of days until neutrophil engraftment
Median number of days until platelet engraftment
Day 35
Percentage of participants who adhered to the medication plan
Percentage of participants who developed clinically significant transaminitis, renal dysfunction and rhabdomyolysis
Day 35
Measurement of sphingosine-1-phosphate (S1P) levels in plasma

Trial Safety

Safety Progress

1 of 3

Side Effects for

Pitavastatin 4 mg QD
2%Headache
2%Fatigue
2%Blood CK increased
2%Upper abdominal pain
2%Diarrhoea
1%Respiratory failure
1%Gastroenteritis viral
1%Transient ischaemic attack
1%Gastroenteritis
1%Atrial septal defect
1%Enterovesical fistula
1%Herpes dermatitis
1%Diverticulitis
1%Chronic obstructive pulmonary disease
1%Chest pain
1%Multiple fractures
1%Nausea
0%Arteriosclerosis coronary artery
0%Muscle haemorrhage
0%Cerebrovascular accident
0%Myocardial infarction
This histogram enumerates side effects from a completed 2013 Phase 4 trial (NCT01301066) in the Pitavastatin 4 mg QD ARM group. Side effects include: Headache with 2%, Fatigue with 2%, Blood CK increased with 2%, Upper abdominal pain with 2%, Diarrhoea with 2%.

Trial Design

1 Treatment Group

Pravastatin Prophylactic Treatment
1 of 1
Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Pravastatin · No Placebo Group · Phase 1

Pravastatin Prophylactic Treatment
Drug
Experimental Group · 1 Intervention: Pravastatin · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pravastatin
2010
Completed Phase 4
~11530

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline
Closest Location: Cincinnati Children's Hospital Medical Center · Cincinnati, OH
Photo of Cincinnati  1Photo of Cincinnati  2Photo of Cincinnati  3
2011First Recorded Clinical Trial
1 TrialsResearching Endothelial Injury
706 CompletedClinical Trials

Eligibility Criteria

Age < 65 · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are aged 2 to 25 years old.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.