What side effects are associated with this type of intervention?

Common treatments for endothelial injury, particularly those with lipid-lowering and anti-inflammatory effects like pravastatin, often have side effects such as muscle-related issues (myopathy and rare rhabdomyolysis), elevated liver enzymes, gastrointestinal problems (nausea and constipation), and an increased risk of diabetes. PCSK9 inhibitors, another lipid-lowering option, can cause injection site reactions, hypersensitivity reactions, and occasionally neurocognitive symptoms.

What prior FDA approvals exist?

Statins, including atorvastatin and simvastatin, have been FDA-approved primarily for lowering cholesterol but also offer endothelial protection through lipid-lowering and anti-inflammatory effects. Other lipid-lowering agents like ezetimibe and PCSK9 inhibitors (e.g., evolocumab) have also been approved and may provide similar endothelial benefits. While these drugs are not specifically approved for endothelial injury, their mechanisms support endothelial health.

What other types of research exist?

Promising novel alternatives to Pravastatin for endothelial protection in 2024 include Evinacumab, a monoclonal antibody targeting ANGPTL3, and TA-8995, a CETP inhibitor. Both have shown significant lipid-lowering and anti-inflammatory effects in clinical trials.

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HMG-CoA Reductase Inhibitor

Pravastatin for Endothelial Injury in Pediatric Patients

Phase 1

Recruiting

Led By Jane Koo, MD

Research Sponsored by Children's Hospital Medical Center, Cincinnati

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if pravastatin, a cholesterol-lowering drug, can protect blood vessel linings in children with high BMI undergoing bone marrow transplants. The goal is to see if it can prevent serious complications by keeping these blood vessels healthy.

Who is the study for?

This trial is for pediatric patients aged 2-25 with an elevated BMI who are scheduled for a stem cell transplant. It's open to all diagnoses, but not for those with kidney issues, taking certain drugs, allergic to pravastatin, unable to take oral medication, or have specific muscle disorders.

What is being tested?

The study tests if Pravastatin can prevent damage to the endothelial lining in blood vessels after bone marrow transplants in young patients with high body weight. The goal is to see if it reduces complications like GVHD and VOD.

What are the potential side effects?

Potential side effects of Pravastatin may include muscle pain or weakness (rhabdomyolysis), liver enzyme changes, and possible allergic reactions. However, this study aims to confirm its safety in the given patient population.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 100 days after bone marrow transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~100 days after bone marrow transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 100 days after bone marrow transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of participants who adhered to the medication plan

Percentage of participants who developed clinically significant transaminitis, renal dysfunction and rhabdomyolysis

Secondary study objectives

Assessment of SLCO1B1 genotyping

Measurement of sphingosine-1-phosphate (S1P) levels in plasma

Median number of days until neutrophil engraftment

+4 more

Side effects data

From 2013 Phase 4 trial • 252 Patients • NCT01301066

Headache

Upper abdominal pain

Blood CK increased

Diarrhoea

Fatigue

Enterovesical fistula

Diverticulitis

Atrial septal defect

Transient ischaemic attack

Multiple fractures

Respiratory failure

Chronic obstructive pulmonary disease

Chest pain

Gastroenteritis

Gastroenteritis viral

Herpes dermatitis

Nausea

100%

80%

60%

40%

20%

Study treatment Arm

Pitavastatin 4 mg QD

Pravastatin 40 mg QD

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Pravastatin Prophylactic TreatmentExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pravastatin

2010

Completed Phase 4

~11530

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Pravastatin, a commonly used statin, works by inhibiting the enzyme HMG-CoA reductase, which plays a crucial role in cholesterol synthesis. This lipid-lowering effect reduces the amount of low-density lipoprotein (LDL) cholesterol in the blood, which is beneficial because high levels of LDL cholesterol can lead to atherosclerosis and further endothelial damage. Additionally, pravastatin has anti-inflammatory properties that help reduce vascular inflammation, a key factor in endothelial injury. By lowering both cholesterol levels and inflammation, pravastatin helps protect the endothelial lining of blood vessels, reducing the risk of complications such as graft-vs-host disease, thrombotic microangiopathy, and veno-occlusive disease in patients undergoing treatments like bone marrow transplants.

Sustained Focal Vascular Inflammation Accelerates Atherosclerosis in Remote Arteries.Hydroxymethylglutaryl co-enzyme A reductase inhibition attenuates endotoxin-mediated inflammatory responses.