Phase 2 Study to Evaluate Brincidofovir for the Prevention of Adenovirus Disease

Phase 2

Completed

Brief Summary: This study was designed to assess the safety and efficacy of preemptive treatment with oral brincidofovir (BCV), as compared to placebo, for the prevention of adenovirus (AdV) disease in recipients of hematopoietic stem cell transplantation (HCT) with asymptomatic AdV viremia.

Detailed Description: This was a Phase 2, randomized, multicenter, placebo-controlled study for pediatric and adult subjects who had undergone hematopoietic stem cell transplantation (HCT) and who had been identified as having asymptomatic adenovirus (AdV) viremia \[i.e., had detectable AdV DNA in plasma based on polymerase chain reaction testing performed at the local laboratory with no AdV disease symptoms\]. The primary objectives of the study were to assess the safety and tolerability of oral brincidofovir (BCV), and to estimate the treatment failure rate based on an efficacy endpoint with 2 different dosing regimens of oral BCV versus placebo.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Brincidofovir	Brincidofovir	* Adult subjects: 200mg BCV administered as 50mg tablets taken orally either once weekly (QW; 4 tablets) or twice weekly (BIW; 2 tablets). * Pediatric subjects: 4mg/kg BCV (not to exceed a total single dose of 200mg) administered using a 10 mg/mL liquid formulation taken orally either QW (as 4 mg/kg) or BIW (as 2 mg/kg).
Placebo	Placebo	* Adult subjects: Matching placebo tablets taken orally either once weekly (QW; 4 tablets) or twice weekly (BIW; 2 tablets). * Pediatric subjects: Matching liquid placebo taken orally either QW (as 4 mg/kg) or BIW (as 2 mg/kg).

Primary Outcome Measures

Name	Time	Method
Number of Participants With Clinically Significant AdV Infection	12 weeks	The primary objective of this study was to evaluate the safety and efficacy of preemptive treatment with brincidofovir (BCV) versus placebo for the prevention of adenovirus (AdV) disease in recipients of hematopoietic stem cell transplantation (HCT) with asymptomatic AdV viremia. The outcome measure for the primary endpoint was treatment failure, a composite endpoint that consisted of the following: * Progression to probable AdV disease (other positive causes/agents have been ruled out and subject has disease-targeted organ-specific signs or symptoms) or definitive AdV disease (AdV detected in disease-targeted organ/system biopsy via antigen/immunohistochemistry, culture, and/or polymerase chain reaction and has at least 1 disease-targeted organ-specific sign or symptom); or * Increasing AdV viremia (defined as an increase from baseline in AdV viremia by ≥1 log10, confirmed on a second measurement, at least 1 week apart) and requiring discontinuation from blinded therapy.

Secondary Outcome Measures

Name	Time	Method

Locations (30): Children's Hospital of Alabama
🇺🇸
Birmingham, Alabama, United States
Pheonix Children's Hospital
🇺🇸
Phoenix, Arizona, United States
City of Hope National Medical Center
🇺🇸
Duarte, California, United States
Childrens hospital of LA
🇺🇸
Los Angeles, California, United States
CHOC Children's Hospital
🇺🇸
Orange, California, United States
University of California, San Francisco
🇺🇸
San Francisco, California, United States
Lucile Packard Childrens hopsital at Stanford
🇺🇸
Stanford, California, United States
The Children's Hospital-Denver
🇺🇸
Aurora, Colorado, United States
Children's National Medical Center
🇺🇸
Washington, District of Columbia, United States
Indiana University
🇺🇸
Indianapolis, Indiana, United States
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Children's Hospital of Alabama
🇺🇸Birmingham, Alabama, United States