ET for the prevention of CMV infection/disease in pediatric HSCT recipients

Phase 2

Completed

• Conditions: Pediatric participants receiving HSCT at risk for CMV infection and/or disease

• Registration Number: JPRN-jRCT2080225146
• Lead Sponsor: MSD K.K.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-03-30
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: completed
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

All participants 12 to <18 years old must have documented positive CMV serostatus(CMV IgG seropositive)for the recipient(R+)within 90 days prior to enrollment. Participants from birth to <12 years old must have documented positive CMV serostatus(CMV IgG seropositive)for the recipient(R+)within 90 days prior to enrollment and/or the donor(D+); the donor serostatus should be documented within 1 year prior to enrollment.
- Is the recipient of a first allogeneic HSCT(bone marrow, peripheral blood stem cell, or cord blood transplant).
- Has undetectable CMV DNA from a plasma or whole blood sample collected within 5 days prior to enrollment.
- Is within 28 days post-HSCT at the time of enrollment.
- Females are not pregnant, not breastfeeding, and is not a woman of childbearing potential(WOCBP); or is a WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 28 days after the last dose of study intervention.
- Participants from 2 to <18 years of age must not be on concomitant Cyclosporin A(CsA), and must be able to take LET tablets or the oral granules(either by mouth or via G tube/NG tube), provided the participant does not have a condition that may interfere with the absorption of oral medication(e.g. vomiting, diarrhea, or a malabsorptive condition)from the day of enrollment until the intensive PK sampling is completed in these participants.
- For participants 2 <12 years old their weight should be at least 10 kg; for participants from birth to <2 years old their weight should be at least 2.5 kg and less than or equal to 15 kg at the time of enrollment.

Exclusion Criteria

- Has received a previous allogeneic HSCT(Note: receipt of a previous autologous HSCT is acceptable).
- Has a history of CMV end-organ disease within 6 months prior to enrollment.
- Has evidence of CMV viremia at any time from either signing of the ICF or the HSCT procedure, whichever is earlier, until the time of enrollment.
- Has suspected or known hypersensitivity to active or inactive ingredients of LET formulations.
- Has severe hepatic insufficiency within 5 days prior to enrollment.
- Is a)on renal replacement therapy(eg, hemodialysis, peritoneal dialysis)OR b)has end-stage renal impairment.
- Has both moderate hepatic insufficiency and moderate-to-severe renal insufficiency.
- Has an uncontrolled infection on the day of enrollment.
- Requires mechanical ventilation or is hemodynamically unstable at the time of enrollment.
- Has a documented positive result for a human immunodeficiency virus antibody(HIVAb)test at any time prior to enrollment, or for hepatitis C virus antibody(HCV-Ab)with detectable HCV RNA, or hepatitis B surface antigen(HBsAg)within 90 days prior to enrollment.
- Has active solid tumor malignancies with the exception of localized basal cell or squamous cell skin cancer or the condition under treatment(e.g. lymphomas).
- Has a preexisting cardiac condition a)for which the patient is currently being treated or b)which required hospitalization within the last 6 months or c)that may be expected to recur during the course of the trial.
- Has received within 7 days prior to screening any of the following: ganciclovir; valganciclovir; foscarnet; acyclovir; valacyclovir; famciclovir.
- Has received within 30 days prior to screening of any of the following: cidofovir; CMV immunoglobulin; any investigational CMV antiviral agent/biologic therapy; Rifampin and other strong inducers(such as phenytoin, carbamazepine, St John's wort(Hypericum perforatum), rifabutin and phenobarbital)and moderate inducers such as nafcillin, thioridazine, modafinil and bosentan.
- Has received LET at any time prior to enrollment in this study.
- Is currently participating or has participated in a study with an unapproved investigational compound or device within 28 days, or 5X half-life of the investigational compound(excluding monoclonal antibodies), whichever is longer, of initial dosing in this study.
- Has previously participated in this study or any other study involving LET.
- Has previously participated or is currently participating in any study involving administration of a CMV vaccine or another CMV investigational agent, or is planning to participate in a study of a CMV vaccine or another CMV investigational agent during the course of this study.
- Is pregnant or expecting to conceive, is breastfeeding, or plans to breastfeed from the time of consent through 28 days after the last dose of study intervention.
- Is expecting to donate eggs starting from the time of consent through 28 days after the last dose of study intervention.
- Has clinically relevant drug or alcohol abuse within 12 months of screening that may interfere with participant treatment, assessment, or compliance with the protocol, as assessed by the investigator.

Study & Design

• Study Type: Interventional
• Study Design: Not specified