EDP-938: Recipients of Haematopoietic Cell Transplants with Acute RSV
Research type
Research Study
Full title
A Phase 2b, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Effects of EDP-938 in Adult Hematopoietic Cell Transplant Recipients With Acute Respiratory Syncytial Virus Infection of the Upper Respiratory Tract
IRAS ID
296099
Contact name
Dragana Milojkovic
Contact email
Sponsor organisation
Enanta Pharmaceuticals, Inc.
Eudract number
2020-002213-18
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 3 months, 1 days
Research summary
The purpose of this research study is to see if an experimental drug called EDP-938 is safe and effective when administered to adult haematopoietic cell transplant recipients with an acute RSV infection of the upper respiratory tract, as compared to treating RSV infection with a placebo (a pill that does not have active medicine in it). EDP-938 has already been tested in approximately 380 participants, and the results showed that this experimental drug was well tolerated with a favourable safety profile.
The study involves assessments and procedures including: physical exams, nasal swab, vital signs, electrocardiogram (ECG), questionnaires, blood and urine tests, medical history and a chest x-ray.
The study aims to enrol about 200 participants, aged between 18 and 75 years, inclusive, globally and may include sites in North America, South America, Europe, Africa, Australia, and Asia-Pacific countries. In the UK we aim to enroll approximately 12 participants. Participation for each participant in this study will last about 7 weeks (49 days); consisting of Screening (1 day), Treatment (21 days) and Follow-up period (28 days). Approximately 12 of these patients will be based in the UK across 4 hospitals.
The study participants will be assigned to receive either EDP-938 or placebo (a pill that has no active medication) by chance (like flipping a coin). The chances of receiving EDP-938 is 65% or placebo is 35%; neither the participant nor their study doctor or nurse will be able to pick which medication they receive. What this means is that out of the 200 patients that will take part in this study, 130 will receive EDP-938 and 70 will receive placebo.
Lay Summary of Results
A total of 9 participants who received stem cell transplants, aged between 25 and 63 years, were enrolled in the study to receive either EDP-938 or Placebo. 5 participants received EDP-938 (1 received 150 mg, 1 received 400 mg, and 3 received 800 mg EDP-938) and 4 participants received placebo for a total of 21 days. Due to the small number of participants, efficacy conclusions were not possible. Adverse events were reported in all of the EDP-938 treated participants and 3 of the placebo treated participants and were consistent with RSV infection or transplant complications. Three (3) EDP-938 treated participants experienced serious adverse events: viral oral infection in 1 participant, diarrhea and decreased appetite in 1 participant, and elevated liver enzymes in 1 participant. All 9 participants completed the study. The study was terminated early due to low enrollment.
Has the registry been updated to include summary results?: No
If yes - please enter the URL to summary results:
If no – why not?: Clinicaltrials.gov will be updated to include the results in the next few months.
Did you follow your dissemination plan submitted in the IRAS application form (Q A51)?: No
If yes, describe or provide URLs to disseminated materials:
If pending, date when dissemination is expected: 30/09/2024
If no, explain why you didn't follow it: Dissemination according to the dissemination plan is pending. The results will be posted on clincaltrials.gov and EudraCT in the next few months. The clinical study report will be submitted to the US FDA. The results will not be published in a peer reviewed scientific journal or in a conference presentation because the study only enrolled 9 participants before being terminated due to low enrollment. Due to the small number of participants enrolled, scientifically sound conclusions are not possible.
Have participants been informed of the results of the study?: No
If yes, describe and/or provide URLs to materials shared and how they were shared:
If pending, date when feedback is expected:
If no, explain why they haven't: Study participants have not been informed of the study results as the study was terminated after only 9 subjects were enrolled and interpretion of the safety and efficay data is limited. Should any participant want to be informed of the study results, we will inform them.
Have you enabled sharing of study data with others?: No
If yes, describe or provide URLs to how it has been shared:
If no, explain why sharing hasn't been enabled: Study data will be shared through clinicaltrials.gov in the next few months.
Have you enabled sharing of tissue samples and associated data with others?: No
If yes, describe or provide a URL:
If no, explain why: Tissue samples were not shared as tissue was not collected. Nasal swab samples were collected and used for viral analysis.REC name
London - Surrey Borders Research Ethics Committee
REC reference
21/LO/0325
Date of REC Opinion
5 Jul 2021
REC opinion
Further Information Favourable Opinion