Safety and tolerability of IMR-687 in Beta Thalassemia
Research type
Research Study
Full title
A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
IRAS ID
1003229
Contact name
Perla Eleftheriou
Contact email
Eudract number
2019-002989-12
Research summary
Research Summary
β-thalassemia is a rare inherited blood disorder in which the body does not produce
haemoglobin (Hb) or produces very little. Hb is a protein in blood that carries oxygen
from the lungs to the rest of the body. In participants patients with β-thalassemia, low
levels of Hb lead to a lack of oxygen in many parts of the body. This can lead to anaemia
(deficiency of red blood cells or haemoglobin in the blood). Symptoms of β-thalassemia
include fatigue, growth abnormalities, bone disease, heart problems and possibly blood
clots. IMR-687 helps to stimulate the production of Foetal haemoglobin, hence it is
expected to increase the total amount of haemoglobin.
β-thalassemia which requires clinical intervention is classified into two groups: nontransfusion-
dependent β-thalassemia (NTDT) and transfusion-dependent β-thalassemia
(TDT).
The purpose of this study is to evaluate how safe and well-tolerated IMR-687 is in
participants with β-thalassemia. IMR-687 helps to stimulate the production of foetal
haemoglobin, hence it is expected to increase the total amount of
haemoglobin.Participants will be randomly assigned (like flipping a coin) to 1 of 2
treatment groups which include treatment with a low dose of IMR-687 or placebo. Some
participants may be given a higher dose of IMR-687 based on the Data Monitoring
Committee (independent group)after reviewing of safety data from 5 participants
receiving the low dose of IMR-687.
The planned duration of study participation is approximately 44 weeks consisting of:
screening period (4 weeks), treatment period (36 weeks) and safety follow-up assessment
(4 weeks). There is also a retrospective data review to collect information on transfusion
activity in the 12 weeks preceding the Screening visit. Participants will be expected to
take two tablets once a day for 36 weeks. TDT participants will visit the study site every 3
weeks and NTDT participants will visit the study site every 4 weeks. Safety assessments
will be done throughout the study.Summary of Results
The study was early terminated due to the results of the interim analysis which demonstrated that while the study drug (IMR-687) was generally well tolerated, it failed to show any meaningful benefit in transfusion burden or improvement in most disease-related biomarkers. Investigators were notified on 06 April 2022 to contact patients and instruct them to stop taking the study drug and return to the site for their last study visit. Based on these results, the sponsor decided to discontinue this study as well as future development of IMR-687 in subjects with Beta Thalassemia, therefore an abbreviated CSR has been issued.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
20/SC/0150
Date of REC Opinion
2 Jun 2020
REC opinion
Further Information Favourable Opinion