A study to investigate APL-4098 in adults with advanced AML or MDS
Research type
Research Study
Full title
A Phase 1/2 Study to Assess the Safety and Antitumor Activity of APL-4098 Alone and/or in Combination with Azacitidine in Adults with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome/AML (MDS/AML) or Myelodysplastic Syndrome with Excess Blasts (MDS-EB)
IRAS ID
1009360
Contact name
Sunil Kadam
Contact email
Sponsor organisation
Apollo Therapeutics, Limited
Research summary
This study is being done to find out if the investigational medicine called APL-4098 when given alone and/or in combination with azacitidine is safe, tolerable and effective to administer to adult patients with relapsed or refractory (R/R) acute myeloid leukaemia (AML), myelodysplastic syndrome/AML (MDS/AML) or myelodysplastic syndrome with excess blasts (MBS-EB).
Patients with R/R AML or MDS/AML have a very poor prognosis with significant unmet medical need. Additionally, the high-risk MDS with excess blasts (MDS-EB) subtype is strongly associated with increased risk of AML transformation and decreased survival.
Stem Cell Transplantation may be an option of treatment for eligible patients; this is the only remaining pathway to cure. It is recommended that patients with R/R AML and/or high-risk MDS are treated through clinical trials whenever possible.
This is a first in human study and has 2 parts, called Phase 1 and Phase 2. The goal is to find the RP2D(s) of APL-4098 monotherapy (and in combination with azacitidine) and to determine the safety and anti-tumour activity of APL-4098 as single agent ( and/or in combination with azacitidine).
Participants will take APL-4098 orally in the first week of every cycle once daily until relapse/disease progression, experience side effects that make it necessary to stop taking APL-4098, start a new anti-cancer treatment, or consent withdrawal.
The study will enrol approximately 112 participants in several countries (approximately 4 research sites in the United Kingdom, 6 research sites in Australia, and 20 research sites in the United States). Following a screening visit, eligible participants will attend clinic visits on Days 1, 2, 4, 8, 15 and 22 of Cycle 1 and days 1 and 15 of Cycle 2. From Cycle 3 onwards, clinic visits will be on Day 1 only. There will be a safety follow-up visit 28-days after the last dose of AP-4098 and long-term follow up visits once every 3 months.
Study duration is approximately 3 years.REC name
South Central - Berkshire Research Ethics Committee
REC reference
24/SC/0060
Date of REC Opinion
26 Mar 2024
REC opinion
Further Information Unfavourable Opinion