A Phase 1/2 Study of Oral TP-3654 in Patients with Myelofibrosis
Research type
Research Study
Full title
A Phase 1/2, Open-label, Dose-escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral TP-3654 in Patients with Intermediate or High-risk Primary or Secondary Myelofibrosis
IRAS ID
1007189
Contact name
Brian Caselli
Contact email
Sponsor organisation
Sumitomo Pharma Oncology, Inc.
Eudract number
2022-502597-16
Clinicaltrials.gov Identifier
Research summary
Myelofibrosis is a rare type of blood cancer where your bone marrow (the soft, spongy tissue inside of your bones) is replaced by fibrous scar tissue. It’s a form of chronic leukemia and a myeloproliferative disorder.
The purpose of this study is to evaluate the safety of TP-3654 study drug as a treatment for myelofibrosis. This drug can only be used in clinical studies and is not yet approved by health authorities. The main goals of this study are to evaluate different doses of the study drug to find the dose level most likely to be safe for people with myelofibrosis. Other goals of the study are to get information on the potential clinical effects of study drug that might benefit patients with myelofibrosis.
The main result measured for a study is to assess the safety of different doses of the study drug by measuring the type, frequency, and intensity of side effects during the course of the study.
Other important results:
reduction in the size of the spleen (an organ in the abdomen which is enlarge in people with myelofibrosis)
reduction in “bone marrow fibrosis” (an important feature of myelofibrosis) after 24 weeks of treatment
how long, on average, patients live after starting the study drug
how the study drug is absorbed, distributed, or broken down by the body.
This study is for adult men and women with myelofibrosis who have tried the routine standard of care therapy but it has not worked, or who develop unacceptable side effects with the standard therapy, or who are not suitable for treatment with the standard therapy. People who take part in the study must have bone marrow fibrosis and an enlarged spleen. They must also meet certain other criteria which are designed to ensure that people in the study are healthy enough to take part.
This study is sponsored by Sumitomo Pharma Oncology, Inc. it will take place in United States, Japan, Canada, South Korea, Taiwan, Belgium, France, Italy and United Kingdom. other countries may take part in future.REC name
North East - York Research Ethics Committee
REC reference
23/NE/0068
Date of REC Opinion
25 Aug 2023
REC opinion
Further Information Favourable Opinion