In short
Clinical trials investigating Treosulfan are studying how it is used in cancer and transplant settings, mainly in blood cancers and Ewing sarcoma. These trials look at safety, effectiveness, survival, relapse, and transplant outcomes in children, adolescents, and adults.
Key points
- Clinical trials with Treosulfan are mainly studying blood cancers and stem cell transplantation, with one study in Ewing sarcoma. Most trials are Phase 2 or Phase 3, and they focus on safety, survival, relapse, and transplant-related outcomes. Several studies test Treosulfan as part of conditioning before allogeneic stem cell transplantation, especially in AML, MDS, myelofibrosis, and ALL. Some trials compare Treosulfan with other conditioning treatments or use it in combination with other drugs. The target groups include children, adolescents, adults, and older patients, depending on the study. Overall, the trial data show that Treosulfan is being researched in both cancer treatment and transplant support settings.
Overview of Treosulfan trials
The trial data show that Treosulfan is being studied in interventional clinical trials, which means researchers give a treatment and then measure what happens. Most studies are in the setting of cancer care, especially before or around allogeneic stem cell transplantation, where stem cells come from another person. One trial studies Treosulfan in Ewing Sarcoma, while many others focus on blood cancers such as AML, MDS, ALL, CLL, and myelofibrosis.
Several studies compare Treosulfan-based treatment with another conditioning plan, while others test it as part of a combination regimen. The main goal is to learn whether these plans are safe, feasible, and effective for the target patient groups.
Conditions being studied
The trials cover a wide range of diseases, but most are in blood cancers and transplant-related care. The main conditions include acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), chronic myelomonocytic leukemia (CMML), myelofibrosis, and secondary AML.
One study is in children with autosomal recessive osteopetrosis caused by TCIRG1 gene mutations, which is a rare inherited bone disease. Another study focuses on newly diagnosed high-risk and very high-risk Ewing Sarcoma, a cancer that usually starts in bone or soft tissue.
Trial designs and phases
Most Treosulfan studies are in Phase 2 or Phase 3, which are later trial stages used to study effectiveness and safety in larger groups. There is also a Phase 1 study that mainly looks at safety and the best way to combine treatments, and a Phase 1/2 study in children with osteopetrosis.
Some trials are randomized, meaning patients are assigned to one treatment group or another by chance. Others are open-label, which means both the researchers and the participants know which treatment is being used. One large Phase 4 platform trial includes several linked sub-studies in children and young adults with acute lymphoblastic leukemia.
Who can take part
Eligibility depends on the disease and the treatment plan in each trial. Some studies include children and adolescents, such as the Ewing Sarcoma trial, the osteopetrosis study, and the ALL transplant trial.
Other studies are for adults, including people aged 40 to under 65 years with AML, elderly patients with AML or MDS, and adults with high-risk MDS, CMML, or secondary AML before transplant. Some trials require a planned allogeneic hematopoietic cell transplant or a specific disease status such as first or second complete remission, which means the cancer is under control after treatment.
Main outcomes and endpoints
The trials measure different outcomes depending on the study question. Common endpoints include overall survival, event-free survival, and leukemia-free survival, which show how long patients live or stay free from major problems after treatment.
Several studies also measure transplant-related outcomes such as graft-versus-host disease, rejection, relapse, graft failure, and second cancers. Safety is often measured by the number and severity of adverse effects and serious adverse effects, sometimes using CTCAE grading, which is a standard way to rate side effects in trials.
Some studies use more specific endpoints, such as minimal residual disease negativity in AML, undetectable MRD in CLL, or disease- and rejection-free survival after transplant. The Ewing Sarcoma study measures both safety and 36-month event-free survival.
Key trials at a glance
- Ewing Sarcoma study — a Phase 2 multicenter trial in 60 patients, testing Treosulfan/Melphalan as consolidation treatment and measuring adverse effects and 36-month event-free survival.
- RELEVANT — a Phase 2 randomized study in AML and MDS transplant patients, comparing Treosulfan with melphalan conditioning and measuring overall survival.
- Myelofibrosis haplo-identical transplant trial — a Phase 2 study in 28 patients, looking at disease- and rejection-free survival 12 months after transplant.
- AML and MDS GVHD prevention study — a Phase 3 trial in 324 patients, comparing two graft-versus-host disease prevention strategies after fludarabine-Treosulfan conditioning.
- ALL FORUM trial — a large Phase 4 study in children and young people with ALL, with Treosulfan as one of the conditioning options and overall survival or event-free survival as key outcomes.