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New treatment option for High-risk Myelodysplastic Syndrome
Official title A Study of CBX-250 in Participants With Relapsed or Refractory Myeloid Leukemias
ClinicalTrials.gov ID: NCT06994676
What this study is testing
What is CBX-250?
CBX-250 is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for high-risk myelodysplastic syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Study CBX-250-001 is a Phase 1, open-label, dose-escalation study of CBX-250 in participants with relapsed/refractory AML, HR-MDS, CMML, and CML. Participants aged ≥ 12 years are planned to be enrolled.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Participants are eligible to be included in the study only if all of the following criteria apply: Age
- Dose Escalation: Male or female participants aged ≥18 years.
- Backfill Cohorts: Male or female participants aged ≥12 years for whom no curative treatment options, including transplantation, are available...
- Participants with histological confirmation of advanced hematologic malignancy including:
- R/R AML, as defined by standardized criteria (e.g., European LeukemiaNet criteria [Dohner 2022]; after standard of care therapy. Participants with...
You likely can't join if
- Participants are excluded from the study if any of the following criteria apply: Diagnosis
- Previous CTSG targeted therapy or treatment with any pMHC T-cell engager.
- Isolated extramedullary relapse.
- Active central nervous system (CNS) disease. Participants with prior CNS history can be enrolled if the participant has a negative lumbar puncture...
- Known HIV infection.
- Active hepatitis B infection (participants with documented clearance following treatment are allowed).
See the full eligibility criteria
Who can join
- Participants are eligible to be included in the study only if all of the following criteria apply: Age
- Dose Escalation: Male or female participants aged ≥18 years.
- Backfill Cohorts: Male or female participants aged ≥12 years for whom no curative treatment options, including transplantation, are available. Diagnosis \& Disease Characteristics
- Participants with histological confirmation of advanced hematologic malignancy including:
- R/R AML, as defined by standardized criteria (e.g., European LeukemiaNet criteria [Dohner 2022]; after standard of care therapy. Participants with persistent leukemia after initial therapy or with recurrence of leukemia...
- R/R HR-MDS or very high risk MDS as per the Revised International Prognostic Scoring System (IPSS-R; Greenberg 2012) or Molecular International Prognostic Scoring System (IPPS-M, Bernard 2022) who are resistant or...
- R/R CMML who are resistant or refractory to 4-6 cycles of hypomethylating agents (HMA; decitabine or azacitidine).
- White blood cells must be below 25,000/µL at time of enrollment. Participants may receive cytoreduction prior to enrollment.
- Historical documented evidence of HLA-A\ 02:01 allele positivity. Performance Level
- ECOG PS score 0-1 (if aged ≥18 years); Karnofsky Performance Scale of ≥70 (if aged ≥16 years and \<18 years); Lansky PS of ≥70 (if aged \<16 years). Prior Therapy
- Any prior treatment-related toxicities resolved to ≤Grade 1 prior to enrollment, with the exception of ≤Grade 2 alopecia.
- Radiation Therapy: At least 60 days from prior total body irradiation, craniospinal radiation and/or ≥50% radiation of the pelvis, or at least 14 days from local palliative radiation therapy (small port).
- Stem Cell Infusion: At least 60 days must have elapsed from HSCT and at least 4 weeks (from first dose) must have elapsed from donor lymphocyte infusion without conditioning.
- Immunotherapy: At least 42 days since prior immunotherapy, including tumor vaccines and checkpoint inhibitors, and at least 21 days since receipt of chimeric antigen receptor therapy.
- Anti-Leukemia Therapy: At least 14 days since the completion of anti-leukemic therapy (for example, but not limited to, small molecule or cytotoxic/myelosuppressive therapy), with the following exceptions:
- Hydroxyurea for cytoreduction can be initiated without restriction related to timing of study entry. Hydroxyurea for cytoreduction can be continued concomitantly with CBX-250, with Study Responsible Physician approval.
- Intrathecal chemotherapy at the time of diagnostic lumbar puncture at least 24 hours prior to the start of CBX-250 and may continue prophylactic intrathecal chemotherapy beginning in Cycle 2, at the treating physician's...
- Hematopoietic Growth Factors: At least 7 days since the completion of therapy with short-acting hematopoietic growth factors and 14 days with long-acting growth factors.
- Biologics (e.g. monoclonal antibody therapy): At least 28 days or 5 half-lives, whichever is shorter, have elapsed since the completion of therapy with a biologic agent. Any AE related to prior biologic treatment must...
- Steroids: At least 7 days since systemic glucocorticoid therapy, unless receiving physiologic dosing (equivalent to ≤10 mg prednisone daily) or cytoreductive therapy. Cytoreductive therapy must have approval of the...
- Estimated glomerular filtration rate ≥ 45 mL/min/1.73 m2 based on local institutional practice for age-appropriate determination (eg, Schwartz formula for pediatric participants or Cockcroft-Gault formula for adults).
- Participants ≥18 years: glomerular filtration rate ≥45 mL/min
- Participants \<18 years: ≥45 mL/min x (participant's body surface area m2/1.73) Adequate liver function defined as:
- Total bilirubin \<1.5 × the upper limit of normal (ULN) for age or normal conjugated bilirubin, or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range in participants with well documented Gilbert's...
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \<3 × ULN (unless attributed to leukemic involvement with discussion with the Study Responsible Physician). Sex and Contraceptive/Barrier Requirements
- If a female of childbearing potential, willing to use a highly effective method of contraception or double barrier method from the time of enrollment through 120 days following the last study drug dose.
- If male of childbearing potential, agrees to use barrier contraception from the time of enrollment through 120 days following the last study drug dose. Informed Consent
- Participant or participant's health care proxy is able and willing to provide written informed consent and able to follow study instructions.
What rules you out
- Participants are excluded from the study if any of the following criteria apply: Diagnosis
- Previous CTSG targeted therapy or treatment with any pMHC T-cell engager.
- Isolated extramedullary relapse.
- Active central nervous system (CNS) disease. Participants with prior CNS history can be enrolled if the participant has a negative lumbar puncture following completion of intrathecal chemotherapy). Infection
- Known HIV infection.
- Active hepatitis B infection (participants with documented clearance following treatment are allowed).
- Active hepatitis C infection (participants with documented clearance following treatment are allowed). Pregnancy and Breastfeeding
- Pregnant or nursing women: Negative serum pregnancy tests are required during Screening and a negative serum or urine pregnancy test is required within 72 hours prior to receiving the first study drug administration, in...
- Cardiac Disease:
- Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class \>II), life-threatening...
- QTc using Fridericia's correction (QTcF) \>480 msec
- Graft-Versus-Host Disease (GVHD): Active acute or chronic GVHD requiring systemic treatment with immunosuppressive medication. Participants may be on physiological doses of steroids.
- Concurrent malignancy in the previous 2 years with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (eg, breast carcinoma, cervical cancer in situ, melanoma in...
- History of or any concurrent condition, therapy, or laboratory abnormality that in the Investigator's opinion might confound the results of the study, interfere with the participant's participation for the full duration...
- Any commercially available or investigational anti-leukemic therapy other than CBX-250, with the following exceptions:
- Intrathecal chemotherapy for CNS prophylaxis is permitted after C1 is complete, at the treating physician's discretion.
- Participation in another therapeutic treatment clinical study in which an investigational agent was administered within 14 days or 5-halflives, whichever is shorter, of starting CBX-250. Participants may continue with...
- Any concurrent systemic treatment to prevent GVHD. Topical treatments for GVHD are permitted.
- Known allergy or sensitivity to study drug, including excipients.
The study team makes the final eligibility decision.
Where it's taking place
- Duarte, California, United States
- Palo Alto, California, United States
- Tampa, Florida, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- St Louis, Missouri, United States
- New York, New York, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Duarte, California, United States; Palo Alto, California, United States; Tampa, Florida, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States; St Louis, Missouri, United States and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.