TerminatedPhase 1

A Study of TAS1553 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms

Stopped: termination due to portfolio prioritization

United States20 participantsStarted 2020-12-21

Plain-language summary

This is a Phase 1, 2-part, open-label, multicenter, first-in-human (FIH) study to assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of TAS1553 administered orally to participants ≥18 years of age with relapsed or refractory (R/R) acute myeloid leukemia (AML) or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and myelodysplastic syndrome (MDS)-transformed into AML. Other myeloid neoplasms include accelerated phase myeloproliferative neoplasms (MPN), and chronic or accelerated phase MPN-unclassifiable (MPN-U) and MDS-MPN. Blast crisis phase of MPNs are considered secondary AML and will be included in the AML cohort. Part 1 is a multicenter, sequential group treatment feasibility study with 1 treatment arm and no masking (dose escalation). Part 2 is a multicenter, two-stage, multiple group, dose confirmation study with 1 treatment arm and no masking (exploratory dose expansion).

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Capable of giving signed informed consent.
✓. Participant must be 18 years of age or older, at the time of signing the informed consent.
✓. Life expectancy of at least 12 weeks as assessed by the investigator.
✓. Participants with R/R AML or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and MDS transformed into AML. Other myeloid neoplasms include accelerated phase MPN, and chronic or accelerated phase MPN-U and MDS-MPN. Blast crisis phase of MPN, MPN-U, and MDS-MPN are considered secondary AML and will be included in the AML cohort.
✓. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
✓. Have platelet count ≥10,000/μL (transfusions to achieve this level are allowed).
✓. Have adequate renal function as demonstrated by a 24-hour urine measured creatinine clearance ≥60 mL/min.
✓. Adequate hepatic function as evidenced by:

Exclusion criteria

What they're measuring

Safety: Number of participants with treatment-emergent adverse events in Part 1

Timeframe: Up to 12 months

Safety: Number of participants with dose-limiting toxicities in Part 1

Timeframe: Up to 12 months

Response rate in Cohort 1 (AML): Number of participants with complete response (CR) + complete response with partial hematological recovery (CRh), and with CR + incomplete blood count recovery (CRi) in Part 2

Timeframe: Up to 33 months

Response rate in Cohort 2 (other myeloid neoplasms): Number of participants with overall response rate (ORR) of CR + partial response (PR) in Part 2

Timeframe: Up to 33 months

Trial details

NCT IDNCT04637009

SponsorAstex Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-02-20

View on ClinicalTrials.gov More Acute Myeloid Leukemia trials

Plain-language summary

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Capable of giving signed informed consent.
✓. Participant must be 18 years of age or older, at the time of signing the informed consent.
✓. Life expectancy of at least 12 weeks as assessed by the investigator.
✓. Participants with R/R AML or other myeloid neoplasms where approved therapies have failed or for whom known life-prolonging therapies are not available. The AML population includes de novo AML, secondary AML, and MDS transformed into AML. Other myeloid neoplasms include accelerated phase MPN, and chronic or accelerated phase MPN-U and MDS-MPN. Blast crisis phase of MPN, MPN-U, and MDS-MPN are considered secondary AML and will be included in the AML cohort.
✓. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
✓. Have platelet count ≥10,000/μL (transfusions to achieve this level are allowed).
✓. Have adequate renal function as demonstrated by a 24-hour urine measured creatinine clearance ≥60 mL/min.
✓. Adequate hepatic function as evidenced by:

Exclusion criteria

What they're measuring

Safety: Number of participants with treatment-emergent adverse events in Part 1

Timeframe: Up to 12 months

Safety: Number of participants with dose-limiting toxicities in Part 1

Timeframe: Up to 12 months

Timeframe: Up to 33 months

Response rate in Cohort 2 (other myeloid neoplasms): Number of participants with overall response rate (ORR) of CR + partial response (PR) in Part 2

Timeframe: Up to 33 months