A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Part of paid clinical trials in Duarte, California.

Sponsor
Edgewood Oncology Inc.
Study ID
NCT04243785
Phase
PHASE1
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • BTX-A51 — DRUG
    Orally administered capsules available in strengths of 0.5 mg, 1.0 mg, 2.0 mg and 7 mg.
  • Azacitidine — DRUG
    Azacitidine will be administered IV or SC 75 mg/m2 QD on Days 1-7 of each 28-day cycle.

Study Details

This is an open-label, dose escalation study to evaluate the safety, toxicity, and pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The study will be done in three parts. Part 1a (Monotherapy Dose Escalation) of this study is designed to determine the dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined, it is planned that an additional 15 participants will be enrolled in Part 1b (Monotherapy Cohort Expansion) of this study for additional experience with safety and efficacy, and to determine the recommended Phase 2 dose (RP2D) which may or may not be different from the MTD. After determination of MTD and RP2D from Part 1a, Part 1c (Azacitidine Combination Dose Escalation) will enroll up to 30 participants. Continued treatment will be available under this study protocol for up to eight 28-day cycles (Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk. Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3 months for up to 2 years after their last treatment for survival status and anticancer therapy (Overall Survival Follow-up).

Key Dates

Start date
Jan 6, 2020
Status verified
Feb 2024
Primary completion
Mar 31, 2026
Completion
Mar 31, 2027

Study Design

Enrollment
80 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part 1a (Monotherapy Cohort Escalation)
    Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). The BTX-A51 starting dose for Cohort 1 is 1 mg, to be given 5 days per week (maximum weekly dose of 5 mg). Beginning with Cohort 2, doses are intended to be administered 3 days per week. Barring dose-limiting toxicity (DLT), sequential dose escalation of BTX-A51 is planned with up to a total of eight dose levels to a maximum of 21 mg (63 mg/week); on the basis of these an MTD will be identified. The numbers of participants and actual doses administered will be determined using a Bayesian optimal interval (BOIN) design to determine the DLTs and MTD of BTX-A51.
  • Experimental: Part 1b (Monotherapy Cohort Expansion)
    Dosing in this phase of the study consists of the first cycle of therapy (i.e., 28 days consisting of 3 weeks of treatment followed by 1 week with no study drug). Part 1b will continue at the MTD or the highest dose achieved in Phase 1a.
  • Experimental: Part 1c (Azacitidine Combination Dose Escalation)
    After determination of MTD and RP2D from Part 1a, combination dose escalation in Part 1c may begin. Patients with AML will receive BTX-A51 combined with azacitidine in escalating BTX-A51 dose cohorts. Dosing in this stage of the study consists of the first cycle of therapy (i.e., 28 days). The starting dose of BTX-A51 will be RP2D. Part 1c will follow a BOIN design as described for Part 1a. The numbers of patients and actual doses administered will be determined in response to DLTs a. There will be at least 3 patients per cohort.

Primary Outcome Measure

Incidence of dose-limiting toxicities (DLTs) [ Time Frame: Up to a total of eight 28-day cycles (approximately 224 days) ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
City of Hope National Medical CenterDuarteCalifornia91010
Brian Ball, MD
[email protected]
626-218-4784
Memorial Sloan-Kettering Cancer CenterNew YorkNew York10065
Eytan Stein, MD
[email protected]
646-608-3749
The University of Texas MD Anderson Cancer CenterHoustonTexas77030
Gautam Borthakur, MD
[email protected]
713-563-1586

Find similar trials in Duarte, CA

By condition
Leukemia (other)
By specialty
OncologyBlood cancer
By research site
City of Hope National Medical Center· Duarte, CAMemorial Sloan-Kettering Cancer Center· New York, NYThe University of Texas MD Anderson Cancer Center· Houston, TX

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