A Clinical Trial of BP1002 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorBio-Path Holdings, Inc.
About this trial
This study evaluates the safety and tolerability of escalating doses of BP1002 (Liposomal Bcl-2 Antisense Oligodeoxynucleotide) in patients with refractory/relapsed AML. The study is designed to assess the safety profile, identify DLTs, biologically effective doses, PK, PD and potential anti-leukemic effects of BP1002 as single agent (dose escalation phase) followed by assessing BP1002 in combination with decitabine (dose expansion phase).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Adults ≥18 years of age, with histologic evidence of refractory/relapsed AML who have failed treatment with available therapies known to be active for refractory/relapsed AML
Eastern Cooperative Oncology Group (ECOG) Performance Status Score of 0, 1 or 2
For the dose expansion phase, participants with documented diagnosis of AML who are eligible for decitabine therapy
Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 times the upper limit of normal (ULN); and
Disqualifiers
Active non-hematologic or lymphoid malignancy other than AML treated with immunotherapy, targeted therapy or chemotherapy within the previous 12 months
Known, active leptomeningeal leukemia requiring intrathecal therapy. NOTE: Participants with a history of CNS disease may be allowed to participate based on at least 1 documented, negative spinal fluid assessment within 28 days prior to Screening
Isolated potentially treatable extramedullary leukemia without also meeting bone marrow criteria for acute leukemia (for AML usually ≥ 5% blasts in BMA or biopsy). Participants may have leukemia with lower blast counts (Döhner 2017). Bio-Path Holdings and Investigator concurrence required.
Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12) PML-RARA
Trial design
Design model
Sequential
Treatments tested in this trial
BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide
DrugDose escalation of BP1002 monotherapy
Decitabine (in combination with BP1002)
DrugDose expansion of BP1002 in combination with decitabine
Treatment groups
48 Participants
are divided into 2 treatment groupsGroup A: Relapsed/Refractory AML - BP1002 monotherapyExperimental treatment 1 intervention
Group B: Relapsed/Refractory AML - BP1002 in combination with decitabineExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
Identify Dose Limiting Toxicity (DLT) of BP1002
Identify DLT of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
Time frame
30 days2
Identify and grade treatment-emergent adverse events (TEAE) of escalating doses of BP1002
Identify TEAE of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
Time frame
30 days3
Identify and grade treatment-emergent laboratory abnormalities of escalating doses of BP1002
Identify and grade treatment-emergent laboratory abnormalities of escalating doses of BP1002 using non-hematologic and hematologic measure per NCI CTCAE criteria
Time frame
30 days4
Recommended Phase 2 (RP2D) of BP1002
Determine RP2D by evaluating Maximally Tolerated Dose (MTD) data
Time frame
210 daysSecondary outcomes
1
Determine evidence of response by bone marrow aspirate
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
Time frame
180 days2
Determine evidence of response by complete blood counts using peripheral blood
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
Time frame
180 days3
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate and complete blood counts
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
Time frame
180 days4
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
Time frame
180 daysOther outcomes
1
Exploratory objective to correlate treatment response with cytogenetic characteristics
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
Time frame
30 days2
Exploratory objective to correlate treatment response with molecular characteristics
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
Time frame
30 daysSponsors and contacts
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Source ClinicalTrials.gov