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Ara-C

Phase 2

Acute Myeloid Leukemia | Small molecule | Oncology |BioLineRx Ltd.|Last Updated: Sep 19, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMC
Total Trials1
Total Enrollment42
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01838395Phase IIa Study Evaluating Safety and Efficacy of BL-8040 in Relapsed/Refractory AML PatientsPHASE2 COMPLETED 42Apr 1, 2013Jul 20, 2023Sep 19, 202410 United States, Israel
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Study Endpoints
Primary Endpoints
Safety and Tolerability
Participants were followed for the duration of the hospital stay and the follow-up period, an expected average of 6 weeks.

Number of participants with Adverse event affecting the safety and tolerability of BL-8040 + Ara-C by dose level (overall, dose limiting events, related Adverse Events (AEs), Serious Adverse Events (SAEs), related SAEs, AE by severity and death) Toxicity grade was assessed according to version V4.03 of NCI-CTCAE

Secondary Endpoints
Response to Treatment by Dose
Final bone marrow evaluation - Between Day 20 and Day 44
Apoptotic Effect
Final evaluation - between Day 20 and Day 44
Assessment of the Pharmacokinetic Profile of BL-8040 - t1/2 (h)
Blood samples for the determination of BL-8040 were collected before dosing and at 0.25, 0.5, 1, 2, 4, 8, and 24 hours after BL-8040 administration on Day 1.
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BL-8040 0.5mg/kg + Ara-C 1.5 or 3 g/m2/d per dose (based on age)EXPERIMENTALArm 1: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 0.5 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
BL-8040 0.75mg/kg + Ara-C 1.5 or 3 g/m2/d (based on age)EXPERIMENTALArm 2: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 0.75 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
BL-8040 1.0mg/kg + Ara-C 1.5 or 3 g/m2/d (based on age)EXPERIMENTALArm 3: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 1 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
BL-8040 1.25mg/kg + Ara-C 1.5 or 3 g/m2/d (based on age)EXPERIMENTALArm 4: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 1.25 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
BL-8040 1.5mg/kg + Ara-C 1.5 or 3 g/m2/d (based on age)EXPERIMENTALArm 5: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 1.5 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
BL-8040 2mg/kg + Ara-C 1.5 or 3 g/m2/d (based on age)EXPERIMENTALArm 6: Participants will be dosed with SC injections of BL-8040 over two days followed by concurrent administration of 2 mg/kg BL-8040 with cytarabine (Ara-C) 1.5 or 3 g/m2/d per dose (based on age) during 5 days.
Interventions
NameTypeDescription
Ara-CDRUGIV (intravenous administration)
BL-8040DRUGSC (subcutaneous injection)
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: 1. Adult men and women subjects aged 18 to 75, inclusive. 2. Confirmed diagnosis of relapsed/refractory AML (WHO criteria) Refractory subjects, up to second consecutive salvage . Relapsed subjects including first and second relapse. 3. AML relapse \> 6 months since autologous or...

Countries:United StatesIsrael
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