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RO5503781 MBP

Phase 1

Myelogenous Leukemia, Acute | Small molecule | Oncology |Roche Holding AG|Last Updated: Jan 25, 2017

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment122

FDA Designations

No designations recorded

Clinical trial landscape

RO5503781 MBP · 1 trial · 1 indication

Phase 1 1
NCT01773408A Study of RO5503781 as a Single Agent or in Combination With Cytarabine in Participants With Acute Myelogenous LeukemiaMyelogenous Leukemia, Acute
COMPLETED122 Analytics
PHASE1COMPLETED
A Study of RO5503781 as a Single Agent or in Combination With Cytarabine in Participants With Acute Myelogenous Leukemia
Myelogenous Leukemia, AcuteUnlock trial analytics

Study Endpoints

Primary Endpoints

Maximum Tolerated Dose (MTD) and/ or Recommended Phase 2 Dose (RP2D) of RO5503781
Day 1 up to Day 42
Proportion of Participants with Dose-Limiting Toxicities (DLTs)
Day 1 up to Day 42
Safety: Proportion of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline up to approximately 2 years

Secondary Endpoints

Pharmacokinetics: Maximum Observed Plasma Concentration (Cmax) (in Micrograms per Milliliters [mcg/mL]) of RO5503781, Cytarabine,1-beta-D-Arabinofuranosyluracil, Daunorubicin, Daunorubicinol, Idarubicin, and Idarubicinol
Predose (0 Hr) on Cycle (Cy) 1 Day 1 up to Cy 1 Day 15 (Cy length=28 days) (detailed timeframe for individual drug is provided in outcome measure description)
Pharmacokinetics: Time to Reach Maximum Plasma Concentrations (Tmax) in hours of RO5503781, Cytarabine, 1-beta-D-Arabinofuranosyluracil, Daunorubicin, Daunorubicinol, Idarubicin, and Idarubicinol
Predose (0 Hr) on Cy1 Day 1 up to Cy 1 Day 15 (Cy length=28 days) (detailed timeframe for individual drug is provided in outcome measure description).
Pharmacokinetics: Terminal Half-Life (t1/2) (in hours) of RO5503781, Cytarabine, 1-beta-D-Arabinofuranosyluracil, Daunorubicin, Daunorubicinol, Idarubicin, and Idarubicinol
Predose (0 Hr) on Cy1 Day 1 up to Cy 1 Day 15 (Cy length=28 days) (detailed timeframe for individual drug is provided in outcome measure description)
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1: RO5503781EXPERIMENTALParticipants will receive RO5503781 alone in escalating doses on Days 1 to 5 of each 28-day cycle until disease progression or unacceptable toxicity.
Part 2: RO5503781 + CytarabineEXPERIMENTALParticipants will receive RO5503781 in escalating doses on Days 1 to 5 and cytarabine on Days 1 to 6 of each 28-day cycle until disease progression or unacceptable toxicity.
Part3:RO5503781+Cytarabine+AnthracyclineEXPERIMENTALParticipants will receive RO5503781 on Days 1 to 5, cytarabine on Days 1 to 7, and anthracycline (daunorubicin or idarubicin) on Days 1 to 3 of each 28-day cycle until disease progression or unacceptable toxicity.
Part 4: Optimized RO5503781 + CytarabineEXPERIMENTALParticipants will receive optimized RO5503781 formulation on Days 1 to 5 and cytarabine on Days 1 to 6 of each 28-day cycle until disease progression or unacceptable toxicity.

Interventions

NameTypeDescription
RO5503781 MBPDRUGParticipants will receive RO5503781 tablets daily (current formulation) containing microprecipitated bulk powder (MBP) at a starting dose of 400 milligrams (mg).
RO5503781 SDPDRUGParticipants will receive RO5503781 tablets daily (new optimized formulation) containing spray-dried powder (SDP) at recommended dose(s) for development from Phase 1b to Phase 3.
IdarubicinDRUGIdarubicin will be administered as per standard clinical practice.
DaunorubicinDRUGDaunorubicin will be administered as per standard clinical practice.
CytarabineDRUGParts 2 and 4: Participants will receive cytarabine 1000 milligrams per meter squared (mg/m\^2) intravenous (IV) infusion daily. Part 3: Participants will receive cytarabine 100-200 mg/m\^2 IV infusion daily.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: * Documented/confirmed acute myelogenous leukemia (AML), except for acute promyelocytic leukemia * Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 in Part 1 and Part 2, participants enrolled in the extension/tail portion, Part 3 and Part 4 must have an ECOG p...

Countries:United StatesAustraliaCanadaFranceItalySouth KoreaUnited Kingdom
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Frequently asked questions about RO5503781 MBP

What is RO5503781 MBP used for?

RO5503781 MBP is an investigational small molecule being studied for the treatment of acute myelogenous leukemia (AML). It is being evaluated as a single agent or in combination with cytarabine in patients with this condition. The drug is currently in Phase 1 clinical development.

Who makes RO5503781 MBP?

RO5503781 MBP is being developed by Roche Holding AG, which trades under the ticker RHHBY. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with acute myelogenous leukemia.

What phase is RO5503781 MBP in?

RO5503781 MBP is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 study of the drug has been completed, with results expected to inform further development.

What clinical trials is RO5503781 MBP in?

RO5503781 MBP has been studied in one completed Phase 1 clinical trial, identified as NCT01773408. This trial evaluated the drug as a single agent or in combination with cytarabine in 122 participants with acute myelogenous leukemia across multiple countries.

Is RO5503781 MBP the same as RO5503781?

RO5503781 MBP is a formulation of the investigational drug RO5503781. The designation MBP indicates a specific formulation or manufacturing process. In clinical trial records, the drug is referred to as RO5503781, and the MBP suffix is used to distinguish this particular version.