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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04951778
Registration number
NCT04951778
Ethics application status
Date submitted
23/06/2021
Date registered
7/07/2021
Titles & IDs
Public title
Study to Evaluate Safety and Tolerability of CC-91633 (BMS-986397) in Participants With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes
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Scientific title
A Phase 1, Open-label, Dose-finding Study of CC-91633 (BMS-986397) in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes
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Secondary ID [1]
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2020-005329-95
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Secondary ID [2]
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CC-91633-AML-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Leukemia, Myeloid, Acute
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Myelodysplastic Syndromes
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Condition category
Condition code
Cancer
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Leukaemia - Acute leukaemia
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Cancer
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Leukaemia - Chronic leukaemia
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Cancer
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Children's - Leukaemia & Lymphoma
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Blood
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Haematological diseases
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Blood
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Other blood disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - CC-91633
Experimental: Participants with R/R AML and R/R HR-MDS - Part A - Part A (Dose Escalation) of the study will enroll R/R AML (Relapsed or Refractory Acute Myeloid Leukemia) and R/R HR-MDS (Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes) participants and will evaluate the safety and tolerability of escalating doses of CC-91633, administered orally, and determine the maximum tolerated dose (MTD) or preliminary recommended Phase 2 doses (RP2D) and schedule.
Experimental: Participants with Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) - Part B (expansion part) will confirm tolerability of the selected doses and schedules and evaluate whether efficacy is in a range that warrants further clinical development for R/R AML participants.
Experimental: Participants with Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes (HR-MDS) - Part B (expansion part) will confirm tolerability of the selected doses and schedules and evaluate whether efficacy is in a range that warrants further clinical development for R/R HR-MDS participants.
Treatment: Drugs: CC-91633
Administered orally according to the assigned treatment schedule
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Maximum Tolerated Dose (MTD)
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Assessment method [1]
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Defined as the dose with highest posterior probability of the Dose-limiting toxicity (DLT) rate falling in the target interval and fulfilling escalation with overdose control (EWOC).
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Timepoint [1]
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Up to 2 years
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Primary outcome [2]
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Dose-limiting Toxicity (DLT)
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Assessment method [2]
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Defined as toxicities such as non-hematologic, confirmed Hy's law case, hematologic, or any AE toxicities meeting protocol specified DLT criteria and occurring within the DLT assessment period, unless the toxicity can clearly be determined to be due to other specified causes.
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Timepoint [2]
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Up to 42 days after first dose of study treatment in Part A
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Primary outcome [3]
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Incidence of Adverse Events (AEs)
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Assessment method [3]
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An AE is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the participant's health, including laboratory test values regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a preexisting condition) should be considered an AE.
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Timepoint [3]
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Up to 4 years
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Secondary outcome [1]
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Complete Remission Rate (CRR)
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Assessment method [1]
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Complete remission rate (CRR) is defined as the percent of participants whose best response is CRs including complete remission (CR), complete remission with partial hematologic recovery (CRh) and complete remission with incomplete hematologic recovery (CRi).
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Timepoint [1]
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Up to 4 years
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Secondary outcome [2]
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Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Minimal residual disease negative complete remission rate (CRRMRD-)
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Assessment method [2]
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Minimal residual disease negative complete remission rate is defined as the percent of participants with Minimal residual disease negative complete remission.
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Timepoint [2]
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Up to 4 years
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Secondary outcome [3]
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Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Combined Complete Remission Rate (cCRR)
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Assessment method [3]
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Combined complete remission rate (cCRR), is defined as the percent of participants whose best response is complete remission, includes minimal residual disease negative complete remission rate (CRRMRD-), morphologic complete remission, complete remission with incomplete hematologic recovery (CRi), complete remission with partial hematologic recovery (CRh).
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Timepoint [3]
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Up to 4 years
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Secondary outcome [4]
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Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Morphologic Leukemia-free State Rate (MLFSR)
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Assessment method [4]
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The Morphologic Leukemia-free State Rate is defined as the percent of participants with the best response of Morphologic Leukemia-free State.
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Timepoint [4]
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Up to 4 years
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Secondary outcome [5]
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Partial Remission Rate (PRR)
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Assessment method [5]
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Partial Remission Rate is defined as the percent of participant with the best response of Partial Remission.
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Timepoint [5]
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Up to 4 years
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Secondary outcome [6]
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Stable Disease Rate (SDR)
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Assessment method [6]
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Stable Disease Rate is defined as the percent of participants with the best response of Stable Disease.
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Timepoint [6]
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Up to 4 years
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Secondary outcome [7]
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Progression-free Survival (PFS) rate at 3 and 9 months
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Assessment method [7]
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Progression free survival rate is defined as the percent of participants with progression free for at least 3/9 months.
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Timepoint [7]
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At 3 months and 9 months of PFS
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Secondary outcome [8]
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Overall Survival (OS) rate
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Assessment method [8]
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Overall survival rate is defined as the percent of participant who have survived for at least 6/12 months.
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Timepoint [8]
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At 6 and 12 months of survival
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Secondary outcome [9]
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Overall Response Rate (ORR)
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Assessment method [9]
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Overall response rate is defined as the percent of participants whose best response is any of those composite complete response rate (cCRR) or morphologic Leukemia-free state (MLFS) or partial remission (PR) for AML and any of CR, marrow CR with HI (mCRHIR), PR, hematologic improvement (HI) for MDS.
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Timepoint [9]
0
0
Up to 4 years
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Secondary outcome [10]
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Overall Survival (OS)
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Assessment method [10]
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Overall Survival is measured as the time from the first dose of CC-91633 to death due to any cause.
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Timepoint [10]
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Up to 4 years
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Secondary outcome [11]
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Relapse-free Survival (RFS)
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Assessment method [11]
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Relapse-free survival is defined only for participants who have achieved the best response of any of CR/CRh/CRi/CRRMRD- or any of PR/MLFS/mCRHIR/HI, and is measured as the interval from the date of first achieved of any CR/CRh/Cri/CRRMRD- or any of PR/ MLFS/mCRHIR/HI to the date of disease relapse or death from any cause, whichever occurs first.
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Timepoint [11]
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0
Up to 4 years
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Secondary outcome [12]
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Progression-free Survival (PFS)
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Assessment method [12]
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Progression-Free Survival is defined as the time from the first dose of CC-91633 to the first occurrence of relapse or progression or death from any cause.
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Timepoint [12]
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Up to 4 years
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Secondary outcome [13]
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Event-free Survival (EFS)
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Assessment method [13]
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Event-free Survival is defined as the interval from the date of the first dose to an event including disease progression, treatment failure, relapse, or death from any cause, whichever occurs first.
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Timepoint [13]
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Up to 4 years
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Secondary outcome [14]
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Duration of remission/response
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Assessment method [14]
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For participants with best response of any of CR/CRh/ CRi/CRRMRD- or any of PR/MLFS/mCRHIR/HI, duration of remission/response is measured from the time when criteria for the best response of any of CR/CRh/ Cri/CRRMRD- or any of PR/ MLFS/mCRHIR/HI are first met (whichever is first recorded) until the first date at which relapse, or progressive disease is objectively documented assessment.
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Timepoint [14]
0
0
Up to 4 years
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Secondary outcome [15]
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Time to remission/response
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Assessment method [15]
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Time to onset of first remission/response is defined as the time interval from the date of first dose and the earliest date any remission/response (any CRs or PR) is observed.
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Timepoint [15]
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Up to 4 years
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Secondary outcome [16]
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Efficacy: Time to transformation to Acute Myeloid Leukemia (AML) for High-Risk Myelodysplastic Syndrome (HR-MDS)
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Assessment method [16]
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Time interval from first dose to onset date of having 20% more bone marrow (BM) or peripheral blood (PB) blasts.
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Timepoint [16]
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Up to 4 years
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Secondary outcome [17]
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CC-91633 Pharmacokinetics - Cmax
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Assessment method [17]
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Maximum plasma drug concentration.
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Timepoint [17]
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Up to 4 years
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Secondary outcome [18]
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CC-91633 Pharmacokinetics - AUC(0-T)
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Assessment method [18]
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Area under the plasma concentration-time curve from time zero to time t, where t is the time point of the last measurable concentration.
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Timepoint [18]
0
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Up to 4 years
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Secondary outcome [19]
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CC-91633 Pharmacokinetics - AUC(TAU)
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Assessment method [19]
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Area under the plasma concentration time-curve from time 0 to 24 hours postdose.
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Timepoint [19]
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Up to 4 years
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Secondary outcome [20]
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0
CC-91633 Pharmacokinetics - Tmax
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Assessment method [20]
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Time to peak (maximum) plasma concentration.
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Timepoint [20]
0
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Up to 4 years
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Secondary outcome [21]
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0
CC-91633 Pharmacokinetics - T-HALF
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Assessment method [21]
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Half-life.
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Timepoint [21]
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Up to 4 years
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Secondary outcome [22]
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CC-91633 Pharmacokinetics - CLT/F
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Assessment method [22]
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Apparent total clearance of the drug from plasma after oral administration, as appropriate.
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Timepoint [22]
0
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Up to 4 years
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Secondary outcome [23]
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0
CC-91633 Pharmacokinetics - Vz/F
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Assessment method [23]
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0
Apparent volume of distribution, as appropriate.
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Timepoint [23]
0
0
Up to 4 years
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Secondary outcome [24]
0
0
CC-2004772 Pharmacokinetics - Cmax
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Assessment method [24]
0
0
Maximum plasma drug concentration, if possible.
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Timepoint [24]
0
0
Up to 4 years
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Secondary outcome [25]
0
0
CC-2004772 Pharmacokinetics - AUC(0-T)
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Assessment method [25]
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0
Area under the plasma concentration-time curve from time zero to time t, where t is the time point of the last measurable concentration, if possible.
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Timepoint [25]
0
0
Up to 4 years
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Secondary outcome [26]
0
0
CC-2004772 Pharmacokinetics - AUC(TAU)
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Assessment method [26]
0
0
Area under the plasma concentration time-curve from time 0 to 24 hours postdose, if possible.
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Timepoint [26]
0
0
Up to 4 years
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Secondary outcome [27]
0
0
CC-2004772 Pharmacokinetics - Tmax
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Assessment method [27]
0
0
Time to peak (maximum) plasma concentration, if possible.
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Timepoint [27]
0
0
Up to 4 years
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Secondary outcome [28]
0
0
CC-2004772 Pharmacokinetics - T-HALF
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Assessment method [28]
0
0
Half-life, if possible.
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Timepoint [28]
0
0
Up to 4 years
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Secondary outcome [29]
0
0
CC-2004772 Pharmacokinetics - CLT/F
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Assessment method [29]
0
0
Apparent total clearance of the drug from plasma after oral administration, if possible.
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Timepoint [29]
0
0
Up to 4 years
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Secondary outcome [30]
0
0
CC-2004772 Pharmacokinetics - Vz/F
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Assessment method [30]
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Apparent volume of distribution, if possible.
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Timepoint [30]
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Up to 4 years
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Eligibility
Key inclusion criteria
Participants must satisfy the criteria below to be enrolled in the Dose Escalation (Part A) or the Dose Expansion (Part B) of this study.
* Participant is = 18 years of age, at the time of signing the ICF.
* Participant must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
* Participant is willing and able to adhere to the study visit schedule and other protocol requirements.
* Relapsed or refractory acute myeloid leukemia (R/R AML) and relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS) as defined by the World Health Organization (WHO) criteria who have failed or are ineligible for all available therapies which may provide clinical benefit
* Participant has Eastern Cooperative Oncology Group Performance Status of 0 to 2.
* Participants must have the following screening laboratory values:
* Total White Blood Cell count (WBC) < 25 x 109/L prior to first infusion.
* Aspartate aminotransferase (AST)/serum glutamic oxaloacetic transaminase (SGOT) and alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) = 3.0 x upper limit of normal (ULN), unless considered due to leukemic organ involvement, in which case AST and ALT can be = 5.0 x ULN.
* Uric acid = 7.5 mg/dL (446 µmol/L).
* Serum total bilirubin = 1.5 x ULN, unless considered due to Gilbert's syndrome
* Estimated serum creatinine clearance of = 60 mL/min using the Cockcroft-Gault equation. Measured creatinine clearance from a 24-hour urine collection is acceptable if clinically indicated.
* INR < 1.5 x ULN and partial thromboplastin time (PTT) < 1.5 x ULN.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
The presence of any of the following will exclude a participant from enrollment:
* Participant has any condition, including active or uncontrolled infection, or the presence of laboratory abnormalities, which places the participant at unacceptable risk if the participant were to participate in the study.
* Any other significant medical condition, laboratory abnormality, or psychiatric illness which places the participant at unacceptable risk if he/she were to participate in the study or that would prevent the participant from complying with the study.
* Participant has any condition that confounds the ability to interpret data from the study.
* Participants with acute promyelocytic leukemia.
* Participants with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia.
* Participants with immediately life-threatening, severe complications of leukemia such as disseminated/uncontrolled infection, uncontrolled bleeding, and/or uncontrolled disseminated intravascular coagulation.
* Participants with impaired cardiac function or clinically significant cardiac diseases,
* Participants who have undergone major surgery = 2 weeks prior to starting CC-91633. Participants must have recovered from any clinically significant effects of recent surgery.
* Pregnant or nursing individuals.
* Participants with known human immunodeficiency virus infection.
* Participants with known chronic, active hepatitis B virus or hepatitis C virus C (HCV) infection.
* Participants with ongoing treatment with chronic, therapeutic dosing of anticoagulants (eg, warfarin, low molecular weight heparin, Factor Xa inhibitors).
* Participants with history of concurrent second cancers requiring active, ongoing systemic treatment
* Participants with clinically significant diarrhea, vomiting or malabsorption felt to limit absorption of orally administered medications.
* Participants with known or suspected hypersensitivity to any of the components or excipients of the study treatment or to similar class drugs (eg, lenalidomide).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
2/12/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
5/05/2027
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Actual
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Sample size
Target
180
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA,VIC
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Recruitment hospital [1]
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Local Institution - 604 - Adelaide
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Recruitment hospital [2]
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Royal Adelaide Hospital - Adelaide
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Recruitment hospital [3]
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Monash Health - Clayton
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Recruitment hospital [4]
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The Alfred Hospital - Melbourne
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Recruitment hospital [5]
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Local Institution - 603 - Melbourne
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Recruitment hospital [6]
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St Vincent's Hospital - Melbourne
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment postcode(s) [3]
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3004 - Melbourne
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Recruitment postcode(s) [4]
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3065 - Melbourne
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
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0
Arizona
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Country [2]
0
0
United States of America
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State/province [2]
0
0
California
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Illinois
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Massachusetts
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Missouri
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Country [6]
0
0
United States of America
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State/province [6]
0
0
Ohio
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Country [7]
0
0
United States of America
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State/province [7]
0
0
Pennsylvania
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Country [8]
0
0
United States of America
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State/province [8]
0
0
Texas
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Country [9]
0
0
United States of America
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State/province [9]
0
0
Washington
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Country [10]
0
0
Canada
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State/province [10]
0
0
British Columbia
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Country [11]
0
0
Canada
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State/province [11]
0
0
Ontario
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Country [12]
0
0
Spain
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State/province [12]
0
0
Barcelona
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Country [13]
0
0
Spain
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State/province [13]
0
0
Madrid
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Country [14]
0
0
Spain
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State/province [14]
0
0
Sevilla
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Country [15]
0
0
United Kingdom
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State/province [15]
0
0
Greater Manchester
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Country [16]
0
0
United Kingdom
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State/province [16]
0
0
London, City Of
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Country [17]
0
0
United Kingdom
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State/province [17]
0
0
Leeds
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Country [18]
0
0
United Kingdom
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State/province [18]
0
0
London
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Country [19]
0
0
United Kingdom
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State/province [19]
0
0
Oxford
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Celgene
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Study CC-91633-AML-001 is a Phase 1, open-label, dose escalation and expansion, first-in-human (FIH) clinical study of CC-91633 (BMS-986397) in participants with relapsed or refractory acute myeloid leukemia (R/R AML) or in participants with relapsed or refractory higher-risk myelodysplastic syndromes (R/R HR-MDS). The Dose Escalation part (Part A) of the study will enroll participants with R/R AML and R/R HR-MDS and will evaluate the safety and tolerability of escalating doses of CC-91633 (BMS-986397), administered orally, and determine the maximum tolerated dose (MTD) or preliminary recommended Phase 2 doses (RP2D) and schedule. Throughout the study, final decisions on dose escalation/de-escalation will be made by the safety review committee (SRC). Approximately 60 participants may be enrolled in Part A of the study. The expansion part (Part B) will confirm tolerability of the selected doses and schedules and evaluate whether efficacy is in a range that warrants further clinical development. Approximately 60 response-evaluable subjects per indication (R/R AML or R/R HR-MDS) may be enrolled. Parts A and B will consist of 3 periods: Screening, Treatment, and Follow-up.
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Trial website
https://clinicaltrials.gov/study/NCT04951778
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Bristol-Myers Squibb
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Address
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Bristol-Myers Squibb
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Country
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0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
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0
BMS Study Connect Contact Center www.BMSStudyConnect.com
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Address
0
0
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Country
0
0
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Phone
0
0
855-907-3286
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Fax
0
0
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Email
0
0
[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Information relating to our policy on data sharing and the process for requesting data can be found at the following link:
https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/
Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR), Analytic code
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When will data be available (start and end dates)?
See Plan Description
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Available to whom?
See Plan Description
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04951778