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Leukemia, Myeloid, AcutePhase 1NCT04951778

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

A Phase 1 study testing CC-91633 in people with Leukemia, Myeloid, Acute and Myelodysplastic Syndromes, run by Celgene at 9 sites in 2 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CC-91633
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
56 participants
Where
9 sitesin 2 countries
Study dates
2 Dec 2021to 30 Jul 2025
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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).

Trial locations and contacts

9 sites in 2 countries.

9 sites in 2 countries

United States5 sites

  • Local Institution - 107

    Boston, Massachusetts, 02114

  • Local Institution - 101

    Boston, Massachusetts, 02215

  • Local Institution - 104

    Houston, Texas, 77030

  • Local Institution - 109

    Seattle, Washington, 98104

  • Local Institution - 105

    St Louis, Missouri, 63110

Other trials in United States

Spain4 sites

  • Local Institution - 302

    Barcelona, 08035

  • Local Institution - 301

    Barcelona, 08036

  • Local Institution - 303

    Madrid, 28041

  • Local Institution - 304

    Seville, 41013

Other trials in Spain

About this study

Official 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

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.

Being tested
CC-91633
Keywords
Relapsed or Refractory Acute Myeloid Leukemia, Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CC-91633Drug

    Also called BMS-986397

    Administered orally according to the assigned treatment schedule

Study groups in NCT04951778
Study groupRoleWhat they receive
Participants with R/R AML and R/R HR-MDS - Part AExperimentalPart 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.
Receives: Drug: CC-91633
Participants with Relapsed or Refractory Acute Myeloid Leukemia (R/R AML)ExperimentalPart 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.
Receives: Drug: CC-91633
Participants with Relapsed or Refractory Higher-Risk Myelodysplastic Syndromes (HR-MDS)ExperimentalPart 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.
Receives: Drug: CC-91633

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Tolerated Dose (MTD)

    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).

    Measured: Up to 2 years

  2. Dose-limiting Toxicity (DLT)

    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.

    Measured: Up to 42 days after first dose of study treatment in Part A

  3. Incidence of Adverse Events (AEs)

    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.

    Measured: Up to 4 years

Secondary outcomes (30)

  1. Complete Remission Rate (CRR)

    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).

    Measured: Up to 4 years

  2. Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Minimal residual disease negative complete remission rate (CRRMRD-)

    Minimal residual disease negative complete remission rate is defined as the percent of participants with Minimal residual disease negative complete remission.

    Measured: Up to 4 years

  3. Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Combined Complete Remission Rate (cCRR)

    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).

    Measured: Up to 4 years

  4. Efficacy determined by response rates of Acute Myeloid Leukemia (AML) - Morphologic Leukemia-free State Rate (MLFSR)

    The Morphologic Leukemia-free State Rate is defined as the percent of participants with the best response of Morphologic Leukemia-free State.

    Measured: Up to 4 years

  5. Partial Remission Rate (PRR)

    Partial Remission Rate is defined as the percent of participant with the best response of Partial Remission.

    Measured: Up to 4 years

  6. Stable Disease Rate (SDR)

    Stable Disease Rate is defined as the percent of participants with the best response of Stable Disease.

    Measured: Up to 4 years

  7. Progression-free Survival (PFS) rate at 3 and 9 months

    Progression free survival rate is defined as the percent of participants with progression free for at least 3/9 months.

    Measured: At 3 months and 9 months of PFS

  8. Overall Survival (OS) rate

    Overall survival rate is defined as the percent of participant who have survived for at least 6/12 months.

    Measured: At 6 and 12 months of survival

  9. Overall Response Rate (ORR)

    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.

    Measured: Up to 4 years

  10. Overall Survival (OS)

    Overall Survival is measured as the time from the first dose of CC-91633 to death due to any cause.

    Measured: Up to 4 years

  11. Relapse-free Survival (RFS)

    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.

    Measured: Up to 4 years

  12. Progression-free Survival (PFS)

    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.

    Measured: Up to 4 years

  13. Event-free Survival (EFS)

    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.

    Measured: Up to 4 years

  14. Duration of remission/response

    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.

    Measured: Up to 4 years

  15. Time to remission/response

    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.

    Measured: Up to 4 years

  16. Efficacy: Time to transformation to Acute Myeloid Leukemia (AML) for High-Risk Myelodysplastic Syndrome (HR-MDS)

    Time interval from first dose to onset date of having 20% more bone marrow (BM) or peripheral blood (PB) blasts.

    Measured: Up to 4 years

  17. CC-91633 Pharmacokinetics - Cmax

    Maximum plasma drug concentration.

    Measured: Up to 4 years

  18. CC-91633 Pharmacokinetics - AUC(0-T)

    Area under the plasma concentration-time curve from time zero to time t, where t is the time point of the last measurable concentration.

    Measured: Up to 4 years

  19. CC-91633 Pharmacokinetics - AUC(TAU)

    Area under the plasma concentration time-curve from time 0 to 24 hours postdose.

    Measured: Up to 4 years

  20. CC-91633 Pharmacokinetics - Tmax

    Time to peak (maximum) plasma concentration.

    Measured: Up to 4 years

  21. CC-91633 Pharmacokinetics - T-HALF

    Half-life.

    Measured: Up to 4 years

  22. CC-91633 Pharmacokinetics - CLT/F

    Apparent total clearance of the drug from plasma after oral administration, as appropriate.

    Measured: Up to 4 years

  23. CC-91633 Pharmacokinetics - Vz/F

    Apparent volume of distribution, as appropriate.

    Measured: Up to 4 years

  24. CC-2004772 Pharmacokinetics - Cmax

    Maximum plasma drug concentration, if possible.

    Measured: Up to 4 years

  25. CC-2004772 Pharmacokinetics - AUC(0-T)

    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.

    Measured: Up to 4 years

  26. CC-2004772 Pharmacokinetics - AUC(TAU)

    Area under the plasma concentration time-curve from time 0 to 24 hours postdose, if possible.

    Measured: Up to 4 years

  27. CC-2004772 Pharmacokinetics - Tmax

    Time to peak (maximum) plasma concentration, if possible.

    Measured: Up to 4 years

  28. CC-2004772 Pharmacokinetics - T-HALF

    Half-life, if possible.

    Measured: Up to 4 years

  29. CC-2004772 Pharmacokinetics - CLT/F

    Apparent total clearance of the drug from plasma after oral administration, if possible.

    Measured: Up to 4 years

  30. CC-2004772 Pharmacokinetics - Vz/F

    Apparent volume of distribution, if possible.

    Measured: Up to 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Jul 2021
  2. Study start2 Dec 2021
  3. Primary completion30 Jul 2025
  4. Study completion30 Jul 2025

Why it stopped: Efficacy endpoint met; however, overall experimental dosing regimen is not considered optimal to support further clinical development in this patient population

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
56 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04951778
Sponsor's protocol ID
CC-91633-AML-001
Other IDs
2020-005329-95
Status last verified
Aug 2025
First submitted
23 Jun 2021
Last updated
27 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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/

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CC-91633 in people with Leukemia, Myeloid, Acute and Myelodysplastic Syndromes, run by Celgene at 9 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 27 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Efficacy endpoint met; however, overall experimental dosing regimen is not considered optimal to support further clinical development in this patient population). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Tolerated Dose (MTD) (time frame: Up to 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04951778.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Aug 2025.

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