Trial Outcomes & Findings for Rituximab + High-Dose Methylprednisolone Debulking Prior to Venetoclax for CLL & SLL Patients (NCT NCT04981912)

NCT ID: NCT04981912

Last Updated: 2025-10-15

Results Overview

Percentage of patients that have a decrease in tumor burden from levels of disease that meet "Medium/High-tumor burden" criteria to meet "Low tumor burden" criteria for disease burden following 1 or 2 cycles of HDMP + Rituximab. Low Tumor Burden = All measurable lymph nodes with the largest diameter \< 5 cm by radiologic assessment AND ALC \< 25k/uL Medium Tumor Burden = Any measurable lymph node with the largest diameter \>/= 5 cm but \< 10 cm by radiographic assessment OR ALC \>/= 25k/uL High Tumor Burden = Any measurable lymph node with the largest diameter \>/= 10 cm by radiologic assessment OR ALC \>/= 25k/uL AND any measurable lymph node with the largest diameter \>/= 5 cm

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE1

Target enrollment

4 participants

Primary outcome timeframe

28 or 56 days

Results posted on

2025-10-15

Participant Flow

This study accrued four patients in total.

The the pre-planned sample size was 17.

Participant milestones

Participant milestones
Measure
Arm A
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Overall Study
STARTED
4
Overall Study
COMPLETED
2
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Arm A
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Overall Study
Physician Decision
2

Baseline Characteristics

Rituximab + High-Dose Methylprednisolone Debulking Prior to Venetoclax for CLL & SLL Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=99 Participants
Age, Categorical
>=65 years
3 Participants
n=99 Participants
Age, Continuous
69 years
n=99 Participants
Sex: Female, Male
Female
1 Participants
n=99 Participants
Sex: Female, Male
Male
3 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=99 Participants
Race (NIH/OMB)
White
2 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
2 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
4 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Prior Lines of Therapy
1.5 Lines
n=99 Participants

PRIMARY outcome

Timeframe: 28 or 56 days

Population: All participants who received at least one dose of treatment.

Percentage of patients that have a decrease in tumor burden from levels of disease that meet "Medium/High-tumor burden" criteria to meet "Low tumor burden" criteria for disease burden following 1 or 2 cycles of HDMP + Rituximab. Low Tumor Burden = All measurable lymph nodes with the largest diameter \< 5 cm by radiologic assessment AND ALC \< 25k/uL Medium Tumor Burden = Any measurable lymph node with the largest diameter \>/= 5 cm but \< 10 cm by radiographic assessment OR ALC \>/= 25k/uL High Tumor Burden = Any measurable lymph node with the largest diameter \>/= 10 cm by radiologic assessment OR ALC \>/= 25k/uL AND any measurable lymph node with the largest diameter \>/= 5 cm

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Percentage of Patients That Have a Decrease in Tumor Burden From Medium or High to Low
2 Participants

SECONDARY outcome

Timeframe: 3 years

Population: All participants who received at least one dose of treatment.

Percentage of patients that have a decrease in tumor burden from levels of disease that meet "Medium/High-tumor burden" criteria to meet "Low tumor burden" criteria for disease burden following 1 cycle of HDMP + Rituximab. Low Tumor Burden = All measurable lymph nodes with the largest diameter \< 5 cm by radiologic assessment AND ALC \< 25k/uL Medium Tumor Burden = Any measurable lymph node with the largest diameter \>/= 5 cm but \< 10 cm by radiographic assessment OR ALC \>/= 25k/uL

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Percentage of Patients That Have a Decrease in Tumor Burden From Medium to Low
2 Participants

SECONDARY outcome

Timeframe: 56 days

Population: All participants who received at least one dose of treatment.

Percentage of patients that have a decrease in tumor burden from levels of disease that meet "Medium/High-tumor burden" criteria to meet "Low tumor burden" criteria for disease burden following 2 cycles of HDMP + Rituximab Low Tumor Burden = All measurable lymph nodes with the largest diameter \< 5 cm by radiologic assessment AND ALC \< 25k/uL Medium Tumor Burden = Any measurable lymph node with the largest diameter \>/= 5 cm but \< 10 cm by radiographic assessment OR ALC \>/= 25k/uL High Tumor Burden = Any measurable lymph node with the largest diameter \>/= 10 cm by radiologic assessment OR ALC \>/= 25k/uL AND any measurable lymph node with the largest diameter \>/= 5 cm

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Percentage of Patients That Have a Decrease in Tumor Burden if 2 Cycles of HDMP + Rituximab
2 Participants

SECONDARY outcome

Timeframe: 2.15 years

Population: All participants who received at least one dose of treatment.

Rate of laboratory TLS (from start of treatment until completion of venetoclax ramp-up)

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Rate of Laboratory TLS (Tumor Lysis Syndrome)
0 Participants

SECONDARY outcome

Timeframe: 2.15 years

Population: All participants who received at least one dose of treatment.

Rate of clinical TLS (from start of treatment until completion of venetoclax ramp-up)

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Rate of Clinical TLS
0 Participants

SECONDARY outcome

Timeframe: 2.15 years

Population: All participants who received at least one dose of treatment.

Number of patients with non-hematological treatment-related adverse events by CTCAE4 definitions of grade 3 or higher, regardless of attribution

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Adverse Events by CTCAE4 Definitions
Number of patients with non-hematological treatment-related adverse events of grade 3 or higher · Grade 3 or higher, related to treatment
1 Participants
Adverse Events by CTCAE4 Definitions
Number of patients with non-hematological treatment-related adverse events of grade 3 or higher · < Grade 3, related or unrelated to treatment
2 Participants
Adverse Events by CTCAE4 Definitions
Number of patients with non-hematological treatment-related adverse events of grade 3 or higher · No AEs
1 Participants
Adverse Events by CTCAE4 Definitions
Number of patients with hematological treatment-related adverse events of grade 3 or higher · Grade 3 or higher, related to treatment
0 Participants
Adverse Events by CTCAE4 Definitions
Number of patients with hematological treatment-related adverse events of grade 3 or higher · < Grade 3, related or unrelated to treatment
3 Participants
Adverse Events by CTCAE4 Definitions
Number of patients with hematological treatment-related adverse events of grade 3 or higher · No AEs
1 Participants

SECONDARY outcome

Timeframe: 9 months and at end of study up to 3 years

Population: All participants who received at least one dose of treatment.

Overall Response rate, Partial Response rate, and Complete response rate per iwCLL criteria after 9 months of venetoclax and at completion of treatment Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Response Rates
Overall response rate around 9 months · Partial response
0 Participants
Response Rates
Overall response rate around 9 months · Complete response
2 Participants
Response Rates
Overall response rate around 9 months · Other
2 Participants
Response Rates
Overall response rate over the course of the study · Partial response
1 Participants
Response Rates
Overall response rate over the course of the study · Complete response
2 Participants
Response Rates
Overall response rate over the course of the study · Other
1 Participants

SECONDARY outcome

Timeframe: 3 years

Population: All participants who received at least one dose of treatment.

Undetectable minimal residual disease (MRD) rate based on bone marrow biopsy after 9 months of venetoclax, and at completion of treatment

Outcome measures

Outcome measures
Measure
Arm A
n=4 Participants
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Undetectable Minimal Residual Disease (MRD) Rate
Undetectable MRD at 9 months · Undetectable MRD
2 Participants
Undetectable Minimal Residual Disease (MRD) Rate
Undetectable MRD at 9 months · Detectable
2 Participants
Undetectable Minimal Residual Disease (MRD) Rate
Overall response rate over the course of the study · Undetectable MRD
3 Participants
Undetectable Minimal Residual Disease (MRD) Rate
Overall response rate over the course of the study · Detectable
1 Participants

Adverse Events

Arm A

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Arm A
n=4 participants at risk
HDMP + rituximab as a means of debulking prior to initiating venetoclax. HDMP + rituximab as a means of debulking prior to initiating venetoclax.: - Patients will receive HDMP + Rituximab for 1 cycle, followed by reassessment of Tumor Burden, * If Tumor Burden classification is low after HDMP + Rituximab (lymph nodes \< 5cm in diameter AND absolute lymphocyte count \< 25k/uL), patients will initiate venetoclax dose ramp-up, with ramp-up schedule according to venetoclax package insert. * Patients who still have a disease burden (lymphadenopathy \> 5 cm or ALC \> 25k/iL) that meets criteria for medium or high risk of TLS after 1 cycle of HDMP + Rituximab are given the option to repeat a 2nd cycle of HDMP + Rituximab prior to venetoclax dose ramp-up.
Ear and labyrinth disorders
Ear and Labyrinth disorders - other
25.0%
1/4 • Number of events 2 • 3 years
Ear and labyrinth disorders
Vertigo
25.0%
1/4 • Number of events 1 • 3 years
Gastrointestinal disorders
Mucositis oral
25.0%
1/4 • Number of events 1 • 3 years
Gastrointestinal disorders
Nausea
25.0%
1/4 • Number of events 2 • 3 years
General disorders
Edema limbs
50.0%
2/4 • Number of events 2 • 3 years
General disorders
Edema trunk
25.0%
1/4 • Number of events 1 • 3 years
General disorders
Fatigue
25.0%
1/4 • Number of events 1 • 3 years
General disorders
Fever
25.0%
1/4 • Number of events 1 • 3 years
General disorders
General disorders and administration site conditions - Other
25.0%
1/4 • Number of events 1 • 3 years
General disorders
Infusion related reaction
50.0%
2/4 • Number of events 3 • 3 years
Infections and infestations
Upper respiratory infection
25.0%
1/4 • Number of events 2 • 3 years
Injury, poisoning and procedural complications
Bruising
25.0%
1/4 • Number of events 1 • 3 years
Metabolism and nutrition disorders
Hyperglycemia
25.0%
1/4 • Number of events 1 • 3 years
Nervous system disorders
Dizziness
25.0%
1/4 • Number of events 1 • 3 years
Nervous system disorders
Headache
50.0%
2/4 • Number of events 2 • 3 years
Nervous system disorders
Peripheral sensory neuropathy
25.0%
1/4 • Number of events 1 • 3 years
Respiratory, thoracic and mediastinal disorders
Dyspnea
25.0%
1/4 • Number of events 1 • 3 years
Respiratory, thoracic and mediastinal disorders
Hoarseness
25.0%
1/4 • Number of events 1 • 3 years
Skin and subcutaneous tissue disorders
Rash acneiform
25.0%
1/4 • Number of events 1 • 3 years

Additional Information

Michael Choi

UCSD

Phone: 8585341765

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place