Trial Outcomes & Findings for Ofatumumab for Minimal Residual Disease (MRD) and Maintenance Therapy (NCT NCT01258933)

Last Updated: 2025-10-01

Results Overview

Response assessment according to 2008 International Working Group for CLL (IWCLL), prior to 9th dose of ofatumumab (prior to first bimonthly dose). Responses of (complete remission (CR) conversion to minimal residual disease (MRD) negative, partial remission (PR) conversion to nodular partial remission nPR or CR, and nPR conversion to complete remission (CR)) evaluated by physical examination, CBC, CT of chest, abdomen, pelvis, and bone marrow aspirate and biopsy with evaluation of residual disease (MRD) by 4-color flow cytometry.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Week 12

Results posted on

2025-10-01

Participant Flow

Participant milestones

Participant milestones
Measure	Ofatumumab Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ofatumumab for Minimal Residual Disease (MRD) and Maintenance Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ofatumumab n=4 Participants Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	70 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: Week 12

Outcome measures

Outcome measures
Measure	Ofatumumab n=4 Participants Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Number of Patients With Objective Response	0 Participants

SECONDARY outcome

Timeframe: Start of study drug up to approximately 1 year and 7 months.

Time from date of treatment start until the date of failure or death from any cause.

Outcome measures

Outcome measures
Measure	Ofatumumab n=4 Participants Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Time-to-Treatment Failure (TTF)	8.6 Months Interval 1.6 to 12.7

SECONDARY outcome

Timeframe: Start of study drug up to approximately 7 years and 6 months.

Time from date of treatment start until the date of progression or death from leukemia.

Outcome measures

Outcome measures
Measure	Ofatumumab n=4 Participants Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Progression-Free Survival (PFS)	26.0 Months Interval 25.1 to 90.0

Adverse Events

Ofatumumab

Serious events: 0 serious events

Other events: 4 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Ofatumumab n=4 participants at risk Ofatumumab 300 mg dose 1, then 1,000 mg weekly \* 7, (treatment) then 1,000 mg every 2 months beginning on week 12 for a total of 2 years of treatment or until progression (maintenance) of disease. The follow-up period will be the period after completion of maintenance. Ofatumumab: 300 mg Dose 1, then 1,000 mg weekly x 7, (treatment) then 1,000 mg every 2 months beginning on week 12
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal Cell Carcinoma	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Nervous system disorders Dysgeusia	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Renal and urinary disorders Urinary incontinence	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Surgical and medical procedures Splenectomy	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders Infusion reaction	75.0% 3/4 • Number of events 3 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Metabolism and nutrition disorders Hypophosphatemia	75.0% 3/4 • Number of events 3 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Infections and infestations Pneumonia	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Blood and lymphatic system disorders thrombocytopenia	100.0% 4/4 • Number of events 4 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Psychiatric disorders Anxiety	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders Chest Pain	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Respiratory, thoracic and mediastinal disorders Cough	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders edema	50.0% 2/4 • Number of events 2 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders fatigue	50.0% 2/4 • Number of events 2 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders fever	75.0% 3/4 • Number of events 3 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
General disorders Flu-like Syndrome	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Skin and subcutaneous tissue disorders Rash	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Respiratory, thoracic and mediastinal disorders Shortness of Breath	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.
Respiratory, thoracic and mediastinal disorders Sore Throat	25.0% 1/4 • Number of events 1 • Adverse Events monitored monthly until follow-up, assessed up to 3 years. Mortality was monitored up to approximately 13 years, 3 months.

Additional Information

William Wierda, MD./Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-0428

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place