Trial Outcomes & Findings for A Single Arm Study Evaluating the Efficacy, Safety and Tolerability of Ofatumumab in Patients With Relapsing Multiple Sclerosis (NCT NCT04486716)

NCT ID: NCT04486716

Last Updated: 2026-01-13

Results Overview

Magnetic Resonance Imaging (MRI) was used to measure presence of new or reduction in number of gadolinium enhancing T1 lesions. Each MRI scan was previewed by a local neuroradiologist. The quality of each scan performed was assessed by a central MRI reading center and evaluated for quality, completeness and adherence to the protocol. A nonresponder imputation (NRI) for missing data approach was applied. NRI assumes that a participant was a treatment failure, i.e. non-responder, if they did not have a valid Month 12 MRI assessment, or if they discontinued the study prematurely and did not have a valid Month 12 MRI assessment.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 111 participants
• Primary outcome timeframe: Baseline (assessed at screening visit), Month 12
• Results posted on: 2026-01-13

Participant Flow

This study is conducted in 20 sites in the United States.

The study had a Core phase and a Post-Treatment Safety Follow-up (FU). The Core phase covered a 28-day Screening Period, 12-Month Treatment Period, and 30-Day Telephone Safety FU. Upon completing the study, participants could opt to continue ofatumumab therapy through commercial services. Those not continuing with commercial ofatumumab or switching therapies entered the Post-Treatment Safety FU period.

Participant milestones

Measure	Ofatumumab 20 mg Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Core Phase STARTED	111
Core Phase Full Analysis Set / Safety Analysis Set	102
Core Phase COMPLETED	89
Core Phase NOT COMPLETED	22
Post-Treatment Safety FU STARTED	2
Post-Treatment Safety FU COMPLETED	2
Post-Treatment Safety FU NOT COMPLETED	0

Reasons for withdrawal

Measure	Ofatumumab 20 mg Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Core Phase Subject decision	6
Core Phase Adverse Event	5
Core Phase Lost to Follow-up	1
Core Phase Physician Decision	1
Core Phase Enrolled subjects not treated	9

Baseline Characteristics

A Single Arm Study Evaluating the Efficacy, Safety and Tolerability of Ofatumumab in Patients With Relapsing Multiple Sclerosis

Baseline characteristics by cohort

Measure	Ofatumumab 20 mg n=102 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Age, Continuous	43.51 years STANDARD_DEVIATION 8.244 • n=210 Participants
Sex: Female, Male Female	69 Participants n=210 Participants
Sex: Female, Male Male	33 Participants n=210 Participants
Race/Ethnicity, Customized White	78 Participants n=210 Participants
Race/Ethnicity, Customized Black or African American	20 Participants n=210 Participants
Race/Ethnicity, Customized Asian	3 Participants n=210 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	1 Participants n=210 Participants

PRIMARY outcome

Timeframe: Baseline (assessed at screening visit), Month 12

Population: Participants in the Full Analysis Set with a value for the outcome measure at baseline.

Magnetic Resonance Imaging (MRI) was used to measure presence of new or reduction in number of gadolinium enhancing T1 lesions. Each MRI scan was previewed by a local neuroradiologist. The quality of each scan performed was assessed by a central MRI reading center and evaluated for quality, completeness and adherence to the protocol.

A nonresponder imputation (NRI) for missing data approach was applied. NRI assumes that a participant was a treatment failure, i.e. non-responder, if they did not have a valid Month 12 MRI assessment, or if they discontinued the study prematurely and did not have a valid Month 12 MRI assessment.

Outcome measures

Measure	Ofatumumab 20 mg n=97 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Percentage of Participants With no Change or a Reduction From Baseline in the Number of Gadolinium Enhancing (GdE) Lesions at Month 12 Using Non-responder Imputation	86.6 percentage of participants Interval 78.17 to 92.67

PRIMARY outcome

Timeframe: Baseline (assessed at screening visit), Month 12

Population: Participants in the Full Analysis Set with a value for the outcome measure at both baseline and Month 12.

Magnetic Resonance Imaging (MRI) was used to measure presence of new or reduction in number of gadolinium enhancing T1 lesions. Each MRI scan was previewed by a local neuroradiologist. The quality of each scan performed was assessed by a central MRI reading center and evaluated for quality, completeness and adherence to the protocol.

A sensitivity analysis of the primary endpoint was performed based on an observed data approach.

Outcome measures

Measure	Ofatumumab 20 mg n=84 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Percentage of Participants With no Change or a Reduction From Baseline in the Number of Gadolinium Enhancing (GdE) Lesions at Month 12 Based on Observed Data	100 percentage of participants Interval 95.7 to 100.0

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: Full Analysis Set

Retention on study treatment from baseline to Month 6 and to Month 12 was based on the number of participants who continued study treatment.

Outcome measures

Measure	Ofatumumab 20 mg n=102 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Number of Participants Who Continued Study Treatment From Baseline to Months 6 and 12 Month 6	95 Participants
Number of Participants Who Continued Study Treatment From Baseline to Months 6 and 12 Month 12	89 Participants

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: Participants in the Safety Analysis Set with an available value for the outcome measure at both baseline and the corresponding time point.

Changes from baseline in lymphocytes, including total CD19+ B cell counts and CD20+CD3+ T cell counts, were obtained by fluorescence-activated cell sorting (FACS), which is a specific type of flow cytometry.

Outcome measures

Measure	Ofatumumab 20 mg n=91 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Change From Baseline in CD19+ B Cell Counts Obtained by FACS Month 6	-24.45 CD19+ B cells * 10^6/L Standard Deviation 58.295
Change From Baseline in CD19+ B Cell Counts Obtained by FACS Month 12	-17.53 CD19+ B cells * 10^6/L Standard Deviation 52.182

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: Participants in the Safety Analysis Set with an available value for the outcome measure at both baseline and the corresponding time point.

Changes from baseline in lymphocytes, including total CD19+ B cell counts and CD20+CD3+ T cell counts, were obtained by fluorescence-activated cell sorting (FACS), which is a specific type of flow cytometry.

Outcome measures

Measure	Ofatumumab 20 mg n=88 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Change From Baseline in CD20+ CD3+ T Cell Counts Obtained by FACS Month 6	-11.144 CD20+ CD3+ T cells * 10^6/L Standard Deviation 39.1520
Change From Baseline in CD20+ CD3+ T Cell Counts Obtained by FACS Month 12	-2.590 CD20+ CD3+ T cells * 10^6/L Standard Deviation 32.1112

SECONDARY outcome

Timeframe: Baseline (all prior history), Post-baseline (up to Month 12)

Population: Safety Analysis Set

The Columbia-Suicide Severity Rating Scale (C-SSRS) is a questionnaire that assesses suicidal ideation and suicidal behavior. The suicidal ideation section includes 5 items (Categories 1 to 5), and the suicidal behavior section includes 5 items (Categories 6 to 10). Additionally, there is one item about Self-injurious behavior, without suicidal intent.

The C-SSRS was given multiple times throughout the study from baseline up to Month 12. The number of participants who answered 'Yes' to any of the items in C-SSRS at baseline and at any timepoint post-baseline is summarized in this record.

Baseline refers to all prior history.

Outcome measures

Measure	Ofatumumab 20 mg n=102 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Baseline: Any suicidal ideation (Categories 1 to 5)	18 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Baseline: Any suicidal behavior (Categories 6 to 10)	6 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Baseline: Any suicidal ideation or behavior (Categories 1 to 10)	19 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Baseline: Self-injurious behavior, without suicidal intent	4 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Post-baseline: Any suicidal ideation (Categories 1 to 5)	4 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Post-baseline: Any suicidal behavior (Categories 6 to 10)	0 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Post-baseline: Any suicidal ideation or behavior (Categories 1 to 10)	4 Participants
Number of Participants With Suicidal Ideation or Behavior Based on Columbia-Suicide Severity Rating Scale (C-SSRS) Post-baseline: Self-injurious behavior, without suicidal intent	0 Participants

SECONDARY outcome

Timeframe: Baseline, Month 6, Month 12

Population: Participants in the Full Analysis Set with an available value for the outcome measure at each time point.

The Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) is a 9-item general instrument that measures the major dimensions of satisfaction with a medication. The questionnaire consists of 3 domains: effectiveness (items 1 to 3), convenience (items 4 to 6) and global satisfaction (items 7 to 9). The scores of each domain range from 0 to 100 with higher scores representing higher satisfaction on that domain.

Outcome measures

Measure	Ofatumumab 20 mg n=51 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Effectiveness Score - Baseline	61.11 Score on scale Interval 0.0 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Effectiveness Score - Month 6	66.67 Score on scale Interval 5.6 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Effectiveness Score - Month 12	66.67 Score on scale Interval 11.1 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Convenience Score - Baseline	61.11 Score on scale Interval 22.2 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Convenience Score - Month 6	88.89 Score on scale Interval 50.0 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Convenience Score - Month 12	86.11 Score on scale Interval 50.0 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Global Satisfaction Score - Baseline	63.89 Score on scale Interval 5.6 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Global Satisfaction Score - Month 6	77.78 Score on scale Interval 33.3 to 100.0
Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Scores at Baseline, Month 6 and Month 12 TSQM-9 Global Satisfaction Score - Month 12	42.22 Score on scale Interval 33.3 to 100.0

SECONDARY outcome

Timeframe: From first dose of study drug (Day 1) up to 30 days after last dose (Month 13)

Population: Safety Analysis Set

TEAEs are defined as any adverse events (AEs) that started on or after the day of first dose of study drug, or before 30 days after the treatment end date, if severity at baseline was missing or if postbaseline severity was greater than baseline severity.

Outcome measures

Measure	Ofatumumab 20 mg n=102 Participants Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Participants with AEs (any AE, regardless of seriousness)	86 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) Participants with serious adverse events (SAEs)	6 Participants

Adverse Events

Ofatumumab 20 mg

Serious events: 6 serious events

Other events: 53 other events

Deaths: 0 deaths

Serious adverse events

Measure	Ofatumumab 20 mg n=102 participants at risk Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
Musculoskeletal and connective tissue disorders Pain in extremity	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Nervous system disorders Cerebrovascular accident	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Vascular disorders Deep vein thrombosis	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Infections and infestations Urosepsis	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Injury, poisoning and procedural complications Sciatic nerve injury	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Injury, poisoning and procedural complications Vascular graft complication	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Infections and infestations COVID-19	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Infections and infestations Infective myositis	0.98% 1/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).

Other adverse events

Measure	Ofatumumab 20 mg n=102 participants at risk Subcutaneous injection of ofatumumab 20 mg on Day 1, Day 7, Day 14 and thereafter once monthly starting at Month 1 and up to Month 12.
General disorders and administration site conditions Fatigue	11.8% 12/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
General disorders and administration site conditions Pyrexia	5.9% 6/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Infections and infestations COVID-19	30.4% 31/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Infections and infestations Urinary tract infection	10.8% 11/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Nervous system disorders Headache	11.8% 12/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).
Skin and subcutaneous tissue disorders Pruritus	5.9% 6/102 • From first dose (Day 1) through the safety follow-up period, with an average duration of 30 days after last dose (approximately 13 months total) and a maximum follow-up of up to 18 months post-dose (approximately 30 months total).

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: Novartis.email@novartis.com

Results disclosure agreements

• Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. Novartis does not prohibit any investigator from publishing. Any publications from a single site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
• Publication restrictions are in place

Restriction type: OTHER