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Study to Assess the Effect of Ofatumumab in Treatment Naïve, Very Early RRMS Patients Benchmarked Against Healthy Controls.

Phase 4
Active, not recruiting
Conditions
Relapse Remitting Multiple Sclerosis
Interventions
Registration Number
NCT05084638
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

This study will evaluate the impact of ofatumumab in Relapsing Remitting Multiple Sclerosis (RRMS) participants that are very early in the course of their disease using clinical and magnetic resonance imaging (MRI) outcomes. The study will also assess changes in disease using monitoring techniques including digital biometric device use, biomarker analysis and non-conventional MRI. Select outcomes in the ofatumumab treated group will be compared to a group of Healthy participants to determine if there are similarities between the groups after the patients with MS undergo treatment with ofatumumab.

Detailed Description

The study is an open-label, multi-center, prospective 18-month study in 118 MS participants with early RRMS (defined as within 6 months of diagnosis of clinically definite RRMS) and who are treatment naïve. It is designed to determine if RRMS participants treated with 20 mg subcutaneous monthly ofatumumab during the earliest part of their disease will benefit from the use of ofatumumab as their first disease modifying therapy. Additionally, RRMS patients will be compared to age- and sex-matched healthy participants (n=50) for select outcomes to observe similarities and differences between the groups.

After giving consent, participants will have a 28-day screening/qualification period. If they qualify to continue, they will start study measures including assessments of clinical and magnetic resonance imaging (MRI) metrics and use of a digital monitoring watch. Additionally, samples will be collected for laboratory and biomarker analysis. RRMS participants will begin treatment with ofatumumab for the next 18 months. Healthy participants will undergo similar assessments; however they will not receive any treatment during the course of the study. Over the 18 months, participants will have regular clinical visits with assessments and sample collection. After 18 months in the trial, participants in both groups will have the option to enter into a 12-month extension (up to 30 months total in study) to collect further information on long-term clinical and MRI outcomes.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
180
Inclusion Criteria

Participants eligible for inclusion in this study must meet all of the following criteria:

  1. Signed informed consent must be obtained prior to participation in the study

  2. Age 18-35 years

    Patients in the healthy control arm eligible for inclusion must fulfill the following criteria:

  3. Able to obtain MRI (HC with abnormal MRI at Screening will be excluded) and use wearable device

  4. Able to provide blood sample (no CSF will be collected in HC)

    Patients in the ofatumumab-treated arm eligible for inclusion must fulfill the following criteria:

  5. Diagnosis of RRMS per McDonald Criteria (2010/2017)

  6. Within 6 months of diagnosis of clinically definite MS (CDMS)

  7. EDSS 0-3.0 (Inclusive)

  8. Treatment-naïve to MS DMT

  9. Able to obtain MRI and attend study visits at sites

  10. Able to use wearable device

  11. Able to provide blood sample (and CSF for sub-group n=15)

Key

Exclusion Criteria

Participants in the healthy control arm meeting any of the following criteria are not eligible for inclusion in this study:

  1. Confounding medical condition as determined by the investigator

    RRMS patients fulfilling any of the following exclusion criteria are not eligible for inclusion in this study:

  2. Diseases other than multiple sclerosis responsible for the clinical or MRI presentation

  3. Patients with neuromyelitis optica, Radiologic/ Clinically Isolated Syndrome, Secondary Progressive or Primary Progressive MS diagnosis

  4. Use of experimental or investigational drugs for MS

  5. Previous use of Disease Modifying Therapy (DMT) or chemotherapeutic medications for MS

  6. Relapse between screening and Baseline visits

  7. Known sensitivity to gadolinium; patients with chronic, severe kidney disease

  8. Known history of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes

  9. CNS anomalies that are better accounted for by another disease process or MRI anomalies causing clinically apparent impairments

  10. Known active malignancies

  11. Pregnant or nursing (lactating) women

  12. Females of childbearing potential (all women physiologically capable of becoming pregnant) should use effective contraception while receiving ofatumumab and for 6 months after the last treatment of ofatumumab

  13. Patients with an active chronic disease (or stable but treated with immune therapy) of the immune system other than MS or with immunodeficiency syndrome

  14. Patients with active infections including systemic bacterial, viral (including SARS-CoV-2/COVID-19) or fungal infections, or known to have AIDS or to test positive for HIV antibody at Screening

  15. Patients with neurological findings consistent with Progressive Multifocal Leukoencephalopathy (PML), or confirmed PML

  16. Patients with IgG or IgM levels below LLN at Screening

  17. Patients that have received any live or live-attenuated vaccines within 4 weeks prior to first dose of study drug administration

  18. Patients at risk of developing or having reactivation of hepatitis

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
OfatumumabOfatumumabOfatumumab will be provided in an autoinjector for subcutaneous administration. Dosing regimen for this study is an initial dose of 20mg at Baseline/Week 0, followed by Week 1, 2 and every month thereafter, beginning at Week 4 (Month 1) until Month 18. There will be an optional extension of dosing through month 30.
Primary Outcome Measures
NameTimeMethod
Percentage of participants achieving NEDA-3 (No Evidence of Disease Activity-3)Month 6 to month 18

A participant is considered as achieved NEDA-3 if the participant has not had a clinical relapse (recurrence of a disease activity after a recovery), has not had an increase in disability and has no new radiological MRI activity (no new occurrences of contrast-enhancing lesions) during study Months 6 to 18.

Secondary Outcome Measures
NameTimeMethod
Number of participants with NEDA (No Evidence of Disease Activity) - RadiologicalMonth 6 to Month 18

A participant is considered as achieved NEDA-radiological if the participant has has no new radiological MRI activity (no new occurrences of contrast-enhancing lesions) during study Months 6 to 18.

Change from Baseline in Gd+ lesion countBaseline to Month 18 and 30

Change in the number of gadolinium enhancing lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Change from Baseline in Gd+ lesion volumeBaseline to Month 18 and 30

Change in size of gadolinium enhancing lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Change from Baseline in new/enlarging T2 lesion countBaseline to Month 18 and 30

Change in the number of new/enlarging T2 lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Change from Baseline in T2 lesion volumeBaseline to Month 18 and 30

Change in size of T2 lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Change from Baseline in T1 unenhancing lesion volumeBaseline to Month 18 and 30

Change in the size of T1 unenhancing lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Number of relapsesBaseline to Month 18 and 30

Relapses are recurrences of a disease activity after a recovery. A confirmed MS relapse is one accompanied by a clinically relevant change in the EDSS performed by the EDSS Rater, i.e. an increase of at least 0.5 points on the EDSS score, or an increase of 1 point on two functional scores (FSs) or 2 points on one FS, excluding changes involving bowel/bladder or cerebral FS compared to the previous available rating (the last EDSS rating that did not occur during a relapse). Confirmation of MS relapse based on these definitions will be done centrally.

Number of participants that were 3-month Disability Worsening-freeBaseline up to Month 18 and 30

No increase or worsening of disability over a period of 3 months or more

Number of participants with NEDA (No Evidence of Disease Activity) - ClinicalMonth 6 to Month 18

A participant is considered as achieved NEDA-Clinical if the participant has not had a clinical relapse (recurrence of a disease activity after a recovery).

Change from Baseline for NeuroQOLBaseline to Month 18 and 30

The NeuroQOL is a measurement system that evaluates and monitors the physical, mental, and social effects experienced by adults and children living with neurological conditions. The following domains will be measured.

Physical Health, Mental Health, Social Health. Scales can be scored by summing the values of the response to each item to develop a total raw score.

Change from Baseline for Patient Determined Disease Steps (PDDS)Baseline to Month 18 and 30

The PDDS is a standardized rating scale which is a self-assessment scale of functional disability in multiple sclerosis patients primarily based on ambulation. The questionnaire contains 1 question which is scored ranging from 0 (normal) to 8 (bedridden). A score of 0 to 2 indicates mild disability; a score of 3 to 5 indicates moderate disability; a score of 6 to 8 indicates severe disability.

Brain volume loss (BVL) assessment (whole brain and regional)Month 6 to Month 18 and 30

Brain volume loss is a marker of progressive loss of brain structure and function. It is a predictor of disability progression. Evaluate the effect of ofatumumab vs healthy controls on 1) whole brain and regional atrophy measured at month 18/30 after re-baseline at 6 months; and 2) regional atrophy measured 18/30 months from Baseline

Number of participants with treatment emergent adverse eventsBaseline up to approximately Month 30

Adverse event monitoring should be continued following the last dose of study treatment until B cells are repleted. Repletion is defined as a concentration \> the participant's baseline value or \> the lower limit of normal, whichever is observed first. Other safety assessments (physical exam, vital signs, etc) that meet the definition of an adverse event or are considered clinically relevant by the investigator will be reported as an adverse event.

Change from Baseline in new unenhancing T1 lesion numberBaseline to Month 18 and 30

Change in the number of new unenhancing T1 lesions will be measured by Magnetic Resonance Imaging (MRI). Each MRI scan will be previewed by a local neuroradiologist. The quality of each scan performed will be assessed by a central MRI reading center.

Trial Locations

Locations (36)

Sibyl Wray MD Neurology PC

🇺🇸

Knoxville, Tennessee, United States

MD First Research

🇺🇸

Chandler, Arizona, United States

Barrow Neurological Clinics at St Josephs Hospital and MC

🇺🇸

Phoenix, Arizona, United States

Arizona Neuroscience Research LLC

🇺🇸

Phoenix, Arizona, United States

Keck School of Medicine

🇺🇸

Los Angeles, California, United States

Neurology of Central FL Res Ctr

🇺🇸

Altamonte Springs, Florida, United States

Lundquist Inst BioMed at Harbor

🇺🇸

Torrance, California, United States

Regina Berkovich MD PhD Inc

🇺🇸

West Hollywood, California, United States

UC Health Neuroscience Ctr

🇺🇸

Aurora, Colorado, United States

MedStar Health

🇺🇸

Washington, District of Columbia, United States

First Choice Neurology

🇺🇸

Boca Raton, Florida, United States

Univ of Florida College of Medicine

🇺🇸

Gainesville, Florida, United States

Neurology Associates PA

🇺🇸

Maitland, Florida, United States

Orlando Health Clinical Trials

🇺🇸

Orlando, Florida, United States

Emerald Coast Neurology

🇺🇸

Pensacola, Florida, United States

Tallahassee Neurological Clinic

🇺🇸

Tallahassee, Florida, United States

University Of South Florida

🇺🇸

Tampa, Florida, United States

Shepherd Center

🇺🇸

Atlanta, Georgia, United States

Ochsner Cancer Institute

🇺🇸

New Orleans, Louisiana, United States

University of Massachusetts Medical School

🇺🇸

Worcester, Massachusetts, United States

Henry Ford Hospital

🇺🇸

Detroit, Michigan, United States

Renown Institute for Neurosciences

🇺🇸

Reno, Nevada, United States

Neuroscience Institute at Hackensack

🇺🇸

Hackensack, New Jersey, United States

University of New Mexico

🇺🇸

Albuquerque, New Mexico, United States

Velocity Clinical Research

🇺🇸

Raleigh, North Carolina, United States

Neurology Diagnostics Inc

🇺🇸

Dayton, Ohio, United States

Multiple Sclerosis Center of Excellence of OMRF

🇺🇸

Oklahoma City, Oklahoma, United States

Thomas Jefferson University Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

Univ of Texas Southwest Med Center

🇺🇸

Dallas, Texas, United States

UT Health Science Center

🇺🇸

Houston, Texas, United States

Lonestar Neurology of San Antonio

🇺🇸

San Antonio, Texas, United States

Evergreen Health Multiple Sclerosis Center

🇺🇸

Kirkland, Washington, United States

MultiCare Neuroscience Center of Washington

🇺🇸

Tacoma, Washington, United States

West Virginia University Hospital

🇺🇸

Morgantown, West Virginia, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Caribbean Center for Clinical Research, Inc

🇵🇷

Guaynabo, Puerto Rico

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