Observational Study in Patients With Relapsing-Remitting Multiple Sclerosis Switched to Bafiertam® From Dimethyl Fumarate (PERSIST)

An Open-label, Observational Study in Patients With Relapsing-Remitting Multiple Sclerosis Switched to Bafiertam® (Monomethyl Fumarate) From Dimethyl Fumarate (PERSIST)

This is an open-label, multicenter, observational study evaluating GI symptoms in relapsing-remitting multiple sclerosis (RRMS) patients who switch from dimethyl fumarate (DMF) to Bafiertam®.

Study Overview

• Status: Withdrawn
• Conditions: Relapsing Remitting Multiple Sclerosis
• Intervention / Treatment: Drug: monomethyl fumarate

Detailed Description

This is an open-label, multicenter, observational study evaluating GI symptoms in relapsing-remitting multiple sclerosis (RRMS) patients who switch from dimethyl fumarate (DMF) to Bafiertam® (monomethyl fumarate).

Adult patients with a diagnosis of RRMS receiving continuous treatment with DMF monotherapy and who are experiencing GI symptoms will be eligible to enroll in the study.

Patients will be identified and screened by chart review and invited to report GI Symptoms for one baseline week using a study app downloaded on their own smartphone/tablet. During this week, patients will continue their currently prescribed and remaining DMF treatment and will record their GI symptoms daily using the app.

At the end of the Baseline Period, patients will be asked to cease administration of the DMF, and to commence self-treatment with Bafiertam® 95 mg capsules twice a day (total daily dose of 380 mg) approximately 12 hours apart the following day for 28 days. They will be required to continue to record their GI symptoms each day.

On Day 28; End-of Study [EOS]) the patient's diary will be reviewed, adverse events (AE) recorded, and drug accountability performed.

Following EOS patients will be asked if they wish to continue Bafiertam® treatment. Those patients who elect to continue will be contacted one month and two months after EOS to ascertain if they remain on Bafiertam®.

• Study Type: Observational

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with a confirmed diagnosis of relapsing-remitting multiple sclerosis currently treated with dimethyl fumarate (DMF), and experiencing gastrointestinal symptoms attributable to DMF are eligible. Sufficient patients will be enrolled until 50 evaluable patients complete the study.

Description

Key Inclusion Criteria:

1. Male or non-pregnant female aged 18years and older.
2. Diagnosis of RRMS (per McDonald 2010 Criteria) at screening [Polman 2011].
3. Currently being treated with dimethyl fumarate (DMF; Tecfidera® or generic equivalent). At least 1 week and 1 day of continuous treatment with DMF monotherapy prior to initiation of Bafiertam®. Note: continuous treatment with DMF is defined as treatment uninterrupted by other disease-modifying treatment.
4. GI symptoms (defined as any combination of abdominal pain, nausea, vomiting, diarrhea, constipation, bloating, or flatulence) while receiving treatment with DMF.
5. Must be naïve to Bafiertam®.
6. Additional inclusion criteria apply

Key Exclusion Criteria:

1. Diagnosis of primary progressive, secondary progressive, or progressive relapsing MS (as defined by Lublin and Reingold) [Lublin 2014].
2. Females breastfeeding, pregnant, or planning to become pregnant; or women who have a positive pregnancy test result during screening.
3. History of severe hypersensitivity (e.g., anaphylaxis or anaphylactoid reactions) to the active ingredient (monomethyl fumarate), dimethyl fumarate, diroximel fumarate (Vumerity®), or any of the excipients.
4. Lymphocyte counts less than 1000/microliter. Any other clinically significant abnormalities in CBC, blood and urine chemistries, or liver function tests in the past 30 days.
5. History of severe opportunistic infections (including progressive multifocal leukoencephalopathy (PML) and herpes zoster) or any clinically significant, cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic (other than MS), dermatologic, psychiatric, and renal, or other major disease, as determined by the Investigator.
6. Any prior suspicion of PML.

9. Additional exclusion criteria apply.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Comparison of the frequency and severity of GI symptoms before and after switching to Bafiertam®.	The GI tolerability of Bafiertam® following a switch from dimethyl fumarate will be assessed by determining the frequency and severity of GI events using a modified gastrointestinal symptom scale.	28 days

Collaborators and Investigators

• Sponsor: Banner Life Sciences LLC
• Investigators: Study Director: Thomas W Lategan, D.Phil., Banner Life Sciences LLC

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): June 30, 2021
• Primary Completion (ANTICIPATED): August 30, 2022
• Study Completion (ANTICIPATED): September 29, 2022

Study Registration Dates

• First Submitted: June 8, 2021
• First Submitted That Met QC Criteria: June 11, 2021
• First Posted (ACTUAL): June 14, 2021

Study Record Updates

• Last Update Posted (ACTUAL): November 18, 2021
• Last Update Submitted That Met QC Criteria: November 15, 2021
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Keywords: gastrointestinal; Tecfidera; dimethyl fumarate; Bafiertam; monomethyl fumarate
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Sclerosis; Multiple Sclerosis, Relapsing-Remitting
• Other Study ID Numbers: BLS-11-403

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No