Fampridine in MS Patients: A Cognition, Fatigue, Depression and Quality of Life Analysis (FAMILY)

Fampyra, a voltage-dependent potassium channel blocker, is indicated for the improvement of walking in adult patients with multiple sclerosis with walking disability (EDSS 4-7). The medicinal product has been authorized under a so-called "conditional approval" scheme. This means that further evidence on this medicinal product is awaited and this study aims to assess the effect of Fampyra, administered according to standard clinical practice, on cognition, fatigue and quality of life in patients with MS.

This clinical study is a multicenter, prospective, non-interventional, cohort study of MS patients receiving Fampyra in outpatient setting. Patients will be treated according to the local prescribing information of the study medication and routine medical practice in terms of visit frequency and types of assessments performed. The assignment of the patient to this therapeutic strategy is not decided in advance by the study protocol but falls within current practice and the prescription of Fampyra is clearly separated from the physician's decision to include the patient in the current study.

Since this is purely non-interventional study, primary data -which will be obtained prospectively during the study visits through patients' interview and patient reported outcomes or as performed per standard clinical practice- will mainly be employed.

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis
• Intervention / Treatment: Drug: Fampridine

Detailed Description

The study will take place in Greece. Investigator's decision to prescribe Fampyra (according to the indication for treatment as defined in the summary of product characteristics, SmPC and the current clinical practice), should precede entry into the study.

• Study Type: Observational
• Enrollment (Actual): 111

Contacts and Locations

Study Locations

• Greece
  • Attika
    • Athens, Attika, Greece, 11528
      • Αiginitio hospital

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

The target population of the current clinical study is male and female patients with MS, aged ≥18 years, receiving Fampyra for walking disability according to standard clinical practice.

Description

Inclusion Criteria:

• Adult patient (≥18 years old) with MS.
• Patient receiving Fampyra® for walking disability (EDSS 4-7).
• Patient receiving stable doses of any Disease Modifying Treatment (DMT) for at least 3 months prior to study entry.
• Patients will receive Fampyra® in line with the approved Summary of Product Characteristics (SmPC).
• Patients for whom the decision to prescribe therapy with Fampyra according to the locally approved product's summary of product characteristics (SmPC) has already been taken prior to their enrollment in the study and is clearly separated from the physician's decision to include the patient in the current study.
• Patients must have signed an informed consent document.
• Patients must be able to read, understand and complete the study specific questionnaires.

Exclusion Criteria:

• Pregnant or lactating woman.
• Patient with prior history or current presentation of seizure.
• Patient with mild, moderate or severe renal impairment (creatinine clearance < 80 ml/min).
• Concurrent treatment with other medicinal products containing fampridine (4-aminopyridine).
• Concomitant treatment with medicinal products that are inhibitors of Organic Transporter 2 (OCT2), e.g., cimetidine.
• Patients who have received more than 1 dose of fampridine at the time of enrollment into the study.
• Patients that meet any of the contraindications to the administration of the study drug according to the approved SmPC.
• Patients who currently receive treatment with any investigational drug/device/intervention or have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) before the commencement of therapy with fampridine

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in cognition measured with PASAT scale	Correlated with the multidimensional Health Related Quality of Life in MS patients	Assessed at 2 weeks, 12 weeks and 24 weeks compared to Baseline (week 0).
Changes in depression measured with BDI-II scale	Correlated with the multidimensional Health Related Quality of Life in MS patients	Assessed at 2 weeks, 12 weeks and 24 weeks compared to Baseline (week 0).
Changes in fatigue measured with MFIS scale	Correlated with the multidimensional Health Related Quality of Life in MS patients	Assessed at 2 weeks, 12 weeks and 24 weeks compared to Baseline (week 0).
Changes in Quality of Life measured with MusiQoL scale	Correlated with the multidimensional Health Related Quality of Life in MS patients	Assessed at 2 weeks, 12 weeks and 24 weeks compared to Baseline (week 0)
Changes in Quality of Life measured with MSIS-29 scale	Correlated with the multidimensional Health Related Quality of Life in MS patients	Assessed at 2 weeks, 12 weeks and 24 weeks compared to Baseline (week 0)

Collaborators and Investigators

• Sponsor: Genesis Pharma CNS & Specialty

Publications and helpful links

General Publications

• Mitsikostas DD, Doskas T, Gkatzonis S, Fakas N, Maltezou M, Papadopoulos D, Gourgioti R, Mitsias P. A Prospective, Observational, Cohort Study to Assess the Efficacy and Safety of Prolonged-Release Fampridine in Cognition, Fatigue, Depression, and Quality of Life in Multiple Sclerosis Patients: The FAMILY Study. Adv Ther. 2021 Mar;38(3):1536-1551. doi: 10.1007/s12325-020-01606-5. Epub 2021 Feb 2.

Study record dates

Study Major Dates

• Study Start (Actual): March 7, 2017
• Primary Completion (Actual): December 15, 2018
• Study Completion (Actual): December 15, 2018

Study Registration Dates

• First Submitted: March 29, 2017
• First Submitted That Met QC Criteria: May 22, 2017
• First Posted (Actual): May 23, 2017

Study Record Updates

• Last Update Posted (Actual): February 15, 2019
• Last Update Submitted That Met QC Criteria: February 13, 2019
• Last Verified: February 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Molecular Mechanisms of Pharmacological Action; Membrane Transport Modulators; Potassium Channel Blockers; 4-Aminopyridine
• Other Study ID Numbers: NIS-GEN-FMP-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

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