Multiple sclerosis MedDRA version: 14.1 Level: PT Classification code 10063401 Term: Primary progressive multiple sclerosis System Organ Class: 10029205 - Nervous system disorders MedDRA version: 14.1 Level: PT Classification code 10053395 Term: Progressive multiple sclerosis System Organ Class: 10029205 - Nervous system disorders MedDRA version: 14.1 Level: PT Classification code 10063400 Term: Secondary progressive multiple sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Fampyra
Product Name: Fampyra
Product Code: SUB0750MIG
Pharmaceutical Form: Prolonged-release tablet
INN or Proposed INN: FAMPRIDINE
CAS Number: 504-24-5
Concentration unit: mg milligram(s
Sponsors
St. Vincent's University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients with SPMS or PPMS, aged 18-70, who have Kurtzke EDSS scores in the range 4.0 to 7.0 inclusive and a walking speed with or without aids in the T25FW of 8.0 to 14.0 seconds and evidence of significant upper limb dysfunction as defined by a 9HPT of 15 – 90 seconds (dominant or non-dominant hand), will be recruited. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 70 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1) Patients with a history of seizures, significant liver dysfunction, significant renal dysfunction, significant upper or lower limb arthritis or any other disorder including cognitive dysfunction which would affect the ability to accurately complete questionnaires and give full informed consent. 2) Patients with clinically significant upper limb ataxia or upper limb proprioceptive sensory loss, which in the opinion of the Clinician affect upper limb functional assessment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the effect of treatment with fampridine in patients with secondary progressive MS (SPMS) or primary progressive MS (PPMS) with upper limb dysfunction (as defined by a 9-HPT time of between 15-90 seconds) and Kurtzke EDSS scores in the range 4.0-7.0 on 1. Upper limb function assessed by the nine-hole peg test (9-HPT) and the Jebson Taylor Hand Function Test (JTT). 2. Scores of the MSIS-29 (physical), MSWS-12 and the Disabilities of the Arm, Shoulder and Hand Score (DASH) ;Secondary Objective: 1. To assess the effect of treatment with fampridine on walking mobility assessed by the Timed 25 foot Walk (T25FW) and the Multiple Sclerosis Walking Scale (MSWS-12)6,7 2. To determine whether response or non-response to fampridine by the T25FW can be easily determined using self reported changes in the MSWS-12 alone within 4 weeks in the course of active therapy 3. To determine whether response or non-response to fampridine by the 9HPT or JTT can be easily determined using self reported changes in the DASH alone within 4 weeks on the course of active therapy 4. To determine whether any relation exists between improvement / lack of improvement on the T25FW and improvement/ lack of improvement on the 9-HPT or JTT. 5. To examine the safety of fampridine in this patient population 6. To assess the impact of fampridine on health related quality of life in patients with multiple sclerosis (HRQOL) as measured by the SF-36 questionnaire. ;Primary end point(s): An upper limb responder to fampridine will be defined as a patient with both of the two “on treatment” 9-HPT assessments (assessments 4 & 5 or 7 & 8) improving 20% from the average of the baseline assessments (1, 2 & 3). ;Timepoint(s) of evaluation of this end point: The study will run for 22 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): a) A secondary measure of upper limb responsiveness will be defined as a 20% improvement in from baseline in the average time taken to complete all seven tasks on the JTT “on treatment” (assessments 4 & 5 or 7 & 8) compared with baseline assessments (assessments 1,2 & 3). b) A mobility responder to dalfampridine will be defined as a patient with both of the two “on treatment” T25FW assessments (assessments 4 & 5 or 7 & 8) being better than the maximum of any of the four “off treatment” assessments (assessments 1, 2, 3, & 9). Otherwise the patient will be deemed a non-responder. c) The responders to the 9-HPT and/or JTT will be compared to the non-responders in relation to their changes in the DASH on treatment and off treatment at the baseline assessment. The change in the DASH in responders at Assessment 5 or 8 (week 10 or 20) will be compared to non-responders at that time-point. d) Upper limb response will be examined at the end of treatment phase 1 and repeated at the end of the trial by comparing patients receiving active drug with those receiving placebo to assess any placebo effect and allow assessment of any residual beneficial effect on those who receive fampridine in treatment phase 1 and then switch to placebo in treatment phase 2. Response will again be considered as a 20% improvement in the 9HPT in the active treatment group compared with placebo and a 20% improvement from baseline in the time taken to complete all seven tasks in the JTT. See earlier comment e) The responders to the T25FW will be compared to the non-responders in relation to their changes in the MSWS-12 on treatment and off treatment at the baseline assessment. The change in the MSWS-12 in responders at Assessment 5 or 8 (week 10 or 20) will be compared to non-responders at that time-point. f) The patients’ scores on the MSIS-29 (physical) and SF-36 will be examined in relation to the objective changes (responder/non-responder) in the 9-HPT, JTT and the T25 | — |
Countries
Ireland
Contacts
Public ContactDr. Christopher McGuigan
St. Vincent's University Hospital
Outcome results
None listed