Fampridine for treatment of functional disability in patients with immune mediated polyneuropathy

Phase 1

• Conditions: Chronic inflammatory demyelinating polyneuropathy; MedDRA version: 20.0Level: LLTClassification code 10072650Term: CIDPSystem Organ Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2020-002438-34-DK
• Lead Sponsor: Department of Neurology, Odense University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-11
• Last Update Posted: 23 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1.Age = 18 and = 80 years.
2.Diagnosis with CIDP or MGUS-associated demyelinating neuropathy fulfilling the criteria of European Federation of Neurological Societies.
3.Stable, ongoing treatment with SCIG
4.Written informed consent.
5.ONLS = 1 for the lower extremities and/or ONLS = 2 for the upper extremities
6.Average SSST for both legs > 8.6 s and/or average 9HPT for both hands > 23.0 s.
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 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1.Diagnosis of claudicatio intermittens or obvious vascular disease manifestation in the lower extremities at the discretion of the physician.
2.Clinically significant back disease or clinical sign of spinal root affection or spinal stenosis at the discretion of the physician.
3.Clinically significant systemic disease at the discretion of the physician.
4.Patient with other diseases that are expected to affect the ability to walk or the use of the arms at the discretion of the physician.
5.Patients who cannot cooperate or are unable to complete the project and patients who do not speak Danish or English.
6.Impaired kidney function (eGFR < 80 ml/min.)
7.Epilepsy or medical history of seizures.
8.Risk factors for seizures (reduced seizure threshold) either due to drug treatment (e.g. antipsychotics, antidepressants, anti-malaria drugs, tramadol, theophylline, sedating anti-histaminergic drugs) or other diseases (e.g. previous significant head trauma or diabetes with frequent episodes of significant hypoglycemia) as evaluated by an experienced neurologist.
9.Risk of important drug interactions (drugs inhibiting OCT2, e.g. cimetidine).
10.Pregnancy or lactation.
11.Women of child-bearing potential, unless they use an acceptable effective contraception measure during the study and at least 2 weeks after or their male partner, is vasectomized and their sole partner. Acceptable effective contraception is defined in the Clinical Trials Facilitation Group (CTFG) http://www.hma.eu/fileadmin/dateien/Human_Medicines/01-About_HMA/Working_Groups/CTFG/2014_09_HMA_CTFG_Contraception.pdf) and include intrauterine device or hormone-releasing system or hormonal contraception or total abstinence when this reflects their usual lifestyle or female sterilization (bilateral oophorectomy or total hysterectomy at least 6 weeks before). They should also have a negative pregnancy test.
12.Known allergy to fampridine or excipients.
13.Concurrent treatment with Fampyra or other medicinal products containing fampridine (4-aminopyridine)
14.Patients inappropriate for placebo.
15.Planned surgery.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The main purpose of the study is to determine the effect of treatment with the potassium-channel-inhibitor, fampridine, on clinically significant residual impairments and symptoms in patients with chronic inflammatory demyelinating polyneuropathy during stable treatment with subcutaneous immunoglobulin (SCIG).;Secondary Objective: Test if fampridine change ion channel function in CIDP;Primary end point(s): Time to perform six-spot-step-test and nine-hole-pegboard-test.;Timepoint(s) of evaluation of this end point: Baseline, two weeks, and four weeks (end of treatment).

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Patients global impression of change.<br>Grip strength<br>Composite muscle strength score (MRC)<br>Sensory function (INCAT sensory sum score)<br>Timed-10-meter-walk<br>Functional performance score (ONLS og R-bODS)<br>Nerve conduction study<br>Nerve threshold tracking;Timepoint(s) of evaluation of this end point: Baseline, two weeks, and four weeks (end of treatment).<br>Besides threshold tracking and nerve conduction study are only performed af baseline and at four weeks.<br>Patient global impression of change is only performed af four weeks.