Clinical Trials Register

Summary
EudraCT Number:	2020-002438-34
Sponsor's Protocol Code Number:	FAM1
National Competent Authority:	Denmark - DHMA
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2020-06-11
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Denmark - DHMA

A.2

EudraCT number

2020-002438-34

A.3

Full title of the trial

Fampridine for treatment of residual neurological deficits in patients treated with immunoglobulins for chronic inflammatory demyelinating polyneuropathy

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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

A.4.1

Sponsor's protocol code number

FAM1

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Department of Neurology, Odense University Hospital
B.1.3.4	Country	Denmark
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Novo Nordisk Foundation
B.4.2	Country	Denmark
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Odense University Hospital
B.5.2	Functional name of contact point	Neuromuscular Clinic
B.5.3	Address:
B.5.3.1	Street Address	J. B. Winsløws Vej 4
B.5.3.2	Town/ city	Odense
B.5.3.3	Post code	5000
B.5.3.4	Country	Denmark
B.5.4	Telephone number	004565412471
B.5.6	E-mail	soeren.sindrup@rsyd.dk

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Fampyra
D.2.1.1.2	Name of the Marketing Authorisation holder	Biogen
D.2.1.2	Country which granted the Marketing Authorisation	Denmark
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Fampyra
D.3.4	Pharmaceutical form	Tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	FAMPRIDINE
D.3.9.1	CAS number	504-24-5
D.3.9.4	EV Substance Code	SUB07505MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Chronic inflammatory demyelinating polyneuropathy

E.1.1.1

Medical condition in easily understood language

Immune mediated polyneuropathy

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10072650
E.1.2	Term	CIDP
E.1.2	System Organ Class	100000004852

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

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).

E.2.2

Secondary objectives of the trial

Test if fampridine change ion channel function in CIDP

E.2.3

Trial contains a sub-study

E.3

Principal 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.

E.4

Principal 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.

E.5 End points

E.5.1

Primary end point(s)

Time to perform six-spot-step-test and nine-hole-pegboard-test.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline, two weeks, and four weeks (end of treatment).

E.5.2

Secondary end point(s)

Patients global impression of change.
Grip strength
Composite muscle strength score (MRC)
Sensory function (INCAT sensory sum score)
Timed-10-meter-walk
Functional performance score (ONLS og R-bODS)
Nerve conduction study
Nerve threshold tracking

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline, two weeks, and four weeks (end of treatment).
Besides threshold tracking and nerve conduction study are only performed af baseline and at four weeks.
Patient global impression of change is only performed af four weeks.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

Yes

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

Last visit last subject

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2020-07-02

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-08-19

P. End of Trial
P.	End of Trial Status	Ongoing

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