Effect of Dalfampridine in Patients With Hereditary Spastic Paraplegia

NCT05613114 | Completed

• 1 other identifier: 45/20
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

There are limited but encouraging results supporting the use of dalfampridine in patients with hereditary spastic paraplegia. The investigators aimed to investigate the effects of dalfampridine on walking speed, muscle length, spasticity, functional strength, and functional mobility in patients with hereditary spastic paraplegia. In this triple-blinded, randomized, placebo-controlled trial, 4 patients with hereditary spastic paraplegia received dalfampridine (10 mg twice daily) plus physiotherapy (2 times per week), and 4 patients received placebo plus physiotherapy for a total duration of 8 weeks. The assessor and treating physiotherapists, and patients were masked to the group allocation. The primary outcome was Timed 25-foot Walk Test at the end of the 8-week treatment. The secondary outcome measures were functional mobility, functional muscle strength, muscle length, and spasticity.

Conditions

Hereditary Spastic Paraplegia

Outcome Measures

Primary Outcomes (1)

• Timed 25-foot Walk Test

  The Timed 25-foot walk test (T25FW) is considered the "best characterized objective measure of walking disability and can be used across a wide range of walking disabilities". For the T25-FW, patients were instructed to walk as fast as they could in a safemanner along amarked 25-foot course. The time in seconds to complete each test was recorded, and the test was immediately repeated.

  Change from baseline to week 8

Secondary Outcomes (4)

• Sit to Stand Test

  Change from baseline to week 8

• Timed Up and Go test

  Change from baseline to week 8

• Modified Ashworth Scale

  Change from baseline to week 8

• Muscle Length Measurement

  Change from baseline to week 8

Study Arms (2)

Medication

EXPERIMENTAL

Dalfampridine plus physiotherapy

Drug: Dalfampridine 10 MG; Behavioral: Physiotherapy

No Medication

PLACEBO COMPARATOR

Placebo plus physiotherapy

Drug: Placebo; Behavioral: Physiotherapy

Interventions

Dalfampridine 10 MG DRUG

The participants in the experimental group received dalfampridine administered as 10 mg extended-release tablets every twelve hours for 8 weeks.

Medication

Placebo DRUG

Control group received a placebo drug with the same administration method (2 times per week for the total duration of 8 weeks).

No Medication

Physiotherapy BEHAVIORAL

Conventional physiotherapy program including stretching and flexibility, strengthening, walking and balance exercises which were mainly focused on the lower limbs and improving walking. The program was applied 2 times per week for the total duration of 8 weeks.

Medication; No Medication

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of Hereditary Spastic Paraplegia at least 1 year ago

You may not qualify if:

• Having another neurological disorder
• An orthopedic deformity in the lower extremity
• Having a serious cognitive impairment

Sponsors & Collaborators

• European University of Lefke: lead

Study Sites (1)

Dr. Burhan Nalbantoğlu State Hospital

Nicosia, Cyprus

Location

Related Publications (1)

• Selcuk Muhtaroglu F, Belgen Kaygisiz B, Usar Incirli S, Kahraman T. Dalfampridine as a promising agent in the management of hereditary spastic paraplegia: A triple-blinded, randomized, placebo-controlled pilot trial. J Clin Neurosci. 2023 Nov;117:136-142. doi: 10.1016/j.jocn.2023.09.026. Epub 2023 Oct 5.

  PMID: 37804674 DERIVED

MeSH Terms

Conditions

Spastic Paraplegia, Hereditary

Interventions

4-Aminopyridine; Physical Therapy Modalities

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Therapeutics; Rehabilitation

Study Officials

• Ferda Selcuk

  European University of Lefke

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assist Prof. Dr.

Study Record Dates

• First Submitted: November 4, 2022
• First Posted: November 14, 2022
• Study Start: August 3, 2020
• Primary Completion: March 12, 2021
• Study Completion: March 12, 2021
• Last Updated: November 22, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

CY (1)