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Use of Dalfampridine in Primary Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT02868567
Recruitment Status : Active, not recruiting
First Posted : August 16, 2016
Last Update Posted : October 3, 2022
Information provided by (Responsible Party):
Hospital for Special Surgery, New York

Brief Summary:
This study will comprise an 18-week open label safety and tolerability trial. In this study, a total of 35 subjects with primary lateral sclerosis PLS or upper motor neuron predominate ALS will be enrolled. At the initial screening evaluation, a baseline T25FW will be obtained. This baseline test will be repeated at weeks 2, 4, 6, 10, 14 18. The validity of this measure was shown in MS studies when compared to the MSWS-12 (12 item walking scale) and CGI (clinical global impression) scales (35-37). A consistent responder will be defined as improvement in 3 of 4 Timed 25Foot Walk while on medication, compared with the baseline results while off medication.

Condition or disease Intervention/treatment Phase
Motor Neuron Disease, Upper Drug: dalfampridine Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 35 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, 18-week Open Label Safety and Efficacy Trial of Dalfampridine in Primary Lateral Sclerosis
Study Start Date : March 2016
Estimated Primary Completion Date : July 2023
Estimated Study Completion Date : July 2023

Arm Intervention/treatment
Experimental: Ampyra
Ampyra open label
Drug: dalfampridine
Pill open label
Other Name: ampyra

Primary Outcome Measures :
  1. consistent improvement in the Timed 25 Foot Walk test [ Time Frame: over the duration of the study at week 2, 4, 6, 10, 14, 18 ]
    speed of walking 25 feet

Secondary Outcome Measures :
  1. Effect of Dalfampridine on quality of life [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]

  2. Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]

  3. Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    CGI, SGI

  4. Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    2MW, TUG

  5. Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    PPT, Hand and Foot tapping.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 99 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female, aged 18-99;
  2. Diagnosis of upper motor neuron disease, compatible with PLS but may include upper motor neuron (UMN) predominant ALS, defined as only upper motor neuron (UMN) features in at least 2 body regions on examination.
  3. EMG within 3 months of enrollment with minimal or no evidence of lower motor neuron disease,
  4. Time from symptom onset > 18 months
  5. No previous allergy to dalfampridine
  6. No current or exposure to any therapeutic agent targeting PLS or ALS within 30 days of enrollment.
  7. Must have a forced vital capacity (FVC) ≥ 60% of expected
  8. Written informed consent prior to screening is present.
  9. Subjects on a stable dose of or have not taken Riluzole for at least thirty days
  10. Impaired walking as measured by a Hauser Index of greater than 1 and less than 7 (2 to 6, inclusive);
  11. Mini Mental Status Score > 22 and deemed by the PI of being capable of providing informed consent and following trial procedures.
  12. Geographically accessible to the site.
  13. Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for ≥ 3 months, barrier method in conjunction with spermicide, or another adequate method.

Exclusion Criteria:

  1. History of clinically significant liver disease, renal disease, peripheral neuropathy, serious peripheral vascular disease, known HSP or + C9orf72 or SPG4 mutation, or any other medical condition felt to be exclusionary by the investigator;
  2. Unwillingness to sign informed consent or any other reasons for which the investigator feels the subject cannot complete the study;
  3. Women who are pregnant, breastfeeding, or trying to become pregnant;
  4. Active cancer within the previous 2 years, except treated basal cell carcinoma of the skin;
  5. Subjects taking any other experimental drugs within 30 days prior to enrollment;
  6. Patient has any history of seizures; brain surgery, brain implants, any metallic implants above the neck, cardiac pacemakers, cochlear implants, piercing or body modification above the neck, known history of TMS related complications or side-effects, tinnitus.
  7. Patient has moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute;
  8. Patient has been administered botulinum toxin in the lower extremities within 6 months prior to the screening visit and/or is expected to receive botulinum toxin in the lower extremities during the course of the study;
  9. Patient has a known allergy to pyridine-containing substances or any of the inactive ingredients of the dalfampridine tablet (colloidal silicon dioxide, hydroxypropyl methylcellulose, magnesium stearate, microcrystalline cellulose, polyethylene glycol, and titanium dioxide);
  10. Patient has a history of drug or alcohol abuse within the past year;
  11. Patient has clinically significant abnormal laboratory values.
  12. Anything else that, in the opinion of the SI, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02868567

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United States, Florida
University of Florida Gainsville
Gainesville, Florida, United States, 32607
United States, Massachusetts
Mass General Hospital
Boston, Massachusetts, United States, 02114
United States, New York
Shara Holzberg
New York, New York, United States, 10021
Sponsors and Collaborators
Hospital for Special Surgery, New York
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Principal Investigator: Dale Lange, MD HSS
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Responsible Party: Hospital for Special Surgery, New York
ClinicalTrials.gov Identifier: NCT02868567    
Other Study ID Numbers: 2016-247
First Posted: August 16, 2016    Key Record Dates
Last Update Posted: October 3, 2022
Last Verified: September 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action