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Use of Dalfampridine in Primary Lateral Sclerosis

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ClinicalTrials.gov Identifier: NCT02868567
Recruitment Status : Recruiting
First Posted : August 16, 2016
Last Update Posted : March 2, 2020
Sponsor:
Collaborator:
Hospital for Special Surgery, New York
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Tracking Information
First Submitted Date  ICMJE March 17, 2016
First Posted Date  ICMJE August 16, 2016
Last Update Posted Date March 2, 2020
Study Start Date  ICMJE March 2016
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 14, 2016)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 4, 2016)
  • Effect of Dalfampridine on quality of life [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    ALSFRS-R,
  • Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    MSWS-12
  • Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    CGI, SGI
  • Effects of Dalfampridine on functional status [ Time Frame: over the course of study at weeks 2, 4, 6, 10, 14, 18 ]
    2MW, TUG
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: August 14, 2016)
Effect of Dalfampridine on quality of life [ Time Frame: over the course of study as weeks 2, 4, 6, 10, 14, 18 ]
McGill 1 point quality of life scale
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Use of Dalfampridine in Primary Lateral Sclerosis
Official Title  ICMJE A Multicenter, 18-week Open Label Safety and Efficacy Trial of Dalfampridine in Primary Lateral Sclerosis
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Motor Neuron Disease, Upper
Intervention  ICMJE Drug: dalfampridine
Pill open label
Other Name: ampyra
Study Arms  ICMJE Experimental: Ampyra
Ampyra open label
Intervention: Drug: dalfampridine
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 14, 2016)
35
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2021
Estimated Primary Completion Date January 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 99 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Mona Shahbazi, NP 2127742361 ext 6467978917 shahbazim@hss.edu
Contact: Shara Holzberg, MS 6467978592 ext 6467978917 holzbergs@hss.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02868567
Other Study ID Numbers  ICMJE 2016-247
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Weill Medical College of Cornell University
Study Sponsor  ICMJE Weill Medical College of Cornell University
Collaborators  ICMJE Hospital for Special Surgery, New York
Investigators  ICMJE
Principal Investigator: Dale Lange, MD HSS
PRS Account Weill Medical College of Cornell University
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP