Motor Neuron Disease, UpperPhase 1NCT02868567
Use of Dalfampridine in Primary Lateral Sclerosis
A Phase 1 study testing dalfampridine in people with Motor Neuron Disease, Upper, run by Hospital for Special Surgery, New York at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Motor Neuron Disease, Upper
- What is being tested?
- dalfampridine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 3 sitesUnited States
- Study dates
- Mar 2016to May 2025
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female, aged 18-99;
- 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.
- EMG within 3 months of enrollment with minimal or no evidence of lower motor neuron disease,
- Time from symptom onset > 18 months
- No previous allergy to dalfampridine
- No current or exposure to any therapeutic agent targeting PLS or ALS within 30 days of enrollment.
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- Must have a forced vital capacity (FVC) ≥ 60% of expected
- Written informed consent prior to screening is present.
- Subjects on a stable dose of or have not taken Riluzole for at least thirty days
- Impaired walking as measured by a Hauser Index of greater than 1 and less than 7 (2 to 6, inclusive);
- Mini Mental Status Score > 22 and deemed by the PI of being capable of providing informed consent and following trial procedures.
- Geographically accessible to the site.
- 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.
Who cannot join
- 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;
- Unwillingness to sign informed consent or any other reasons for which the investigator feels the subject cannot complete the study;
- Women who are pregnant, breastfeeding, or trying to become pregnant;
- Active cancer within the previous 2 years, except treated basal cell carcinoma of the skin;
- Subjects taking any other experimental drugs within 30 days prior to enrollment;
- 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.
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- Patient has moderate or severe renal impairment as defined by a calculated creatinine clearance of ≤50 mL/minute;
- 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;
- 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);
- Patient has a history of drug or alcohol abuse within the past year;
- Patient has clinically significant abnormal laboratory values.
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Mass General Hospital
Boston, Massachusetts, 02114
University of Florida Gainsville
Gainesville, Florida, 32607
Shara Holzberg
New York, New York, 10021
About this study
Official title: A Multicenter, 18-week Open Label Safety and Efficacy Trial of Dalfampridine in Primary Lateral Sclerosis
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.
- Conditions
- Motor Neuron Disease, Upper
- Being tested
- dalfampridine
- Medical subject headings
- Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dalfampridineDrug
Also called ampyra
Pill open label
| Study group | Role | What they receive |
|---|---|---|
| Ampyra | Experimental | Ampyra open label Receives: Drug: dalfampridine |
What is being measured
The primary outcome is the main question the study is designed to answer.
consistent improvement in the Timed 25 Foot Walk test
speed of walking 25 feet
Measured: over the duration of the study at week 2, 4, 6, 10, 14, 18
Secondary outcomes (5)
Effect of Dalfampridine on quality of life
ALSFRS-R,
Measured: over the course of study at weeks 2, 4, 6, 10, 14, 18
Effects of Dalfampridine on functional status
MSWS-12
Measured: over the course of study at weeks 2, 4, 6, 10, 14, 18
Effects of Dalfampridine on functional status
CGI, SGI
Measured: over the course of study at weeks 2, 4, 6, 10, 14, 18
Effects of Dalfampridine on functional status
2MW, TUG
Measured: over the course of study at weeks 2, 4, 6, 10, 14, 18
Effects of Dalfampridine on functional status
PPT, Hand and Foot tapping.
Measured: over the course of study at weeks 2, 4, 6, 10, 14, 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2016 (estimated)
- Study startMar 2016
- Primary completionMay 2025
- Study completionMay 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Motor Neuron Disease, UpperStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital for Special Surgery, New York
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dale Lange, MD, Principal investigator, HSS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02868567
- Sponsor's protocol ID
- 2016-247
- Status last verified
- Sep 2024
- First submitted
- 17 Mar 2016
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No