Compression NeuropathyPhase 2CTSNCT06294821
4AP for Carpal Tunnel Syndrome (CTS)
A Phase 2 study testing 4-Aminopyridine in people with Compression Neuropathy and Carpal Tunnel Syndrome, run by John Elfar.
Trial summary
The investigators have found recent promising data supporting the use of a currently FDA approved drug, 4-aminopyridine, in the treatment of nerve injury including compression neuropathy. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Compression Neuropathy, Carpal Tunnel Syndrome
- What is being tested?
- 4-Aminopyridine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 160 participants
- Where
- No sites listed yet
- Study dates
- Nov 2026to Jul 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients diagnosed with non-traumatic carpal tunnel syndrome without any other confounding diagnosis (e.g. cubital tunnel syndrome, cervical nerve compression)
- Standard of care EDX testing within 6 months of study enrollment
- Cognitive ability to report sensory and motor deficit during examination.
- Able to complete twice daily dosing of 4-AP for the entire 6 week treatment period.
- Adults subject aged 18-90
- Ability to give written informed consent.
- Capable of safely undergoing electrodiagnostic testing (EDX).
- Availability for all study visits.
Who cannot join
- Inability to complete twice daily dosing during 6 week treatment period.
- History of multiple sclerosis, stroke or any other diagnosed neurological disorder
- History of hypersensitivity to AMPYRA® or 4-aminopyridine
- Current use of aminopyridine medications, including other compounded 4-AP
- Suspected renal impairment based on the Choyke questionnaire.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
John Elfar, MD contact
About this study
Official title: 4-Aminopyridine to Delay Carpal Tunnel Release or Resolve Carpal Tunnel Syndrome
The investigators have found recent promising data supporting the use of a currently FDA approved drug, 4-aminopyridine, in the treatment of nerve injury including compression neuropathy. The purpose of this research is determine whether 4-aminopyridine can treat carpal tunnel syndrome or delay the need for formal surgical release in patients with known carpal tunnel syndrome who would otherwise undergo surgery.
Read the detailed description
Carpal tunnel syndrome affects somewhere between 3 and 10% of the Unites States population. Symptoms associated with this disorder include numbness and tingling in the radial three digits of the hand. These symptoms often cause sleeplessness. They have been attributed to work related activities. The diagnosis of carpal tunnel syndrome is made clinically. Several confirmatory tests exist in the setting of carpal tunnel syndrome. In the United States the gold standard diagnostic for carpal tunnel syndrome is electrodiagnostic evaluation. This notwithstanding, recent literature suggests that other diagnostic methods including ultrasound evaluation of the median nerve at the carpal tunnel as well as clinical diagnostic criteria are sufficient to adequately and specifically diagnose carpal tunnel syndrome.
Several treatments exist for carpal tunnel syndrome. Treatment range from non-operative braces or positional movements. Moving on to intervention treatments there are several injections that have been used to treat carpal tunnel syndrome. Carpal Tunnel Syndrome is most definitively and completely treated by surgical release of the median nerve at the carpal tunnel. Surgical releases themselves have been performed under a variety of techniques from formal median nerve exploration to mini-open release as well endoscopic release. Resolution of symptoms after carpal tunnel release has been regarded as the gold standard to which other treatments can be compared.
Several registries of compression neuropathy patients have been compiled. These registries reveal that non-invasive assessment with standardize validated questionnaires can be used to measure the progression of symptomology, and the resolution of treatment. Given the history and wealth of experience in the diagnosis and treatment in this condition, it's natural to ask are there non-operative pharmacological treatments that may delay or remove the need for carpal tunnel release surgery. Certainly, the comparison to surgical decompression as the gold standard is desirable.
More research is needed to evaluate the role of 4-aminopyridine (4AP) in the treatment of compression neuropathy. The investigational treatment will be used to test the hypothesis that 4AP improves symptoms and electrodiagnostic parameters in carpal tunnel syndrome patients.
- Conditions
- Compression Neuropathy, Carpal Tunnel Syndrome
- Being tested
- 4-Aminopyridine, Placebo
- Keywords
- 4 aminopyridine, carpal tunnel syndrome, CTR, 4AP, CTS, Carpal tunnel release, compression neuropathy
- Medical subject headings
- Tomaculous neuropathy, Carpal Tunnel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-AminopyridineDrug
Also called 4AP
Subjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome
PlaceboOther
Subjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome
| Study group | Role | What they receive |
|---|---|---|
| 4-aminopyridine | Active comparator | Study drug: dalfampridine (generic) 10mg ER capsule Receives: Drug: 4-Aminopyridine |
| Placebo | Placebo comparator | Placebo-1 capsule Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Results of symptomatic improvement in the Boston Carpal Tunnel Syndrome Questionnaire (BCTSQ)
Improvement of Carpal Tunnel Syndrome symptoms will be measured using the Boston Carpal Tunnel Syndrome Questionnaire (BCTSQ) - a validated patient reported outcome measure. The scale contains 6 items on the severity and frequency of night and daytime numbness, tingling and pain. Each item is scored on a 5-point Likert scale from 1 (no symptoms) to 5 (most severe symptoms). Subjects will complete a baseline questionnaire prior to treatment initiation. The questionnaire will be completed all study visit following initiation of treatment until the final study visit at 56 weeks post treatment.
Measured: Baseline (prior to study treatment) to 56 weeks post treatment
Results of symptomatic improvement in the Michigan Hand Outcomes Questionnaire (MHQ)
Improvement of Carpal Tunnel Syndrome symptoms will be measured with standardized validated outcomes measure, the Michigan Hand Outcomes Questionnaire (MHQ) for improvement in carpal tunnel symptomatology. The MHQ is a tool used to assess patients with hand disorders through the measurement of 6 health domains: overall hand function, activities of daily living, pain, work performance, aesthetics, and patient satisfaction. In the pain scale, high scores indicate greater pain, while in the other five scales, high scores denote better hand performance. Baseline measures will be completed prior to treatment and repeated at each study visit with the final outcome questionnaire completed at the final, 56 week visit.
Measured: Baseline (prior to study treatment) to 56 weeks post treatment.
Secondary outcomes (1)
Electrodiagnostic improvement following treatment
Nerve conduction velocity improvement > 10% compared to pre-treatment nerve conduction velocity. Electrodiagnostic studies will be completed as part of standard of care prior to initiation of treatment and repeated at 8 weeks post treatment.
Measured: Baseline (prior to study treatment) and 8 weeks post treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2024
- Study startNov 2026 (estimated)
- Primary completionDec 2027 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Compression NeuropathyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Lead sponsor
- John Elfar
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, John Elfar, University of Arizona
- Investigators
- John Elfar, MD, Principal investigator, University of Arizona
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06294821
- Sponsor's protocol ID
- 00003845
- Acronym
- CTS
- Status last verified
- Jan 2026
- First submitted
- 21 Feb 2024
- Last updated
- 26 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 4-Aminopyridine to Delay Carpal Tunnel Release or Resolve Carpal Tunnel Syndrome
- ClinicalTrials.gov ID
- NCT06294821
- Acronym / sponsor ID
- CTS / 00003845
- Conditions
- Compression Neuropathy, Carpal Tunnel Syndrome
- Keywords
- 4 aminopyridine, carpal tunnel syndrome, CTR, 4AP, CTS, Carpal tunnel release, compression neuropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- John ElfarUniversity, hospital or other
- Study start
- Nov 2026 (estimated)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 6 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigators have found recent promising data supporting the use of a currently FDA approved drug, 4-aminopyridine, in the treatment of nerve injury including compression neuropathy. The purpose of this research is determine whether 4-aminopyridine can treat carpal tunnel syndrome or delay the need for formal surgical release in patients with known carpal tunnel syndrome who would otherwise undergo surgery.
Detailed description
Carpal tunnel syndrome affects somewhere between 3 and 10% of the Unites States population. Symptoms associated with this disorder include numbness and tingling in the radial three digits of the hand. These symptoms often cause sleeplessness. They have been attributed to work related activities. The diagnosis of carpal tunnel syndrome is made clinically. Several confirmatory tests exist in the setting of carpal tunnel syndrome. In the United States the gold standard diagnostic for carpal tunnel syndrome is electrodiagnostic evaluation. This notwithstanding, recent literature suggests that other diagnostic methods including ultrasound evaluation of the median nerve at the carpal tunnel as well as clinical diagnostic criteria are sufficient to adequately and specifically diagnose carpal tunnel syndrome.
Several treatments exist for carpal tunnel syndrome. Treatment range from non-operative braces or positional movements. Moving on to intervention treatments there are several injections that have been used to treat carpal tunnel syndrome. Carpal Tunnel Syndrome is most definitively and completely treated by surgical release of the median nerve at the carpal tunnel. Surgical releases themselves have been performed under a variety of techniques from formal median nerve exploration to mini-open release as well endoscopic release. Resolution of symptoms after carpal tunnel release has been regarded as the gold standard to which other treatments can be compared.
Several registries of compression neuropathy patients have been compiled. These registries reveal that non-invasive assessment with standardize validated questionnaires can be used to measure the progression of symptomology, and the resolution of treatment. Given the history and wealth of experience in the diagnosis and treatment in this condition, it's natural to ask are there non-operative pharmacological treatments that may delay or remove the need for carpal tunnel release surgery. Certainly, the comparison to surgical decompression as the gold standard is desirable.
More research is needed to evaluate the role of 4-aminopyridine (4AP) in the treatment of compression neuropathy. The investigational treatment will be used to test the hypothesis that 4AP improves symptoms and electrodiagnostic parameters in carpal tunnel syndrome patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 4-aminopyridine | Active comparator | Drug: 4-AminopyridineStudy drug: dalfampridine (generic) 10mg ER capsule |
| Placebo | Placebo comparator | Other: PlaceboPlacebo-1 capsule |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patients diagnosed with non-traumatic carpal tunnel syndrome without any other confounding diagnosis (e.g. cubital tunnel syndrome, cervical nerve compression)
- Standard of care EDX testing within 6 months of study enrollment
- Cognitive ability to report sensory and motor deficit during examination.
- Able to complete twice daily dosing of 4-AP for the entire 6 week treatment period.
- Adults subject aged 18-90
Key exclusion criteria (5 listed)
- Inability to complete twice daily dosing during 6 week treatment period.
- History of multiple sclerosis, stroke or any other diagnosed neurological disorder
- History of hypersensitivity to AMPYRA® or 4-aminopyridine
- Current use of aminopyridine medications, including other compounded 4-AP
- Suspected renal impairment based on the Choyke questionnaire.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4-AminopyridineAlso: 4APSubjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome | Drug | 4-aminopyridine |
| PlaceboSubjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome | Other | Placebo |
Primary outcome
Results of symptomatic improvement in the Boston Carpal Tunnel Syndrome Questionnaire (BCTSQ)
Time frame: Baseline (prior to study treatment) to 56 weeks post treatment
Improvement of Carpal Tunnel Syndrome symptoms will be measured using the Boston Carpal Tunnel Syndrome Questionnaire (BCTSQ) - a validated patient reported outcome measure. The scale contains 6 items on the severity and frequency of night and daytime numbness, tingling and pain. Each item is scored on a 5-point Likert scale from 1 (no symptoms) to 5 (most severe symptoms). Subjects will complete a baseline questionnaire prior to treatment initiation. The questionnaire will be completed all study visit following initiation of treatment until the final study visit at 56 weeks post treatment.
Results of symptomatic improvement in the Michigan Hand Outcomes Questionnaire (MHQ)
Time frame: Baseline (prior to study treatment) to 56 weeks post treatment.
Improvement of Carpal Tunnel Syndrome symptoms will be measured with standardized validated outcomes measure, the Michigan Hand Outcomes Questionnaire (MHQ) for improvement in carpal tunnel symptomatology. The MHQ is a tool used to assess patients with hand disorders through the measurement of 6 health domains: overall hand function, activities of daily living, pain, work performance, aesthetics, and patient satisfaction. In the pain scale, high scores indicate greater pain, while in the other five scales, high scores denote better hand performance. Baseline measures will be completed prior to treatment and repeated at each study visit with the final outcome questionnaire completed at the final, 56 week visit.
1 secondary outcome
Electrodiagnostic improvement following treatment
Time frame: Baseline (prior to study treatment) and 8 weeks post treatment.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
John Elfar, MD contact
John Elfar, MD principal investigator
University of Arizona
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 4-Aminopyridine in people with Compression Neuropathy and Carpal Tunnel Syndrome, run by John Elfar.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 26 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Carly Deal (520-626-4656, carlyjdeal@arizona.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Results of symptomatic improvement in the Boston Carpal Tunnel Syndrome Questionnaire (BCTSQ) (time frame: Baseline (prior to study treatment) to 56 weeks post treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jan 2026.