4AP for Carpal Tunnel Syndrome (CTS)

NCT06294821 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: 00003845
• Study Type: interventional
• Target: 160
• Locations: 0 countries
• Sites: N/A

Brief 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. 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.

Conditions

Compression Neuropathy; Carpal Tunnel Syndrome

Keywords

4 aminopyridine; carpal tunnel syndrome; CTR; 4AP; CTS; Carpal tunnel release; compression neuropathy

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline (prior to study treatment) to 56 weeks post treatment.

Secondary Outcomes (1)

• Electrodiagnostic improvement following treatment

  Baseline (prior to study treatment) and 8 weeks post treatment.

Study Arms (2)

4-aminopyridine

ACTIVE COMPARATOR

Study drug: dalfampridine (generic) 10mg ER capsule

Drug: 4-Aminopyridine

Placebo

PLACEBO COMPARATOR

Placebo-1 capsule

Other: Placebo

Interventions

4-Aminopyridine DRUG

Subjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome

Also known as: 4AP

4-aminopyridine

Placebo OTHER

Subjects will receive either the active study drug (4-aminopyridine) or placebo to determine the effects of 4AP on carpal tunnel syndrome

Placebo

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• John Elfar: lead

MeSH Terms

Conditions

Tomaculous neuropathy; Carpal Tunnel Syndrome

Interventions

4-Aminopyridine

Condition Hierarchy (Ancestors)

Median Neuropathy; Mononeuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Nervous System Diseases; Nerve Compression Syndromes; Cumulative Trauma Disorders; Sprains and Strains; Wounds and Injuries

Intervention Hierarchy (Ancestors)

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

Study Officials

• John Elfar, MD

  University of Arizona

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Carly Deal

CONTACT

520-626-4656; carlyjdeal@arizona.edu

John Elfar, MD

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Tenured Professor, Chairman, Department of Orthopaedic Surgery

Study Record Dates

• First Submitted: February 21, 2024
• First Posted: March 6, 2024
• Study Start (Estimated): November 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): July 1, 2028
• Last Updated: January 26, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share