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Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

(SYNAPSE-CMT)

Sponsored by NMD Pharma A/S

Quick Self-Assessment

See if you qualify and express your interest

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Current Status

Completed

Auto-verified from ClinicalTrials.gov

Verified Nov 12, 2025

Phase

Clinical Trial

Sponsor

NMD Pharma A/S

Enrollment Target

80

Start Date

Sep 2024

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1 of 80 participants interested
1% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

This Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.

Who Can Participate

Inclusion Criteria

Male or female participants must be 18 to 70 years inclusive at the time of signing the ICF.
Diagnosis of CMT type 1 or 2 confirmed by genetic testing.
Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Participant is capable of and has given signed informed consent

Exclusion Criteria

Participants with other significant disease that may interfere with the interpretation of study data (e.g., other neuromuscular diseases) and/or ability to complete the tests, in the opinion of the Investigator.
Participants with laboratory test result abnormalities at screening considered clinically significant by the Investigator.
Participants who have received treatment with another IMP within 30 days (or 5 half-lives of the medication, whichever is longer)

❓ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

80 participants

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Age Range

18 Years - 70 Years

⚧

Gender

ALL

🏒

Sponsor

NMD Pharma A/S

Study Locations

Choose your preferred location or select flexible during enrollment

Unknown

Kansas City

Kansas

Location available
View Kansas City location page
Unknown

Boston

Massachusetts

Location available
View Boston location page
Unknown

Columbia

Missouri

Location available
View Columbia location page
Unknown

New York

New York

Location available
View New York location page
Unknown

Rochester

New York

Location available
View Rochester location page
Unknown

Columbus

Ohio

Location available
View Columbus location page
Unknown

Austin

Texas

Location available
View Austin location page
Unknown

Spokane

Washington

Location available
View Spokane location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

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Looking for Charcot-Marie-Tooth Disease Treatment in Kansas City?

Join others in Kansas exploring innovative treatment options through clinical research

Charcot-Marie-Tooth Disease Treatment Options in Kansas City, Kansas

If you're searching for Charcot-Marie-Tooth Disease treatment in Kansas City, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Kansas City, Boston, Columbia and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Charcot-Marie-Tooth Disease. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Kansas
Now Enrolling
Up to 80 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Charcot-Marie-Tooth Disease?

Potential Benefits

  • βœ“Access to new treatment approaches before public availability
  • βœ“Close monitoring by experienced medical professionals
  • βœ“Study-related care provided at no cost
  • βœ“Contribute to medical research for Charcot-Marie-Tooth Disease

What to Expect

  • β†’Initial screening to determine eligibility
  • β†’Regular check-ups and monitoring visits
  • β†’Possible compensation for time and travel
  • β†’You can withdraw at any time

Frequently Asked Questions About This Charcot-Marie-Tooth Disease Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06482437. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.