The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease.

Last updated: February 13, 2025
Sponsor: Cellatoz Therapeutics, Inc
Overall Status: Completed

Phase

1

Condition

Peripheral Neuropathy

Neuropathy

Treatment

CLZ-2002

Clinical Study ID

NCT05947578
CLZ-CMT-101
  • Ages > 18
  • All Genders

Study Summary

A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1)

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Subjects aged 18 years or older

  • Subjects with a proven diagnosis of Charcot-Marie-Tooth disease type 1 at the timeof screening visit

  • Subjects who have muscle weakness in at least foot dorsiflexion (clinicalassessment) at the time of screening visit

  • Subjects with a CMT neuropathy score (CMTNS-v2) of 2 or more and 30 or fewer pointsat the time of screening visit

  • Subjects who can understand and are willing to sign a written informed consentdocument are willing to comply with all study procedures and schedule visits.

Exclusion

Exclusion Criteria:

  • Subjects who have any other neuromuscular diseases

  • Subjects who have undergone upper and lower limb bone surgery within six monthsbefore screening visit

  • Subjects who have concerns about muscle strength measurements due to the previoussurgery

  • Subjects who have severe active infection including severe/purulent cellulitis atthe injection sites at screening visit

  • Subjects who have a history of hospitalization due to hypersensitivity toantihistamines or allergy or hypersensitivity to certain substances such as food ordrugs

  • Subjects who have a history of unstable cardiovascular disease defined by thepresence of myocardial infarction (STEMI or NSTEMI) within 6 months before thescreening or the presence of unstable angina pectoris (in the case of increasedfrequency of symptoms, increased severity, or signs of prolonged symptoms atmoderate activity or rest)

  • Subjects who have a history of a transient ischemic attack (TIA) or stroke within 6months before screening visit

  • Subjects who have a positive HIV antibody test, hepatitis B antigen, or hepatitis Cantibody test result

  • Subjects who have a history of malignant tumors within 5 years before screeningvisit

  • Subjects who have received systemic steroids (inhaled steroids are allowed),immunotherapy, or cytotoxic therapy within 14 days before screening, or who areexpected to receive such treatment during the study period

  • Subjects who have participated in other clinical trials within 30 days beforescreening visit

  • Pregnant and lactating women or women of childbearing potential and men who plan apregnancy or are unwilling to use adequate birth control methods& until 30 daysafter the end of drug administration

Study Design

Total Participants: 9
Treatment Group(s): 1
Primary Treatment: CLZ-2002
Phase: 1
Study Start date:
June 19, 2023
Estimated Completion Date:
August 09, 2024

Study Description

This study is the First In Human (FIH) clinical trial for evaluating the safety and tolerability of IM injections of CLZ-2002 in patients with Charcot-Marie-Tooth disease (CMT) Type 1.

CLZ-2002 is the allogeneic mesenchymal stem cell-derived Neuronal Regeneration Promoting Cells. These cells are Schwann cell-like cells differentiated from tonsillar mesenchymal stem cells. CLZ-2002 helps the remyelination of the damaged peripheral nerves by restoring the myelin sheaths. It also induces the nerve regeneration and myelination pathways in the sciatic nerve and restores abnormal muscle tissues in Charcot-Marie-Tooth disease type 1 (CMT1).

Connect with a study center

  • Samsung Medical center

    Seoul, Gangnam-gu 06351
    Korea, Republic of

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.