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

Cellatoz Therapeutics

Status and phase

Completed

Phase 1

Conditions

Charcot Marie Tooth Disease, Type 1

Treatments

Drug: CLZ-2002

Study type

Interventional

Funder types

Industry

Identifiers

NCT05947578

CLZ-CMT-101

Details and patient eligibility

About

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)

Full 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).

Enrollment

9 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Subjects aged 18 years or older
Subjects with a proven diagnosis of Charcot-Marie-Tooth disease type 1 at the time of screening visit
Subjects who have muscle weakness in at least foot dorsiflexion (clinical assessment) at the time of screening visit
Subjects with a CMT neuropathy score (CMTNS-v2) of 2 or more and 30 or fewer points at the time of screening visit
Subjects who can understand and are willing to sign a written informed consent document are willing to comply with all study procedures and schedule visits.

Exclusion criteria

Subjects who have any other neuromuscular diseases
Subjects who have undergone upper and lower limb bone surgery within six months before screening visit
Subjects who have concerns about muscle strength measurements due to the previous surgery
Subjects who have severe active infection including severe/purulent cellulitis at the injection sites at screening visit
Subjects who have a history of hospitalization due to hypersensitivity to antihistamines or allergy or hypersensitivity to certain substances such as food or drugs
Subjects who have a history of unstable cardiovascular disease defined by the presence of myocardial infarction (STEMI or NSTEMI) within 6 months before the screening or the presence of unstable angina pectoris (in the case of increased frequency of symptoms, increased severity, or signs of prolonged symptoms at moderate activity or rest)
Subjects who have a history of a transient ischemic attack (TIA) or stroke within 6 months before screening visit
Subjects who have a positive HIV antibody test, hepatitis B antigen, or hepatitis C antibody test result
Subjects who have a history of malignant tumors within 5 years before screening visit
Subjects who have received systemic steroids (inhaled steroids are allowed), immunotherapy, or cytotoxic therapy within 14 days before screening, or who are expected to receive such treatment during the study period
Subjects who have participated in other clinical trials within 30 days before screening visit
Pregnant and lactating women or women of childbearing potential and men who plan a pregnancy or are unwilling to use adequate birth control methods& until 30 days after the end of drug administration