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)
Overview
- Phase
- Phase 1
- Intervention
- CLZ-2002
- Conditions
- Charcot Marie Tooth Disease, Type 1
- Sponsor
- Cellatoz Therapeutics, Inc
- Enrollment
- 9
- Locations
- 1
- Primary Endpoint
- Safety and tolerability of intramuscular (IM) injections of CLZ-2002 in Participants
- Status
- Completed
- Last Updated
- last year
Overview
Brief 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)
Detailed 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).
Investigators
Eligibility Criteria
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
Arms & Interventions
CLZ-2002
CLZ-2002 injection is intramuscular in lower limbs on Day 1.
Intervention: CLZ-2002
Outcomes
Primary Outcomes
Safety and tolerability of intramuscular (IM) injections of CLZ-2002 in Participants
Time Frame: Day 1 (visit 2) to Week 24 (visit 6)
Frequency and percentage of treatment-emergent adverse events (TEAEs) and serious adverse events after injection through laboratory tests, physical examinations, vital signs, and electrocardiogram measurements conducted during the clinical trial.
Secondary Outcomes
- Changes from baseline in the overall neuropathy limitation scale (ONLS) at Weeks 4, 12 and 24(Weeks 4, 12 and 24)
- Changes from baseline in the velocity of Motor and Sensory nerve conduction at Weeks 4, 12, and 24(Weeks 4, 12, and 24)
- Changes from baseline in neurological examination at Weeks 1, 4, 12 and 24(Weeks 1, 4, 12 and 24)
- Changes from baseline in functional disability using the functional disability scale (FDS) at Weeks 4, 12, and 24(Weeks 4, 12, and 24)
- Changes from baseline in the 10-meter walk test at Weeks 4, 12, and 24(Weeks 4, 12, and 24)
- Change from baseline in the fatty infiltration level of lower limb muscles at Week 24.(Week 24)
- Changes from baseline in the disease severity of CMTNS-v2 score at Weeks 4, 12, and 24.(Weeks 4, 12 and 24)