Study Purpose:Phase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients with Amyotrophic Lateral Sclerosis.
Amyotrophic Lateral Sclerosis
Type of Intervention:
Study Chair(s)/Principal Investigator(s):
Neil Shneider, MD, PhD, Columbia University
Coordinating Center Contact Information
Full Study Summary:
Estimated Study Start Date:
04 / 03 / 2023
Estimated Study Completion Date:
12 / 01 / 2027
Posting Last Modified Date:
04 / 06 / 2023
Date Study Added to neals.org:
01 / 25 / 2023
95 YearsInclusion Criteria:
- Ability of the subject or his/her legally authorized representative to provide informed consent.
- Adult ALS subjects (≥18 years of age)
- Diagnosis of ALS, according to the Revised El Escorial Criteria for ALS
- Subjects with disease onset ≤ 5 years
- Upright (sitting position) Slow Vital Capacity (SVC) as adjusted for sex, age and height ≥ 50% predicted
- Subjects must have documented ALSFRSR score of 36-45 at baseline.
- Subjects taking concomitant Riluzole or Edaravone or Albrioza at study entry must be on a stable dose for ≥ 30 days prior to the first dose of study treatment (Day 1).
- Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.
- Agree to practice highly effective contraception during the study and continue contraception for 90 days after their last dose of study treatment.
- Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol medical monitor is the final arbiter of eligibility.
- Antiplatelet or anticoagulant therapy within the 14 days prior to Day 1 or anticipated use during the study, including but not limited to daily aspirin including low dose aspirin (defined as ≤ 150 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban
- Clinically significant low platelet count (defined as < 100,000/mm3), coagulation tests, or laboratory abnormalities that would render a subject unsuitable for inclusion
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- Have any other conditions, which, in the opinion of the Investigator would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the study
- Concurrent participation in any other interventional clinical study
- Treatment with another investigational drug, biological agent, or device, including, but not limited to sodium phenylbutyrate, within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer
- Treatment of cancer in the last 5 years (except in situ carcinoma of the cervix or basal cell carcinoma)
- Female subjects who are pregnant or currently breastfeeding
- Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the subject unsuitable for enrollment.
Columbia University Irving Medical Center | Recruiting
Neil Shneider, MD, PhD
Principal Investigator : Neil Shneider, MD, PhD
New York, New York