Regulatory T Cells for Amyotrophic Lateral Sclerosis

NCT05695521 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CK0803-101-1
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Cellenkos; T Regulatory Cells; Randomized Placebo Control Trial; Umbilical cord blood; Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (2)

• Treatment Limiting Toxicity (TLT)

  TLT of CK0803 as assessed by the incidence and severity of AE and SAEs using NCI-CTCAE Version 5.0 criteria. TLT is a primary endpoint for bothe phase 1 safety run-in and phase 1b RCT part

  28 days

• Combined assessment of function and survival (CAFS)

  CAFS ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score where score = 0 is worst and score = 48 is best. Each patient's outcome is compared to every other patient's outcome, assigned a score, and the summed scores are ranked. The mean rank score for each treatment group can then be calculated. A higher mean CAFS score indicates a better group outcome.

  24 weeks

Secondary Outcomes (9)

• Incidence of all cause AEs and SAEs

  baseline and at weeks 1, 2, 3, 4, 5, 8, 12 and/or 13, 16, 20, 24 and/or 25, 36 and 48 from first infusion and/or at end of treatment (EOT)

• ALS Functional Rating Scale-Revised (ALSFRS-R) Score

  Each measured at baseline and at weeks 5, 8, 12 and/or 13, 16, 20, 24 and/or 25, 36 and 48 from first infusion and/or at EOT

• Amyotrophic Lateral Sclerosis Specific Quality of Life - Revised (ALSSQOL-R)

  Each measured at baseline and at weeks 5, 8, 12 and/or 13, 16, 20, 24 and/or 25, 36 and 48 from first infusion and/or at EOT

• Slow Vital Capacity (SVC)

  Each measured at baseline and at weeks 5, 8, 12 and/or 13, 16, 20, 24 and/or 25, 36 and 48 from first infusion and/or at EOT

• Handheld dynamometer (HHD)

  Each measured at baseline and at weeks 5, 8, 12 and/or 13, 16, 20, 24 and/or 25, 36 and 48 from first infusion and/or at EOT

Study Arms (2)

CK0803

EXPERIMENTAL

CK0803 (cryopreserved, allogeneic, cord blood derived T regulatory cells that express neurotropic homing markers) will be administered intravenously Dose: 100 million Treg cells (fixed dose) Dose regimen: * Induction: one infusion every 7 days (+/-3) x 4 doses * Consolidation: one infusion every 28 days (+/-3) x 5 doses

Biological: CK0803

Placebo

PLACEBO COMPARATOR

Excipient

Other: Excipient

Interventions

CK0803 BIOLOGICAL

CK0803 (cryopreserved, allogeneic, cord blood derived T regulatory cells that express neurotropic homing markers) will be administered intravenously

CK0803

Excipient OTHER

Excipient

Placebo

Eligibility Criteria

• Age: 18 Years - 95 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
• 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.

Sponsors & Collaborators

• Cellenkos, Inc.: lead

Study Sites (3)

Columbia University Irving Medical Center

New York, New York, 10033, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Michael E. DeBakey Veterans Affairs Medical Center

Houston, Texas, 77030, United States

Location

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  BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Excipients

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pharmaceutical Vehicles; Pharmaceutic Aids; Pharmaceutical Preparations; Specialty Uses of Chemicals; Chemical Actions and Uses

Study Officials

• Neil Shneider, MD, PhD

  Columbia University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double Blinded, Placebo Control
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a randomized two-arm, multi-center phase Ib trial of CK0803 (cryopreserved, allogeneic, cord blood derived T regulatory cells that express neurotropic homing markers) versus placebo for treatment of adults with amyotrophic lateral sclerosis (ALS) having onset within 5 years of trial entry. This trial will consist of a 6-patient safety run-in followed by a randomized comparative trial with up to two stages (stages 1 and 2), each stage of size 30 patients, with a safety rule applied after stage 1 that may terminate the trial early, for a maximum total sample size of 6+30+30 = 66 patients.

Study Record Dates

• First Submitted: January 3, 2023
• First Posted: January 25, 2023
• Study Start: April 3, 2023
• Primary Completion: May 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: April 11, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)