Regulatory T Cells for Amyotrophic Lateral Sclerosis (REGALS)

Cellenkos

Status and phase: Active, not recruiting; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Biological: CK0803; Other: Excipient

Study type: Interventional

Funder types: Industry

Identifiers

NCT05695521

CK0803-101-1

Details and patient eligibility

About

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.

Full description

CK0803, neurotrophic allogenic T regulatory Cells (Treg), utilizes Cellenkos' proprietary CRANE technology to generate disease specific products. The primary objective of the upcoming phase 1 study is to establish safety and tolerability of multiple doses of CK0803 in ALS patients. The goal of the phase 1b study is to extend safety and establish efficacy of CK0803 in ALS using the combined assessment of function and survival (CAFS) that ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score.

Enrollment: 66 estimated patients

Sex: All

Ages: 18 to 95 years old

Volunteers: No Healthy Volunteers

Inclusion 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.

Exclusion criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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66 participants in 2 patient groups, including a placebo group

CK0803

Experimental group

Description:

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

Treatment:

Biological: CK0803

Placebo

Placebo Comparator group

Description:

Excipient

Treatment:

Other: Excipient