ALS REGALS Trial Continues to Evaluate Cellenkos’ CK0803 Treg Therapy


The trial is set to dose its second cohort following a positive DSMB recommendation in March.

Phase 1b REGALS Trial of CK0803 for ALS

(Click to enlarge)

CK0803 is a novel, allogeneic, neurotrophic, clinical-grade umbilical cord blood-derived cell therapy that consists of regulatory T-cells developed by Cellenkos. The cells have a high cell surface expression of CD11a that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia in patients with amyotrophic lateral sclerosis (ALS).

CK0803 is being evaluated in patients with ALS in the phase 1 REGALS trial (NCT05695521). The first patient was dosed in the REGALS trial in May 2023.1 Cellenkos announced that the trial was proceeding to dose its second cohort following a positive recommendation by the Data Safety Monitoring Board (DSMB) in March 2024.2

"We are very excited and encouraged by this development," Tara Sadeghi, Chief Operating Officer, Cellenkos, said in a statement.2 "This recommendation from the DSMB is an important step in bringing this potential disease modifying therapy to the patients and takes us closer to our ultimate goal of curing ALS."

Neil Shneider, MD, PhD, associate professor, neurology and director, Eleanor and Lou Gehrig ALS Center, Columbia

Neil Shneider, MD, PhD

Credit: Columbia

The positive DSMB recommendation came after no serious adverse events (AEs) were observed after therapy. The REGALS trial will continue to enroll in its safety run-in study before proceeding to a phase 1b, randomized, double blind, placebo control trial of CK0803 that will access 4 weekly infusions followed by 5 monthly infusions in participants with ALS.

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The trial’s primary outcome measures are treatment limiting toxicities up to 28 days after treatment and a combined assessment of function and survival (CFAS) for up to 24 weeks after treatment. The CFAS is based off of survival time and change in ALS Functional Rating Scale-Revised (ALSFRS-R) score.

The trial’s secondary outcomes are evaluating the incidence of all AEs and serious AEs, ALSFRS-R, ALS quality of life, slow vital capacity (SVC), handheld dynamometer, neurofilament light in the cerebrospinal fluid, neurofilament light in the serum, ventilation assistance-free survival, and overall survival.

CK0803 is developed using Cellenkos' proprietary CRANE® technology, to generate disease-specific products. Cell products do not need to be HLA or ABO-matched to the recipient and multiple doses of CK0803 can be manufactured from a single umbilical cord blood unit and cryopreserved until ready for use.

"We are excited to participate in this important study of this novel allogeneic Treg cell product in patients with ALS," principal investigator Neil Shneider, MD, PhD, associate professor, neurology and director, Eleanor and Lou Gehrig ALS Center, Columbia, said in an earlier statement.2 "Promising data from six ALS patients previously treated with allogeneic cord blood Treg cells through an expanded access program at our Center supports the clinical development of CK0803, and we are grateful to Cellenkos for their commitment to applying their technology to ALS".

Participants must be 18 years or older and capable of providing informed consent or have a legally authorized representative; they must diagnosed with ALS according to the Revised El Escorial Criteria, with disease onset within the past 5 years; their upright SVC, adjusted for sex, age, and height, should be equal to or greater than 50% predicted; baseline ALSFRSR score should fall between 36-45; those using Riluzole, Edaravone, or Albrioza must have been on a stable dose for at least 30 days prior to the study; coagulation parameters such as platelet count, INR, PT, and APTT must be within normal ranges upon screening; and participants must agree to practice highly effective contraception throughout the study and for 90 days following the conclusion of treatment.

Participants that have uncontrolled infections persisting despite antimicrobial therapy, recent or anticipated use of antiplatelet or anticoagulant medications, clinically significant abnormalities in platelet count or coagulation tests, refusal to adhere to study procedures, concurrent participation in other clinical trials, recent treatment with investigational drugs or devices, cancer treatment within the last 5 years (excluding specific cases), pregnancy or breastfeeding in female participants, and any other conditions deemed unsuitable by the investigator or sponsor will be excluded.

1. Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial. News release. February 19, 2024.
2. Cellenkos Announces First Patient Dosed with CK0803 Cell Therapy for Treatment of Amyotrophic Lateral Sclerosis. News release. Cellenkos. May 2, 2023.
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