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Regulatory T Cells for Amyotrophic Lateral Sclerosis (REGALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05695521
Recruitment Status : Not yet recruiting
First Posted : January 25, 2023
Last Update Posted : February 3, 2023
Sponsor:
Information provided by (Responsible Party):
Cellenkos, Inc.

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: CK0803 Other: Excipient Phase 1

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 66 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 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.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double Blinded, Placebo Control
Primary Purpose: Treatment
Official Title: Phase 1 Safety Run-in Study and Phase 1b Randomized, Double Blinded, Placebo Controlled Trial
Estimated Study Start Date : January 30, 2023
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2027


Arm Intervention/treatment
Experimental: CK0803

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

Placebo Comparator: Placebo
Excipient
Other: Excipient
Excipient




Primary Outcome Measures :
  1. Treatment Limiting Toxicity (TLT) [ Time Frame: 28 days ]
    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

  2. Combined assessment of function and survival (CAFS) [ Time Frame: 24 weeks ]
    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.


Secondary Outcome Measures :
  1. Incidence of all cause AEs and SAEs [ Time Frame: 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) ]
    Treatment limiting toxicities of CK0803 as assessed by the incidence and severity of AE and SAEs using NCI-CTCAE Version 5.0 criteria.

  2. ALS Functional Rating Scale-Revised (ALSFRS-R) Score [ Time Frame: 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 ]
    Longitudinal processes of ALSFRS-R score measured at baseline and different time points. ALSFRS-R score 0=worst; 48=best

  3. Amyotrophic Lateral Sclerosis Specific Quality of Life - Revised (ALSSQOL-R) [ Time Frame: 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 ]
    Each item of the ALSSQOL-R is rated by the individual using a 0 to 10 point Likert scale, with 0 being the least desirable situation, and 10 being the most desirable.

  4. Slow Vital Capacity (SVC) [ Time Frame: 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 ]
    SVC is the volume of air expired, on a low complete expiration after a maximal inspiration without forced or rapid effort.

  5. Handheld dynamometer (HHD) [ Time Frame: 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 ]
    HHD allows for objective measurement of muscle strength

  6. Nfl CSF [ Time Frame: 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 ]
    Neurofilament light chain level in the CSF

  7. Nfl Serum [ Time Frame: 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 EOT12 months ]
    Neurofilament light chain level in the Serum

  8. Ventilation assistance-free survival (VAFS) [ Time Frame: 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 ]
    VAFS is defined as the time to the earliest occurrence of 1 of the following events: i) Death, or ii) Permanent ventilation (> 22 hours of mechanical ventilation [invasive or non-invasive] per day for > 21 consecutive days in the absence of an acute potentially reversible event)

  9. Overall Survival (OS) [ Time Frame: 24 weeks and 48 weeks from first infusion and/or at EOT ]
    OS defined as the length of time from the start of treatment that patients are still alive.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 95 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05695521


Contacts
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Contact: Tara Sadeghi 7138064787 tara.sadeghi@cellenkosinc.com
Contact: Stacy Minor 9792821294 stacy.minor@cellenkosinc.com

Locations
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United States, New York
Columbia University Irving Medical Center
New York, New York, United States, 10033
Contact: Neil Shneider, MD, PhD         
Principal Investigator: Neil Shneider, MD, PhD         
Sponsors and Collaborators
Cellenkos, Inc.
Investigators
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Principal Investigator: Neil Shneider, MD, PhD Columbia University
Publications:
Gelman A, C. J., Stern HS, Rubin DB. (1995). Bayesian Data Analysis. New York, Chapman & Hall.
Gladstone DE, H. C., Lyu MA, Mock J, Adams D, Gibbs K, Li L, Huang M, Zeng K, D'Alessio F, Herlihy J, Trevino S, Hari P, Sadeghi T, Parmar S, Slutsky A and Mukherjee S (23 Nov 2021).
Kadia TM, P. N., Yilmaz M, Li L, Lyu M, Huang M, Zeng K, Parmar S, DiNardo CD, Daver N, Issa GC, Jabbour E, Borthakur G, Verstovsek S (2020).
KaplanELandMeierP (1958).
Zeng K, M. H., Popat U, Nieto Y, Ciurea SO, Olson AL, Lyu M, Huang M, Nishimoto M, Qazilbash MH, Ramos JD, Shpall EJ, Champlin RE, Parmar S, Andersson BS. (2019).

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Responsible Party: Cellenkos, Inc.
ClinicalTrials.gov Identifier: NCT05695521    
Other Study ID Numbers: CK0803-101-1
First Posted: January 25, 2023    Key Record Dates
Last Update Posted: February 3, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cellenkos, Inc.:
ALS
Cellenkos
T Regulatory Cells
Randomized Placebo Control Trial
Umbilical cord blood
Amyotrophic Lateral Sclerosis
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases