Amyotrophic Lateral SclerosisPhase 1REGALSNCT05695521
Regulatory T Cells for Amyotrophic Lateral Sclerosis
A Phase 1 study testing CK0803 and Excipient in people with Amyotrophic Lateral Sclerosis, run by Cellenkos, Inc. at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 95 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- CK0803 and Excipient
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 3 sitesUnited States
- Study dates
- 3 Apr 2023to Dec 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 95 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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
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- 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 locations and contacts
3 sites in 1 country.
United States3 sites
Baylor College of Medicine
Houston, Texas, 77030
Michael E. DeBakey Veterans Affairs Medical Center
Houston, Texas, 77030
Columbia University Irving Medical Center
New York, New York, 10033
About this study
Official title: Phase 1 Safety Run-in Study and Phase 1b Randomized, Double Blinded, Placebo Controlled Trial
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- CK0803, Excipient
- Keywords
- ALS, Cellenkos, T Regulatory Cells, Randomized Placebo Control Trial, Umbilical cord blood, Amyotrophic Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CK0803Biological
CK0803 (cryopreserved, allogeneic, cord blood derived T regulatory cells that express neurotropic homing markers) will be administered intravenously
ExcipientOther
Excipient
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Biological: CK0803 |
| Placebo | Placebo comparator | Excipient Receives: Other: Excipient |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 24 weeks
Secondary outcomes (9)
Incidence of all cause AEs and SAEs
Treatment limiting toxicities of CK0803 as assessed by the incidence and severity of AE and SAEs using NCI-CTCAE Version 5.0 criteria.
Measured: 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
Longitudinal processes of ALSFRS-R score measured at baseline and different time points. ALSFRS-R score 0=worst; 48=best
Measured: 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 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.
Measured: 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)
SVC is the volume of air expired, on a low complete expiration after a maximal inspiration without forced or rapid effort.
Measured: 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)
HHD allows for objective measurement of muscle strength
Measured: 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
Nfl CSF
Neurofilament light chain level in the CSF
Measured: 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
Nfl Serum
Neurofilament light chain level in the Serum
Measured: 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
Ventilation assistance-free survival (VAFS)
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)
Measured: 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
Overall Survival (OS)
OS defined as the length of time from the start of treatment that patients are still alive.
Measured: 24 weeks and 48 weeks from first infusion and/or at EOT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2023
- Study start3 Apr 2023
- Primary completionMay 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (47)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellenkos, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Neil Shneider, MD, PhD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05695521
- Sponsor's protocol ID
- CK0803-101-1
- Acronym
- REGALS
- Status last verified
- Oct 2026
- First submitted
- 3 Jan 2023
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Beers DR, Zhao W, Liao B, Kano O, Wang J, Huang A, Appel SH, Henkel JS. Neuroinflammation modulates distinct regional and temporal clinical responses in ALS mice. Brain Behav Immun. 2011 Jul;25(5):1025-35. doi: 10.1016/j.bbi.2010.12.008. Epub 2010 Dec 19.
PubMed 21176785Beers DR, Zhao W, Wang J, Zhang X, Wen S, Neal D, Thonhoff JR, Alsuliman AS, Shpall EJ, Rezvani K, Appel SH. ALS patients' regulatory T lymphocytes are dysfunctional, and correlate with disease progression rate and severity. JCI Insight. 2017 Mar 9;2(5):e89530. doi: 10.1172/jci.insight.89530.
PubMed 28289705Beghi E, Logroscino G, Chio A, Hardiman O, Millul A, Mitchell D, Swingler R, Traynor BJ. Amyotrophic lateral sclerosis, physical exercise, trauma and sports: results of a population-based pilot case-control study. Amyotroph Lateral Scler. 2010 May 3;11(3):289-92. doi: 10.3109/17482960903384283.
PubMed 20433412Berry JD, Cudkowicz ME, Windebank AJ, Staff NP, Owegi M, Nicholson K, McKenna-Yasek D, Levy YS, Abramov N, Kaspi H, Mehra M, Aricha R, Gothelf Y, Brown RH. NurOwn, phase 2, randomized, clinical trial in patients with ALS: Safety, clinical, and biomarker results. Neurology. 2019 Dec 10;93(24):e2294-e2305. doi: 10.1212/WNL.0000000000008620. Epub 2019 Nov 18.
PubMed 31740545Berry JD, Miller R, Moore DH, Cudkowicz ME, van den Berg LH, Kerr DA, Dong Y, Ingersoll EW, Archibald D. The Combined Assessment of Function and Survival (CAFS): a new endpoint for ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener. 2013 Apr;14(3):162-8. doi: 10.3109/21678421.2012.762930. Epub 2013 Jan 17.
PubMed 23323713Brooks BR, Miller RG, Swash M, Munsat TL; World Federation of Neurology Research Group on Motor Neuron Diseases. El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000 Dec;1(5):293-9. doi: 10.1080/146608200300079536. No abstract available.
PubMed 11464847Brunstein CG, Miller JS, Cao Q, McKenna DH, Hippen KL, Curtsinger J, Defor T, Levine BL, June CH, Rubinstein P, McGlave PB, Blazar BR, Wagner JE. Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood. 2011 Jan 20;117(3):1061-70. doi: 10.1182/blood-2010-07-293795. Epub 2010 Oct 15.
PubMed 20952687Brunstein CG, Miller JS, McKenna DH, Hippen KL, DeFor TE, Sumstad D, Curtsinger J, Verneris MR, MacMillan ML, Levine BL, Riley JL, June CH, Le C, Weisdorf DJ, McGlave PB, Blazar BR, Wagner JE. Umbilical cord blood-derived T regulatory cells to prevent GVHD: kinetics, toxicity profile, and clinical effect. Blood. 2016 Feb 25;127(8):1044-51. doi: 10.1182/blood-2015-06-653667. Epub 2015 Nov 12.
PubMed 26563133Cervantes-Barragan L, Firner S, Bechmann I, Waisman A, Lahl K, Sparwasser T, Thiel V, Ludewig B. Regulatory T cells selectively preserve immune privilege of self-antigens during viral central nervous system infection. J Immunol. 2012 Apr 15;188(8):3678-85. doi: 10.4049/jimmunol.1102422. Epub 2012 Mar 9.
PubMed 22407917Choi EY, Orlova VV, Fagerholm SC, Nurmi SM, Zhang L, Ballantyne CM, Gahmberg CG, Chavakis T. Regulation of LFA-1-dependent inflammatory cell recruitment by Cbl-b and 14-3-3 proteins. Blood. 2008 Apr 1;111(7):3607-14. doi: 10.1182/blood-2007-07-103077. Epub 2008 Jan 31.
PubMed 18239087Cudkowicz ME, Lindborg SR, Goyal NA, Miller RG, Burford MJ, Berry JD, Nicholson KA, Mozaffar T, Katz JS, Jenkins LJ, Baloh RH, Lewis RA, Staff NP, Owegi MA, Berry DA, Gothelf Y, Levy YS, Aricha R, Kern RZ, Windebank AJ, Brown RH Jr. A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis. Muscle Nerve. 2022 Mar;65(3):291-302. doi: 10.1002/mus.27472. Epub 2022 Jan 5.
PubMed 34890069Das K, Nag C, Ghosh M. Familial, environmental, and occupational risk factors in development of amyotrophic lateral sclerosis. N Am J Med Sci. 2012 Aug;4(8):350-5. doi: 10.4103/1947-2714.99517.
PubMed 22912943Finkelstein DM, Schoenfeld DA. Combining mortality and longitudinal measures in clinical trials. Stat Med. 1999 Jun 15;18(11):1341-54. doi: 10.1002/(sici)1097-0258(19990615)18:113.0.co;2-7.
PubMed 10399200Garbuzova-Davis S, Haller E, Saporta S, Kolomey I, Nicosia SV, Sanberg PR. Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS. Brain Res. 2007 Jul 9;1157:126-37. doi: 10.1016/j.brainres.2007.04.044. Epub 2007 Apr 24.
PubMed 17512910Garbuzova-Davis S, Hernandez-Ontiveros DG, Rodrigues MC, Haller E, Frisina-Deyo A, Mirtyl S, Sallot S, Saporta S, Borlongan CV, Sanberg PR. Impaired blood-brain/spinal cord barrier in ALS patients. Brain Res. 2012 Aug 21;1469:114-28. doi: 10.1016/j.brainres.2012.05.056. Epub 2012 Jun 27.
PubMed 22750125