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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.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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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
Are there results?
Not yet: the study is under way

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.
Show all 9Show fewer
  • 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
Show all 10Show fewer
  • 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

Other trials in United States

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.

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 groups in NCT05695521
Study groupRoleWhat they receive
CK0803ExperimentalCK0803 (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
PlaceboPlacebo comparatorExcipient
Receives: Other: Excipient

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

  2. 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)

  1. 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)

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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.

  1. First posted25 Jan 2023
  2. Study start3 Apr 2023
  3. Primary completionMay 2027 (estimated)
  4. Study completionDec 2027 (estimated)

Resources

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)
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 21176785

  • Beers 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 28289705

  • Beghi 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 20433412

  • Berry 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 31740545

  • Berry 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 23323713

  • Brooks 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 11464847

  • Brunstein 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 20952687

  • Brunstein 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 26563133

  • Cervantes-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 22407917

  • Choi 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 18239087

  • Cudkowicz 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 34890069

  • Das 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 22912943

  • Finkelstein 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 10399200

  • Garbuzova-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 17512910

  • Garbuzova-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

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CK0803 and Excipient in people with Amyotrophic Lateral Sclerosis, run by Cellenkos, Inc. at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment Limiting Toxicity (TLT) (time frame: 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05695521, which also lists 47 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.

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