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A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS)

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. Identifier: NCT03773328
Recruitment Status : Not yet recruiting
First Posted : December 12, 2018
Last Update Posted : August 30, 2019
Information provided by (Responsible Party):
Cellenkos, Inc.

Brief Summary:
The goal of this research study is to determine whether it is safe and practical to give CK0801 (a cord-blood derived T-regulatory cell product) to patients with Guillain-Barré Syndrome (GBS). Researchers also want to determine the highest possible dose that is safe to be given and to learn if CK0801 may improve the symptoms of GBS. There will be three doses of CK0801 given during this study. A minimum of three patients will be treated in each dose level. The dose a patient receives is dependent on the timing of when they enter the study, as after each dose level is completed the following patients will receive the next highest dose level.

Condition or disease Intervention/treatment Phase
Guillain-Barré Syndrome Biological: CK0801 Phase 1

Detailed Description:
CK0801 (Cord blood-derived T-regulatory cells) consists of ex vivo expanded T-regulatory cells with a flow cytometry phenotype of ≥ 60% CD4+CD25+ T-regulatory (Treg) cells and < 10% CD4-CD8+ T-cytotoxic/suppressor cells. The cellular starting material of CK0801 is a single unit of umbilical cord blood (CBU) from a normal, healthy unrelated donor. Because Treg cells are present only at low frequency in circulating blood or umbilical cord blood, production of clinically relevant Treg cell doses requires ex vivo enrichment and expansion of Treg cells with a CD4+CD25+ phenotype. In their natural state, T-regulatory (Treg) cells play an important role in maintaining immune homeostasis and limiting autoimmune responses by modulating both innate and adaptive immunity. Based on literature reports of animal studies showing induction of immune tolerance by Treg cells in autoimmune diseases, graft-versus-host disease, and solid organ transplant rejection, the administration of Treg cells in human clinical trials has become an attractive strategy to induce immune tolerance in patients in a variety of clinical settings.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Open-label, single arm study using a Simon 3 + 3 design. Study will utilize 3 dose cohorts with a minimum of 3 patients in each cohort.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I Trial To Evaluate The Safety Of CK0801 In Treatment-Resistant Guillain-Barré Syndrome (GBS)
Estimated Study Start Date : February 2020
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: CK0801, 50ml

All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one 50mL intravenous dose of CK0801 (Treg cells) on study Day 0. A total of three cohorts will be evaluated.

Cohort dosing will be as follows:

Dose level 1 = 1x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 2 = 3x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 3 = 1x10e7/kg Treg cells per kg recipient ideal body weight (IBW).

Biological: CK0801
CK0801 (Cord blood-derived T-regulatory cells)

Primary Outcome Measures :
  1. Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 [ Time Frame: 24 hours post-intervention ]
    Number of Participants with Severe (Grade 3 or 4) Toxicity

  2. Number of Participants with Regimen Related Death [ Time Frame: 30 days post-intervention ]
    • severe (grade 3 or 4) cytokine release syndrome (CRS) within 30 days

  3. Number of Participants with Severe Cytokine Release Syndrome (CRS) [ Time Frame: 30 days post-intervention ]
    Number of Participants with Severe (Grade 3 or 4) CRS

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Subject fulfills the diagnostic criteria for Guillain-Barré syndrome (GBS) (Appendix 1).
  2. HLA matched (≥ 3/6 at HLA-A, HLA-B, and HLA-DRB1) cord blood unit available for CK0801 generation.
  3. Subjects age 18 to 70 years.
  4. Subject has GBS disability scale score of 4 and unchanged 1 week after IVIG or PE treatment (Appendix 2).
  5. Subject has completed IVIG/PE treatment ≥ 4 weeks prior to CK0801 infusion.
  6. Subject has modified Erasmus GBS outcome score (mEGOS score) of ≥7 at the time of presentation and unchanged 1 week after IVIG or PE treatment (Table 3).
  7. Bilirubin ≤ 2 x ULN and, ALT ≤ 2 x ULN (unless Gilbert's syndrome).
  8. Calculated creatinine clearance of > 50mL/min using the Cockroft-Gault equation for adult patients 18 - 70 years old.
  9. Female subjects of child bearing potential (FPCP) must have a negative urine or serum pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized. FPCP must agree to use maximally effective birth control or to abstain from heterosexual activity throughout the study. Effective contraceptive methods include intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide).
  10. Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
  11. Subject is willing and able to provide written informed consent. If subject is temporarily unable to sign the consent due to disease-related complications (e.g., upper extremity paralysis), a legally authorized representative (LAR) will be used. The subject will sign the consent as soon as they are capable.

Exclusion Criteria:

  1. Subject has received immunotherapy, chemotherapy, biologic or investigational agent within 4 weeks prior to CK0801 infusion.
  2. Subject has received prior CB Treg therapy.
  3. Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
  4. Subject has received a vaccination with a Live virus (e.g., Measles, Mumps, Rubella, Varicella).
  5. Subject is pregnant or breastfeeding.
  6. HIV seropositivity
  7. Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.

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 identifier (NCT number): NCT03773328

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Contact: Erin Horne 800-217-8247
Contact: Tara Sadeghi 800-217-8247

Sponsors and Collaborators
Cellenkos, Inc.
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Study Director: Tara Sadeghi Sponsor GmbH
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Responsible Party: Cellenkos, Inc. Identifier: NCT03773328    
Other Study ID Numbers: CK0801-201-1
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: August 30, 2019
Last Verified: August 2019
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
Additional relevant MeSH terms:
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Guillain-Barre Syndrome
Pathologic Processes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases