Guillain-Barré SyndromePhase 1NCT03773328
A Clinical Trial of CK0801 (a New Drug) In Patients With Treatment-Resistant Guillain-Barré Syndrome (GBS)
A Phase 1 study testing CK0801 in people with Guillain-Barré Syndrome, run by Cellenkos, Inc..
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Guillain-Barré Syndrome
- What is being tested?
- CK0801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Nov 2022to Feb 2027
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject fulfills the diagnostic criteria for Guillain-Barré syndrome (GBS) (Appendix 1).
- HLA matched (≥ 3/6 at HLA-A, HLA-B, and HLA-DRB1) cord blood unit available for CK0801 generation.
- Subjects age 18 to 70 years.
- Subject has GBS disability scale score of 4 and unchanged 1 week after IVIG or PE treatment (Appendix 2).
- Subject has completed IVIG/PE treatment ≥ 4 weeks prior to CK0801 infusion.
- 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).
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- Bilirubin ≤ 2 x ULN and, ALT ≤ 2 x ULN (unless Gilbert's syndrome).
- Calculated creatinine clearance of > 50mL/min using the Cockroft-Gault equation for adult patients 18 - 70 years old.
- 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).
- Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
- 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.
Who cannot join
- Subject has received immunotherapy, chemotherapy, biologic or investigational agent within 4 weeks prior to CK0801 infusion.
- Subject has received prior CB Treg therapy.
- Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
- Subject has received a vaccination with a Live virus (e.g., Measles, Mumps, Rubella, Varicella).
- Subject is pregnant or breastfeeding.
- HIV seropositivity
- Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.
About this study
Official title: Phase I Trial To Evaluate The Safety Of CK0801 In Treatment-Resistant Guillain-Barré Syndrome (GBS)
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.
Read the 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.
- Conditions
- Guillain-Barré Syndrome
- Being tested
- CK0801
- Medical subject headings
- Guillain-Barre Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CK0801Biological
CK0801 (Cord blood-derived T-regulatory cells)
| Study group | Role | What they receive |
|---|---|---|
| CK0801, 50ml | Experimental | 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). Receives: Biological: CK0801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0
Number of Participants with Severe (Grade 3 or 4) Toxicity
Measured: 24 hours post-intervention
Number of Participants with Regimen Related Death
- severe (grade 3 or 4) cytokine release syndrome (CRS) within 30 days
Measured: 30 days post-intervention
Number of Participants with Severe Cytokine Release Syndrome (CRS)
Number of Participants with Severe (Grade 3 or 4) CRS
Measured: 30 days post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2018
- Study startNov 2022 (estimated)
- Primary completionFeb 2025 (estimated)
- Study completionFeb 2027 (estimated)
Why it stopped: Pending contract negotiations
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Guillain-Barré SyndromeStudies 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
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cellenkos, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tara Sadeghi, Study director, Sponsor GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03773328
- Sponsor's protocol ID
- CK0801-201-1
- Status last verified
- Jan 2023
- First submitted
- 23 Oct 2018
- Last updated
- 4 Jan 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial To Evaluate The Safety Of CK0801 In Treatment-Resistant Guillain-Barré Syndrome (GBS)
- ClinicalTrials.gov ID
- NCT03773328
- Acronym / sponsor ID
- CK0801-201-1
- Conditions
- Guillain-Barré Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Pending contract negotiations
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Cellenkos, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2022 (estimated)
- Primary completion
- Feb 2025 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 12 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| CK0801, 50ml | Experimental | Biological: CK0801All 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). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Subject fulfills the diagnostic criteria for Guillain-Barré syndrome (GBS) (Appendix 1).
- HLA matched (≥ 3/6 at HLA-A, HLA-B, and HLA-DRB1) cord blood unit available for CK0801 generation.
- Subjects age 18 to 70 years.
- Subject has GBS disability scale score of 4 and unchanged 1 week after IVIG or PE treatment (Appendix 2).
- Subject has completed IVIG/PE treatment ≥ 4 weeks prior to CK0801 infusion.
Key exclusion criteria (7 listed)
- Subject has received immunotherapy, chemotherapy, biologic or investigational agent within 4 weeks prior to CK0801 infusion.
- Subject has received prior CB Treg therapy.
- Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
- Subject has received a vaccination with a Live virus (e.g., Measles, Mumps, Rubella, Varicella).
- Subject is pregnant or breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CK0801CK0801 (Cord blood-derived T-regulatory cells) | Biological | CK0801, 50ml |
Primary outcome
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
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
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
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tara Sadeghi study director
Sponsor GmbH
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CK0801 in people with Guillain-Barré Syndrome, run by Cellenkos, Inc..
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 11 inclusion and 7 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: withdrawn, as of the sponsor's last update on 4 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 (time frame: 24 hours post-intervention). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 4 Jan 2023.