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Umbilical Cord Blood Cell Transplant Into Injured Spinal Cord With Lithium Carbonate or Placebo Followed by Locomotor Training

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ClinicalTrials.gov Identifier: NCT03979742
Recruitment Status : Not yet recruiting
First Posted : June 7, 2019
Last Update Posted : June 11, 2019
Sponsor:
Collaborator:
Amarex Clinical Research
Information provided by (Responsible Party):
StemCyte, Inc.

Tracking Information
First Submitted Date  ICMJE June 3, 2019
First Posted Date  ICMJE June 7, 2019
Last Update Posted Date June 11, 2019
Estimated Study Start Date  ICMJE July 1, 2019
Estimated Primary Completion Date September 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 6, 2019)
Walking Index of Spinal Cord Injury (WISCI II) Overall Measure [ Time Frame: At Week 48. ]
The primary endpoint for this study is mean change from baseline of the Walking Index of Spinal Cord Injury (WISCI II).
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03979742 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 6, 2019)
  • Walking Index of Spinal Cord Injury Measure (WISCI II) at Week 6 and 28 [ Time Frame: At Week 6 and 28. ]
    Mean change from baseline of the Walking Index of Spinal Cord Injury (WISCI II).
  • Spinal Cord Independence Measure (SCIM III) [ Time Frame: At Week 6, 28, and 48. ]
    Mean change from baseline of Spinal Cord Independence Measure (SCIM III)
  • Measure of American Spinal Injury Association (ASIA) Motor and Sensory Scores and AIS Grade [ Time Frame: At Week 2, 6, 28, and 48. ]
    American Spinal Injury Association (ASIA) score has three components: (1) Sensory scores: There is a maximum total of 56 points each for light touch and pin prick (sharp/dull discrimination) modalities, for a total of 112 points per side of the body. (2) Motor scores: There is a maximum score of 25 for each extremity, totaling 50 for the upper limbs and 50 for the lower limbs. (3) ASIA Impairment Scale: Injuries are classified in general terms of being neurologically "complete" or "incomplete" based upon the sacral sparing definition.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: June 6, 2019)
  • Exploratory Endpoint - Kunming Locomotor Score (KLS) Measure [ Time Frame: At Week 6, 28, and 48. ]
    Mean change from baseline of Kunming locomotor scores (KLS). Kunming Locomotor Scale (KLS) is a 10-grade Roman numeral locomotion scoring system describing ability to stand, ability to walk, and required support/devices.
  • Exploratory Endpoint - Numerical Rating Scale (NRS) Measure [ Time Frame: At Week 2, 6, 28, and 48. ]
    Mean change from baseline of Numerical Rating Scale (NRS) for neuropathic pain. An 11-unit scale will be used, where 0 represents "No pain" and 10 represents the "Worst possible pain".
  • Exploratory Endpoint - LANSS Scale Measure [ Time Frame: At Week 2, 6, 28, and 48. ]
    Mean change from baseline of Leeds Assessment of Neuropathic Symptoms and Signs (LANSS) scale. An 11-unit scale will be used, where 0 represents "No pain" and 10 represents the "Worst possible pain". The Leeds Assessment of Neuropathic Symptoms and Signs (LANSS) pain scale is an assessment tool used to analyze and classify pain. It is a simple bedside test, conducted in two parts .i.e. a patient-completed questionnaire and a brief clinical assessment. Out of the seven items in the LANSS Pain Scale, five are symptom related and two are examination items.
  • Exploratory Endpoint - SSEP and MEP Measure [ Time Frame: At week 48. ]
    Percentage of subjects with positive change in Somatosensory evoked potential (SSEP) and Motor evoked potentials (MEP).
  • Exploratory Endpoint - Long fiber bundles growth measure [ Time Frame: At Week 6, 28, and 48. ]
    Percentage of subjects with Long fiber bundles growth crossing the injury site by Magnetic resonance diffusion tenor images (MR/DTI).
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Blood Cell Transplant Into Injured Spinal Cord With Lithium Carbonate or Placebo Followed by Locomotor Training
Official Title  ICMJE A Randomized Controlled Phase II, Three-Arm Study of Umbilical Cord Blood Cell Transplant (MC001) Into Injured Spinal Cord in Combination With Lithium Carbonate or Placebo Followed by the Locomotor Training in the Treatment of Chronic Complete Spinal Cord Injuries (SCI)
Brief Summary Umbilical cord blood mononuclear stem cells (UCBMSCs) transplant in combination with 6-week course of oral lithium carbonate (Li2CO3) followed by the intensive locomotor training for up to 6 hours a day, 6 days a week, and for 3-6 months for treatment in patients with chronic, stable and complete spinal cord injury.
Detailed Description

This study is a randomized controlled, Phase II, three-arm study of Umbilical Cord Blood Mononuclear Cell (MC001) transplant into the injured spinal cord in combination with either 6-week course of oral lithium carbonate or placebo followed by the locomotor training for up to 6 hours a day, 6 days a week, for 3-6 months.

A total of 27 subjects with chronic complete spinal cord injury (SCI) will be randomized to one of the three treatment groups. The subjects assigned to Group A and B will receive 6.4 million UCBMNC (MC001) transplanted into the dorsal root entry zones above and below the injury site exposed by a laminectomy. Subjects in Group A and B will be treated with oral lithium carbonate or placebo for six weeks in a double blind manner. All subjects will receive 3-6 months of intensive locomotor training.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Masking Description:
This is a partial double-blind, placebo-controlled study. Subjects in Group A and B will be treated with oral lithium carbonate or placebo for six weeks in a double blind manner. Subjects assigned to Group C are not blinded.
Primary Purpose: Treatment
Condition  ICMJE Spinal Cord Injuries
Intervention  ICMJE
  • Biological: Umbilical Cord Blood Mononuclear Cell
    Active ingredients: Monocytes, CD34+, CD133+ cells Dose: 4 injections of 16-μliter (100,000 cells/μliter)
    Other Name: MC001
  • Drug: Lithium Carbonate
    Active Ingredients: Lithium Dose: 900-1200 mg/day in 300 mg tid or 600 mg bid
  • Other: Placebo
    Placebo
  • Other: Locomotor Training
    Locomotor training for up to 6 hours a day, 6 days a week, and for 3-6 months
Study Arms  ICMJE
  • Experimental: MC001 + lithium
    UCBMNC (MC001) transplant + oral lithium carbonate x 6 weeks
    Interventions:
    • Biological: Umbilical Cord Blood Mononuclear Cell
    • Drug: Lithium Carbonate
    • Other: Locomotor Training
  • Experimental: MC001 + placebo
    UCBMNC (MC001) transplant + oral placebo x 6 weeks
    Interventions:
    • Biological: Umbilical Cord Blood Mononuclear Cell
    • Other: Placebo
    • Other: Locomotor Training
  • Placebo Comparator: No treatment
    No surgery, no transplant, no lithium
    Intervention: Other: Locomotor Training
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: June 6, 2019)
27
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 1, 2020
Estimated Primary Completion Date September 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male and female subjects' ≥18 to ≤60 years.
  • Traumatic SCI at a neurological level (the lowest contiguous spinal cord segmental level that has intact motor and sensory score) between C5 and T11.

Note: For the first three subjects at each study center, the neurological level of SCI will be limited to thoracic region (between T1 and T11).

  • Subjects with chronic SCI (defined as ≥ 12 months post- initial SCI surgery) with stable neurologic findings for at least six months and be able stand at least 1 hour/day using a standing frame, tilt table, or equivalent device.
  • Subjects with a current neurological status of ASIA impairment grade A (complete).
  • The injured site of the spinal cord is within three vertebral levels as confirmed by MRI scan.
  • Subject must be in good enough physical health to tolerate the surgery and participate in the intensive walking program.
  • Clinically normal resting 12-lead ECG at Screening Visit or, if abnormal, considered not clinically significant by the Principal Investigator.
  • Both male and female subjects and their partners of childbearing potential must agree to use medically accepted methods of contraception.
  • Willing and able to participate in all aspects of the study, including completion of subjective evaluations, attendance at scheduled clinic visits, and compliance with all protocol requirements as evidenced by providing written informed consent.

Exclusion Criteria:

  • Clinically significant renal, cardiovascular, hepatic and psychiatric diseases or other conditions that may increase risk of complications during or after surgery or may reduce the ability of the patient to participate in intense locomotor training based on the medical judgment of the investigator.
  • Presence of any clinically significant medical condition(s) or infection (including but not limited to the carrier of hepatitis B virus or HIV) that, in the opinion of the Investigator, could interfere with the treatment or participation in the study.
  • Subjects with flaccid paralysis with absence of deep tendon reflexes in the legs, severe atrophy of the lower limbs, or other evidence of lumbosacral injury, peripheral nerve injury, and motoneuronal loss.
  • Fracture of weight-bearing bones and joints. These include fractures of femur, tibia, and fibula, as well as the ankle, knee, or hip joints. If such fractures have healed, the patient can be included in the trial.
  • Injury to brain, peripheral nerve, or muscle that may interfere with neurologic or walking assessment.
  • Pregnant or lactating woman.
  • Syringomyelic cyst >30% of the cord diameter.
  • Unavailability of HLA-matched umbilical cord blood cells.
  • Current or recent (within 1 month) treatment with lithium.
  • Any contraindication of laminectomy operation, lithium carbonate therapy or locomotor training.
  • Taking medication that may interfere with lithium clearance, such as diuretics, NSAIDs (except acetoaminophen or Tylenol), nitroimidazole antibiotics or medications that has clinically important interactions with lithium.
  • Subjects with abnormal renal function, thyroid or parathyroid levels, cardiovascular disease, depression at screening will be excluded, if considered clinically significant by the Principal Investigator.
  • Subject who is currently participating in another investigational study or has been taking any investigational drug within the last 4 weeks before screening for this study.
  • Any other criteria, which, in the opinion of the investigator, suggests that the subject would not be compliant with the study protocol and/or would not be suitable to participate in this study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kush Dhody, MBBS, MS, CCRA 301-956-2536 kushd@amarexcro.com
Contact: Wise Young, Ph.D., M.D wisey@mac.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03979742
Other Study ID Numbers  ICMJE US105d
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Plan Description: All information supplied by StemCyte, Inc in connection with this study and not previously published, is considered confidential information. This information includes, but is not limited to, the Investigator's Brochure, clinical protocol, case report forms and other scientific data. All data collected during the study are confidential. This confidential information shall remain the sole property of StemCyte, Inc, shall not be disclosed to others without the written consent of StemCyte, Inc, and shall not be used except in the performance of this study.
Responsible Party StemCyte, Inc.
Study Sponsor  ICMJE StemCyte, Inc.
Collaborators  ICMJE Amarex Clinical Research
Investigators  ICMJE Not Provided
PRS Account StemCyte, Inc.
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP