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A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)

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ClinicalTrials.gov Identifier: NCT03482050
Recruitment Status : Recruiting
First Posted : March 29, 2018
Last Update Posted : August 8, 2018
Sponsor:
Information provided by (Responsible Party):
Kadimastem

Tracking Information
First Submitted Date  ICMJE March 14, 2018
First Posted Date  ICMJE March 29, 2018
Last Update Posted Date August 8, 2018
Actual Study Start Date  ICMJE April 12, 2018
Estimated Primary Completion Date August 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 22, 2018)
Number of participants with treatment emergent adverse events as assessed by CTCAE Version 4.03 [ Time Frame: 11 Months ]
Safety and tolerability assessment will be based on treatment emerged adverse events
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03482050 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 22, 2018)
  • Change in the ALS functional rating scale [ Time Frame: 11 Months ]
    Preliminary efficacy assessment to measure response to treatment or progression of disease. Scale includes 12 questions related to tasks. Each task rated on a five-point scale from 0 = unable to do the task, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
  • Change in predicted slow vital capacity (%SVC) [ Time Frame: 11 Months ]
    Preliminary efficacy assessment to measure respiratory muscle strength
  • Change in muscle strength grading by JAMAR grip strength [ Time Frame: 11 Months ]
    Preliminary efficacy assessment to measure hand grip strength
  • Change in muscle strength grading by hand held dynamometer (HHD) [ Time Frame: 11 Months ]
    Preliminary efficacy assessment to measure muscle strength in limb muscles
  • Change in Quality of Life questionnaire (ALSAQ-40) [ Time Frame: 11 Months ]
    Preliminary efficacy assessment to measure the subjective well-being of patients
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)
Official Title  ICMJE A Phase I/IIa, Open Label, Dose-escalating Clinical Study to Evaluate the Safety, Tolerability and Theraputic Effects of Transplantation of Astrocytes Derived From Human Embryonic Stem Cells (hESC), in Patients With Amyotrophic Lateral Sclerosis (ALS)
Brief Summary

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS).

There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment.

The study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Sequential Assignment
Intervention Model Description:
Dose-escalating, four subject-groups clinical study; a single treatment administration of AstroRx, administered in an escalating low, medium and high dose or two consecutive administrations of the medium dose separated by an interval. AstroRx will be administered by intrathecal (spinal) injection to subjects with ALS at the early disease stage.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE ALS (Amyotrophic Lateral Sclerosis)
Intervention  ICMJE Biological: AstroRx
Astrocytes derived from human embryonic stem cells
Study Arms  ICMJE Experimental: AstroRx
Intervention: Biological: AstroRx
Publications * Not Provided

*   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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 22, 2018)
21
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2020
Estimated Primary Completion Date August 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  1. El Escorial criteria for probable or definite ALS
  2. Males and and non pregnant females between 18 and 70 years of age
  3. Patients with an ALS-FRS-R score of at least 30 with an ALS diagnosis of two years or less
  4. No history of active psychiatric disorder.
  5. Patient has a good understanding of the study and nature of the procedure
  6. Patient provides written informed consent prior to any study procedure
  7. Patients should either be on a stable dose of Riluzole and/or Radicava® (if applicable) for 3 months, or not be treated with Riluzole or Radicava®
  8. Patient is medically able to tolerate immunosuppression regimen
  9. Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines

Main Exclusion Criteria:

  1. Patient has a past infection or a positive test for HBV,HCV or HIV
  2. Patient is in need of respiratory support
  3. Patient has a lower than 10/12 in ALS-FRS-R respiratory parameters or below 70% of predicted slow vital capacity (SVC)
  4. Patient has renal failure
  5. Patient has impaired hepatic function
  6. Patient has a Body Mass Index (BMI) of <18.5 or > 30
  7. Patient suffers from significant cardiac disease, diabetes, autoimmune diseases, chronic severe infection, malignant disease or any other disease or condition that may risk the patient or interfere with the ability to interpret the study results
  8. Patient has systemic inflammation or active infections
  9. Patient has been treated previously with any stem cell therapy
  10. Current use of immunosuppressant medication or use of such medication within 6 weeks of Screening visit (Visit 0)
  11. Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study
  12. Any known immunodeficiency syndrome
  13. Any concomitant disease or condition limiting patient safety to participate
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Yael Feinsod +972-2-6779372 HCRC_ALS@hadassah.org.il
Listed Location Countries  ICMJE Israel
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03482050
Other Study ID Numbers  ICMJE ASTRO-001-IL
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Kadimastem
Study Sponsor  ICMJE Kadimastem
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Kadimastem
Verification Date April 2018

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