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Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

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ClinicalTrials.gov Identifier: NCT01082653
Recruitment Status : Suspended (Suspended due to lack of funding.)
First Posted : March 8, 2010
Last Update Posted : May 9, 2014
Sponsor:
Information provided by (Responsible Party):
TCA Cellular Therapy

Study Description
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Brief Summary:

A Phase I, single center, prospective, non-randomized, open label, safety/efficacy study of the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis according to established criteria (1), (2) with a moderate to severe diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.

The primary purpose of this study is to evaluate safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.

Secondary outcomes will include a)neurological evidence of trends toward a slowing down of the decline of the forced vital capacity (FVC) (3) and of the functional rating scale (ALS-FRS) scores, as assessed at 3-month intervals, b)evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z (4) scores and c)patient evaluation that the treatment was effective and consider the possibility of a new cell product stem cell infusion.

Subjects who fulfill inclusion/exclusion criteria and sign informed consent will undergo an aspiration of bone marrow from the iliac crest for preparation of the cellular product.

The day of infusion, the investigational product will be injected into the patient's intrathecal space.

After cell infusion patients will be followed at WK 2, MN 1, MN 2, MN 6 and a long-term followup at MN 12 in the clinic and/or office. Electromyographic (EMG) studies, Forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores will have been used to assess the status of the disease before (historical record acceptable if done within three months of Screening Visit) and during the 12-month study period after cell infusion.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: autologous bone marrow-derived stem cells Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Phase I, Single Center, Prospective, Non-randomized, Open Label, Safety/Efficacy Study of the Infusion of Autologous Bone Marrow-derived Stem Cells, in Patients With Amyotrophic Lateral Sclerosis
Study Start Date : March 2010
Estimated Primary Completion Date : May 2014
Estimated Study Completion Date : May 2014

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Safety
infusion of autologous bone marrow-derived stem cells
 Biological: autologous bone marrow-derived stem cells
All patients will receive a unique one-time intrathecal infusion of the cell product suspended in infusion medium.


Outcome Measures
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Primary Outcome Measures :
  1. Safety [ Time Frame: one year ]
    Safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.


Secondary Outcome Measures :
  1. Efficacy [ Time Frame: one year ]

    Neurological evidence of trends toward a slowing down of the decline of the forced vital capacity and of the functional rating scale scores, as assessed at 3 month intervals.

    Evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores.

    Patient evaluation that the treatment was effective.



Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Adult male and female subjects > 18 years of age.
  2. Good understanding of the protocol and willingness to consent.
  3. Moderate to severe Diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
  4. Vital capacity at least 50% predicted value for gender, height and age.
  5. More than 6 and less than 36 months of evolution of the disease.
  6. Hematocrit greater than 30 % prior to bone marrow aspiration.
  7. Platelet count greater than 100 Thousand/uL at screening.
  8. INR less than or equal to 1.5.

Exclusion Criteria:

  1. Any concurrent illness, which affects the bone marrow.
  2. Any concomitant medication that affects the bone marrow.
  3. Previous stem cell therapy.
  4. Any lymphoproliferative disease.
  5. Riluzole with 4 weeks of study entry and at any time during the study.
  6. Hemophiliacs or subjects with bleeding disorders.
  7. Known hypersensitivity to fetal bovine serum
  8. HIV infection.
  9. Serum creatinine > 3.0 in subjects not on hemodialysis.
  10. Skin infection at the infusion site or systemic infection
  11. Current smoker.
  12. Active drug or alcohol addiction
  13. Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential.
  14. Subjects that are breast feeding.
  15. Any condition that the Principal Investigator considers would render the subject unfit for the study.
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT01082653


Locations
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United States, Louisiana
TCA Cellular Therapy
Covington, Louisiana, United States, 70433
Sponsors and Collaborators
TCA Cellular Therapy
More Information
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Responsible Party: TCA Cellular Therapy
ClinicalTrials.gov Identifier: NCT01082653    
Other Study ID Numbers: 2008-ALS-I
First Posted: March 8, 2010    Key Record Dates
Last Update Posted: May 9, 2014
Last Verified: September 2013
Keywords provided by TCA Cellular Therapy:
ALS
Lou Gehrig's Disease
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases