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

This study has suspended participant recruitment.
(Suspended due to lack of funding.)
Sponsor:
Information provided by (Responsible Party):
TCA Cellular Therapy
ClinicalTrials.gov Identifier:
NCT01082653
First received: March 1, 2010
Last updated: May 7, 2014
Last verified: September 2013
  Purpose

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 Intervention Phase
Amyotrophic Lateral Sclerosis
Biological: autologous bone marrow-derived stem cells
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by TCA Cellular Therapy:

Primary Outcome Measures:
  • Safety [ Time Frame: one year ] [ Designated as safety issue: Yes ]
    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:
  • Efficacy [ Time Frame: one year ] [ Designated as safety issue: No ]

    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.



Estimated Enrollment: 6
Study Start Date: March 2010
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01082653

Locations
United States, Louisiana
TCA Cellular Therapy
Covington, Louisiana, United States, 70433
Sponsors and Collaborators
TCA Cellular Therapy
  More Information

No publications provided

Responsible Party: TCA Cellular Therapy
ClinicalTrials.gov Identifier: NCT01082653     History of Changes
Other Study ID Numbers: 2008-ALS-I
Study First Received: March 1, 2010
Last Updated: May 7, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by TCA Cellular Therapy:
ALS
Lou Gehrig's Disease

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on August 28, 2014