Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Suspended
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
autologous bone marrow-derived stem cells
Sponsored by
About this trial
This is an interventional diagnostic trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Lou Gehrig's Disease
Eligibility Criteria
Inclusion Criteria:
- Adult male and female subjects > 18 years of age.
- Good understanding of the protocol and willingness to consent.
- Moderate to severe Diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
- Vital capacity at least 50% predicted value for gender, height and age.
- More than 6 and less than 36 months of evolution of the disease.
- Hematocrit greater than 30 % prior to bone marrow aspiration.
- Platelet count greater than 100 Thousand/uL at screening.
- INR less than or equal to 1.5.
Exclusion Criteria:
- Any concurrent illness, which affects the bone marrow.
- Any concomitant medication that affects the bone marrow.
- Previous stem cell therapy.
- Any lymphoproliferative disease.
- Riluzole with 4 weeks of study entry and at any time during the study.
- Hemophiliacs or subjects with bleeding disorders.
- Known hypersensitivity to fetal bovine serum
- HIV infection.
- Serum creatinine > 3.0 in subjects not on hemodialysis.
- Skin infection at the infusion site or systemic infection
- Current smoker.
- Active drug or alcohol addiction
- Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential.
- Subjects that are breast feeding.
- Any condition that the Principal Investigator considers would render the subject unfit for the study.
Sites / Locations
- TCA Cellular Therapy
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Safety
Arm Description
infusion of autologous bone marrow-derived stem cells
Outcomes
Primary Outcome Measures
Safety
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
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01082653
Brief Title
Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis
Acronym
ALS
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 Type
Interventional
2. Study Status
Record Verification Date
September 2013
Overall Recruitment Status
Suspended
Why Stopped
Suspended due to lack of funding.
Study Start Date
March 2010 (undefined)
Primary Completion Date
May 2014 (Anticipated)
Study Completion Date
May 2014 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
TCA Cellular Therapy
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Lou Gehrig's Disease
7. Study Design
Primary Purpose
Diagnostic
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Safety
Arm Type
Experimental
Arm Description
infusion of autologous bone marrow-derived stem cells
Intervention Type
Biological
Intervention Name(s)
autologous bone marrow-derived stem cells
Intervention Description
All patients will receive a unique one-time intrathecal infusion of the cell product suspended in infusion medium.
Primary Outcome Measure Information:
Title
Safety
Description
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.
Time Frame
one year
Secondary Outcome Measure Information:
Title
Efficacy
Description
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.
Time Frame
one year
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult male and female subjects > 18 years of age.
Good understanding of the protocol and willingness to consent.
Moderate to severe Diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
Vital capacity at least 50% predicted value for gender, height and age.
More than 6 and less than 36 months of evolution of the disease.
Hematocrit greater than 30 % prior to bone marrow aspiration.
Platelet count greater than 100 Thousand/uL at screening.
INR less than or equal to 1.5.
Exclusion Criteria:
Any concurrent illness, which affects the bone marrow.
Any concomitant medication that affects the bone marrow.
Previous stem cell therapy.
Any lymphoproliferative disease.
Riluzole with 4 weeks of study entry and at any time during the study.
Hemophiliacs or subjects with bleeding disorders.
Known hypersensitivity to fetal bovine serum
HIV infection.
Serum creatinine > 3.0 in subjects not on hemodialysis.
Skin infection at the infusion site or systemic infection
Current smoker.
Active drug or alcohol addiction
Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential.
Subjects that are breast feeding.
Any condition that the Principal Investigator considers would render the subject unfit for the study.
Facility Information:
Facility Name
TCA Cellular Therapy
City
Covington
State/Province
Louisiana
ZIP/Postal Code
70433
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis
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