Amyotrophic Lateral SclerosisPhase 1ALSNCT01082653
Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis
A Phase 1 study testing autologous bone marrow-derived stem cells in people with Amyotrophic Lateral Sclerosis, run by TCA Cellular Therapy at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- autologous bone marrow-derived stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2010to May 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 15Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read more
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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- autologous bone marrow-derived stem cells
- Keywords
- ALS, Lou Gehrig's Disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
autologous bone marrow-derived stem cellsBiological
All patients will receive a unique one-time intrathecal infusion of the cell product suspended in infusion medium.
| Study group | Role | What they receive |
|---|---|---|
| Safety | Experimental | infusion of autologous bone marrow-derived stem cells Receives: Biological: autologous bone marrow-derived stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: one year
Secondary outcomes (1)
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.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2010 (estimated)
- Study startMar 2010
- Primary completionMay 2014 (estimated)
- Study completionMay 2014 (estimated)
Why it stopped: Suspended due to lack of funding.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- TCA Cellular Therapy
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01082653
- Sponsor's protocol ID
- 2008-ALS-I
- Acronym
- ALS
- Status last verified
- Sep 2013
- First submitted
- 1 Mar 2010
- Last updated
- 9 May 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01082653
- Acronym / sponsor ID
- ALS / 2008-ALS-I
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Lou Gehrig's Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: Suspended due to lack of funding.
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- TCA Cellular TherapyIndustry (pharma, biotech, devices)
- Study start
- Mar 2010
- Primary completion
- May 2014 (estimated)
- Study completion
- May 2014 (estimated)
- First posted
- 8 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Safety | Experimental | Biological: autologous bone marrow-derived stem cellsinfusion of autologous bone marrow-derived stem cells |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| autologous bone marrow-derived stem cellsAll patients will receive a unique one-time intrathecal infusion of the cell product suspended in infusion medium. | Biological | Safety |
Primary outcome
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.
1 secondary outcome
Efficacy
Time frame: one year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2014.
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing autologous bone marrow-derived stem cells in people with Amyotrophic Lateral Sclerosis, run by TCA Cellular Therapy at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 9 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (time frame: one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 May 2014.