Amyotrophic Lateral SclerosisPhase 1ALSNCT01348451
Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
A Phase 1 study testing surgical implantation in people with Amyotrophic Lateral Sclerosis, run by Neuralstem Inc. at 1 site in United States.
Trial summary
This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The goal of the study is to see if the cells and the procedure to transplant them are safe. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- surgical implantation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2009to Dec 2016
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) [per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling] and comply with the study procedures.
- Subjects with sporadic or familial ALS diagnosed as laboratory-supported probable,probable or definite according to the World Federation of Neurology El Escorial Criteria (Appendix A), based on examination by the site PI.
- Age 18 years or older.
- Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or surgically sterile [hysterectomy, oophorectomy or surgical sterilization]).
- Geographic accessibility to the study center and the ability to travel to the clinic for study visits.
- Presence of a willing and able caregiver.
Show all 11Show fewer
- Medically able to undergo lumbar or cervical laminectomy as determined by the Investigator, surgeon and anesthesiologist.
- Medically able to tolerate immunosuppression regimen consisting of basiliximab, tacrolimus, mycophenolate mofetil, and methylprednisolone as determined by the site Investigator.
- Agrees to the visit schedule as outlined in the informed consent.
- Not taking riluzole (Rilutek®) or on a stable dose for ≥30 days.
- All required vaccinations current: tetanus/diptheria (TDAP), herpes zoster/shingles(Vostavax®: within last 10 years and must be prior to surgery), pneumonia (Pneumovax®),seasonal/H1N1 flu vaccines (as appropriate for season) for Groups B-E.
Who cannot join
- Etiology of paraplegia or weakness is due to causes other than ALS such as spinal ischemia, traumatic spinal injury, traumatic brain injury, multiple sclerosis, cerebral stroke, cerebral palsy, or infection.
- VC < 60% predicted normal by standard nomogram at the time of screening and VC < 50% predicted normal measured supine for age at the time of surgery.
- Current or peak Panel Reactive Antibody (PRA) due to alloantibodies > 20% receiving their first allograft.
- Any known immunodeficiency syndrome.
- Receipt of any investigational drug,device or biologic within 30 days of surgery.
- Any concomitant medical disease or condition limiting the safety to participate:
Show all 25Show fewer
- Coagulopathy
- Active uncontrolled infection
- Hypotension requiring vasopressor therapy
- Previous spinal surgery at the site of planned transplantation except for anterior cervical dissection fusion (ACDF)
- Skin breakdown over the site of surgery
- Malignancy (except for non-melanoma skin cancer)
- Primary or secondary immune deficiency
- Spinal stenosis.
- Creatinine >1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) > 2x the upper limit of normal, hematocrit/hemoglobin < 30/10, total WBC < 4000, uncontrolled hypertension (defined as systolic >180 or diastolic >100) or uncontrolled diabetes(defined as hemoglobin A1C >8), evidence of GI bleeding by hemoccult test, positive tuberculosis (TB test: PPD/Mantoux), hepatitis B or C, or human immunodeficiency virus (HIV).
- Presence of any of the following conditions:
- Current drug abuse or alcoholism
- Unstable medical conditions
- Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening
- Positive blood test for hepatitis B or C.
- Any condition that the site PI feels may interfere with participation in the study.
- Any condition that the surgeon feels may pose complications for the surgery.
- Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, or methylprednisolone.
- Inability to provide informed consent as determined by screening protocol.
- Inadequate family or caregiver support as determined by the site PI.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase l, Open-label, First in Human, Feasibility and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The goal of the study is to see if the cells and the procedure to transplant them are safe.
Read the detailed description
These stem cells are called Human Spinal Stem Cells (HSSC) and have been engineered from the spinal cord of a single fetus electively aborted after 8 weeks of gestation. The tissue was obtained with the mother's consent. The cells will be transplanted into the ALS patient's spinal cord after laminectomy, an operation that removes bone surrounding the spine. After the spinal cord is exposed, a device manufactured for this purpose will be mounted onto the patient and will hold a syringe filled with the cells. The syringe will have a needle attached and the needle will enter the spinal cord in specified areas. The device will minimize trauma to the spinal cord by the needle by making the puncture precise and steady and injecting the material at a slow and steady speed.
ALS is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is 2-5 years. The cause is unknown and there is no effective treatment. Previous research has shown that on autopsy, ALS patients are found to have increased levels of the amino acid glutamate accumulated in the brain and spinal cord. This increase is thought to be caused by a decrease in the glutamate transporter which normally "cleans up" glutamate from the cells.
Because the HSSC are human in origin, their transplantation will be handled in some ways like other organ transplants in that patients will receive immunosuppressive medications to prevent the rejection of the cells. Right before and immediately after surgery patients will receive infusions of a drug called basiliximab. After surgery they will take prednisone and be tapered off that medication over one month. They will also be given two other immunosuppressive agents, tacrolimus and mycophenolate mofetil after surgery and it is expected that the patients will take these drugs for the rest of their lives.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- surgical implantation
- Keywords
- Human spinal cord derived neural stem cell transplantation, ALS
- 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.
surgical implantationDevice
human spinal cord stem cell implantation in ALS patients
| Study group | Role | What they receive |
|---|---|---|
| surgery | Experimental | A sequential design of five groups will be utilized to reduce risk to subjects. The first group (Group A) will include six subjects and the subsequent groups will include three subjects per group. Each group represents both different inclusion criteria and location of surgery. Receives: Device: surgical implantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary objective of this study is to determine the safety of human spinal cord-derived neural stem cell transplantation for the treatment of amyotrophic lateral sclerosis.
The primary outcome measure is the incidence of adverse events in the study population.
Measured: The primary outcome measure will be assessed at study visits pre and post surgery follow-up visits, for a total of 48 months.
Secondary outcomes (11)
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
The Secondary Outcome Measures of the study are to evaluate spinal stem cell transplantation therapy in this patient population using the following 11 assessments. 1. ALSFRS-R is a self-administered ordinal rating scale questionnaire (rating 0-4 for each question, 4 is most functional, 0-48 total) of 12 functional activities. The most functional total score is 48.
Measured: The ALSFRS-R will be administered at -3, -2 and -1 months screening/presurgery, and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Quantitative muscle strength testing using a hand held dynamometer (HHD): Six proximal muscle groups (knee flexion and extension, hip flexion, elbow flexion and extension and shoulder flexion) and three proximal muscle groups (wrist extension, first dorsal interosseous contraction, and ankle dorsiflexion) will be tested bilaterally using the MICROFET 2 HHD.
Measured: The quantitative muscle strength test will be administered at -3, -2, -1 months screening/presurgery and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Hand grip (bilateral) will be measured using the Jaymar Grip dynamometer.
Measured: The hand grip (bilateral) will be administered at -3, -2 and -1 months screening/presurgery, and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Vital Capacity (VC) will be measured using the Renaissance II spirometer. Eligibility will be determined with seated and supine measurements but the secondary outcome measure will be done seated.
Measured: The vital capacity (VC) will be measured at -3, -2 and -1 months and -7 days screening/presurgery. It will also be measured at 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Negative Inspiratory Force (NIF) will be measured with a Negative Inspiratory Force Gauge (Instrumentation Industries).
Measured: The negative inspiratory force (NIF) will be measured at -3, -2 and -1 months and -7 days screening/presurgery. It will also be measured at 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Electrical Impedance Myography (EIM) is a painless and non-invasive quantitative measure of muscle that has been shown to correlate with other physiological and semi-quantitative measures of disease progression in ALS .
Measured: The Electrical Impedance Myography (EIM) will be measured at -3, -2 and -1 months screening/presurgery. It will also be measured at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Comprehensive pain assessment utilizing the Neuropathic Pain Scale (NPS).
Measured: The comprehensive pain assessment will be performed at -3 months and -14 days screening/presurgery. It will also be performed at 2 weeks, 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- An MRI will be performed on the entire spine and brain with and without gadolinium contrast. An MRI of the targeted region may be performed if clinically indicated at the 2 week visit.
Measured: Performed during the screening and at 24 and 48 month follow-up/post surgery visits. MRI of the surgical region only, at 1, 6, 12, 18 and 36 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Urodynamic parameters including incontinence and urinary retention measured by post void residual.
Measured: Measurements will be taken at -3 months and -14 days at screening/presurgery and at 1, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- ALS Specific Quality of Life Questionnaire - Revised is a scale that balances physical and nonphysical factors in assessing the quality of life in ALS patients
Measured: The questionnaire will be completed at -3 months during the screening/presurgery phase and at the 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
- Ashworth Spasticity Scale.
Measured: The Ashworth Spasticity Scale will be administered at -3, -2, -1 months screening/presurgery and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2011 (estimated)
- Study startJan 2009
- Primary completionDec 2015
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neuralstem Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01348451
- Sponsor's protocol ID
- NS2008-1
- Acronym
- ALS
- Status last verified
- Mar 2016
- First submitted
- 28 Apr 2011
- Last updated
- 10 Mar 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Boulis NM, Federici T, Glass JD, Lunn JS, Sakowski SA, Feldman EL. Translational stem cell therapy for amyotrophic lateral sclerosis. Nat Rev Neurol. 2011 Dec 13;8(3):172-6. doi: 10.1038/nrneurol.2011.191.
PubMed 22158518Lunn JS, Sakowski SA, Federici T, Glass JD, Boulis NM, Feldman EL. Stem cell technology for the study and treatment of motor neuron diseases. Regen Med. 2011 Mar;6(2):201-13. doi: 10.2217/rme.11.6.
PubMed 21391854Glass JD. The promise and the reality of stem-cell therapies for neurodegenerative diseases. Cerebrum. 2010 Nov;2010:24. Epub 2010 Dec 15.
PubMed 23447769Boulis N, Federici T. Surgical approach and safety of spinal cord stem cell transplantation. Neurosurgery. 2011 Feb;68(2):E599-600. doi: 10.1227/NEU.0b013e3182095e2e. No abstract available.
PubMed 21654562Glass JD, Boulis NM, Johe K, Rutkove SB, Federici T, Polak M, Kelly C, Feldman EL. Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients. Stem Cells. 2012 Jun;30(6):1144-51. doi: 10.1002/stem.1079.
PubMed 22415942Riley J, Federici T, Polak M, Kelly C, Glass J, Raore B, Taub J, Kesner V, Feldman EL, Boulis NM. Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes. Neurosurgery. 2012 Aug;71(2):405-16; discussion 416. doi: 10.1227/NEU.0b013e31825ca05f.
PubMed 22565043Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci. 2013 Apr;14(4):248-64. doi: 10.1038/nrn3430. Epub 2013 Mar 6.
PubMed 23463272
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase l, Open-label, First in Human, Feasibility and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT01348451
- Acronym / sponsor ID
- ALS / NS2008-1
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Human spinal cord derived neural stem cell transplantation, ALS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Neuralstem Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2009
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2016 (estimated)
- First posted
- 5 May 2011 (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.
This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The goal of the study is to see if the cells and the procedure to transplant them are safe.
Detailed description
These stem cells are called Human Spinal Stem Cells (HSSC) and have been engineered from the spinal cord of a single fetus electively aborted after 8 weeks of gestation. The tissue was obtained with the mother's consent. The cells will be transplanted into the ALS patient's spinal cord after laminectomy, an operation that removes bone surrounding the spine. After the spinal cord is exposed, a device manufactured for this purpose will be mounted onto the patient and will hold a syringe filled with the cells. The syringe will have a needle attached and the needle will enter the spinal cord in specified areas. The device will minimize trauma to the spinal cord by the needle by making the puncture precise and steady and injecting the material at a slow and steady speed.
ALS is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is 2-5 years. The cause is unknown and there is no effective treatment. Previous research has shown that on autopsy, ALS patients are found to have increased levels of the amino acid glutamate accumulated in the brain and spinal cord. This increase is thought to be caused by a decrease in the glutamate transporter which normally "cleans up" glutamate from the cells.
Because the HSSC are human in origin, their transplantation will be handled in some ways like other organ transplants in that patients will receive immunosuppressive medications to prevent the rejection of the cells. Right before and immediately after surgery patients will receive infusions of a drug called basiliximab. After surgery they will take prednisone and be tapered off that medication over one month. They will also be given two other immunosuppressive agents, tacrolimus and mycophenolate mofetil after surgery and it is expected that the patients will take these drugs for the rest of their lives.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| surgery | Experimental | Device: surgical implantationA sequential design of five groups will be utilized to reduce risk to subjects. The first group (Group A) will include six subjects and the subsequent groups will include three subjects per group. Each group represents both different inclusion criteria and location of surgery. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) [per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling] and comply with the study procedures.
- Subjects with sporadic or familial ALS diagnosed as laboratory-supported probable,probable or definite according to the World Federation of Neurology El Escorial Criteria (Appendix A), based on examination by the site PI.
- Age 18 years or older.
- Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or surgically sterile [hysterectomy, oophorectomy or surgical sterilization]).
- Geographic accessibility to the study center and the ability to travel to the clinic for study visits.
Key exclusion criteria (25 listed)
- Etiology of paraplegia or weakness is due to causes other than ALS such as spinal ischemia, traumatic spinal injury, traumatic brain injury, multiple sclerosis, cerebral stroke, cerebral palsy, or infection.
- VC < 60% predicted normal by standard nomogram at the time of screening and VC < 50% predicted normal measured supine for age at the time of surgery.
- Current or peak Panel Reactive Antibody (PRA) due to alloantibodies > 20% receiving their first allograft.
- Any known immunodeficiency syndrome.
- Receipt of any investigational drug,device or biologic within 30 days of surgery.
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 |
|---|---|---|
| surgical implantationhuman spinal cord stem cell implantation in ALS patients | Device | surgery |
Primary outcome
The primary objective of this study is to determine the safety of human spinal cord-derived neural stem cell transplantation for the treatment of amyotrophic lateral sclerosis.
Time frame: The primary outcome measure will be assessed at study visits pre and post surgery follow-up visits, for a total of 48 months.
The primary outcome measure is the incidence of adverse events in the study population.
11 secondary outcomes
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The ALSFRS-R will be administered at -3, -2 and -1 months screening/presurgery, and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The quantitative muscle strength test will be administered at -3, -2, -1 months screening/presurgery and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The hand grip (bilateral) will be administered at -3, -2 and -1 months screening/presurgery, and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The vital capacity (VC) will be measured at -3, -2 and -1 months and -7 days screening/presurgery. It will also be measured at 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The negative inspiratory force (NIF) will be measured at -3, -2 and -1 months and -7 days screening/presurgery. It will also be measured at 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The Electrical Impedance Myography (EIM) will be measured at -3, -2 and -1 months screening/presurgery. It will also be measured at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The comprehensive pain assessment will be performed at -3 months and -14 days screening/presurgery. It will also be performed at 2 weeks, 1, 2, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: Performed during the screening and at 24 and 48 month follow-up/post surgery visits. MRI of the surgical region only, at 1, 6, 12, 18 and 36 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: Measurements will be taken at -3 months and -14 days at screening/presurgery and at 1, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The questionnaire will be completed at -3 months during the screening/presurgery phase and at the 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
The Secondary Objectives of the study are to evaluate spinal stem cell transplantation therapy in this patient population.
Time frame: The Ashworth Spasticity Scale will be administered at -3, -2, -1 months screening/presurgery and at 1, 3, 6, 9, 12, 18, 24, 30, 36, 42 and 48 month follow-up/post surgery visits.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2016.
- Background: PubMed 22158518Boulis NM, Federici T, Glass JD, Lunn JS, Sakowski SA, Feldman EL. Translational stem cell therapy for amyotrophic lateral sclerosis. Nat Rev Neurol. 2011 Dec 13;8(3):172-6. doi: 10.1038/nrneurol.2011.191.
- Background: PubMed 21391854Lunn JS, Sakowski SA, Federici T, Glass JD, Boulis NM, Feldman EL. Stem cell technology for the study and treatment of motor neuron diseases. Regen Med. 2011 Mar;6(2):201-13. doi: 10.2217/rme.11.6.
- Background: PubMed 23447769Glass JD. The promise and the reality of stem-cell therapies for neurodegenerative diseases. Cerebrum. 2010 Nov;2010:24. Epub 2010 Dec 15.
- Background: PubMed 21654562Boulis N, Federici T. Surgical approach and safety of spinal cord stem cell transplantation. Neurosurgery. 2011 Feb;68(2):E599-600. doi: 10.1227/NEU.0b013e3182095e2e. No abstract available.
- Results publication: PubMed 22415942Glass JD, Boulis NM, Johe K, Rutkove SB, Federici T, Polak M, Kelly C, Feldman EL. Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients. Stem Cells. 2012 Jun;30(6):1144-51. doi: 10.1002/stem.1079.
- Results publication: PubMed 22565043Riley J, Federici T, Polak M, Kelly C, Glass J, Raore B, Taub J, Kesner V, Feldman EL, Boulis NM. Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I safety trial, technical note, and lumbar safety outcomes. Neurosurgery. 2012 Aug;71(2):405-16; discussion 416. doi: 10.1227/NEU.0b013e31825ca05f.
- Derived from this study: PubMed 23463272Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci. 2013 Apr;14(4):248-64. doi: 10.1038/nrn3430. Epub 2013 Mar 6.
- Related InfoLink provided by the sponsor.
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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 surgical implantation in people with Amyotrophic Lateral Sclerosis, run by Neuralstem Inc. 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 11 inclusion and 25 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: status not confirmed, as of the sponsor's last update on 10 Mar 2016. 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?
The primary objective of this study is to determine the safety of human spinal cord-derived neural stem cell transplantation for the treatment of amyotrophic lateral sclerosis. (time frame: The primary outcome measure will be assessed at study visits pre and post surgery follow-up visits, for a total of 48 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01348451, which also lists 7 linked publications.
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 10 Mar 2016.