Motor Neuron DiseasePhase 1/2NCT03067857
Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
A Phase 1/2 study testing Stem Cells in people with Motor Neuron Disease and Amyotrophic Lateral Sclerosis, run by Stem Cells Arabia.
Trial summary
Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route. Read more about this study
- Who is it for?
- Ages 24 to 70 years
- Conditions the study is for
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis
- What is being tested?
- Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- Sep 2016to Aug 2019
- Phase
- Phase 1/2
- 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 24 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients age 24-20 years old
- Patients with the diagnosis of definite Motor Neuron Disease
Who cannot join
- HIV/HBV/HCV
- Malignancies
- Bleeding
- Pneumonia
- Renal failure
- liver dysfunction
Show all 11Show fewer
- Severe anemia (hb < 8)
- Bone Marrow diseases
- Pregnancy and lactation
- Acute medical conditions/infections such as respiratory infections
- ventilatory support
About this study
Official title: Autologous Purified Bone-Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.
Read the detailed description
A motor neuron disease (MND) is any of several neurological disorders that selectively affect motor neurons, the cells that control voluntary muscles of the body. They include amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), progressive bulbar palsy (PBP) and pseudobulbar palsy; spinal muscular atrophies are also sometimes included in the group.They are neurodegenerative diseases that cause increasing disability and eventually, death.
In spite of intensive research, motor neuron diseases (MNDs) are still incurable. Some of the key factors in this failure to find a cure have been the lack of human cell-based disease models for molecular analysis and drug screening and the difficulty of obtaining homogeneous populations of specific cell types for clinical applications. Stem cell biology has the potential to transform our understanding of disease processes and to revolutionize our approach to develop novel therapies for MNDs.
In this study, the investigators suggest a protocol for the treatment of MND by transplantation of purified autologous bone marrow-derived stem cells via the intrathecal and intravenous route.
- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Progressive Bulbar Palsies
- Being tested
- Stem Cells
- Medical subject headings
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Muscular Atrophy, Spinal, Bulbar Palsy, Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem CellsBiological
Autologous bone marrow-derived stem cell populations injected via the intravenous and intrathecal routes.
| Study group | Role | What they receive |
|---|---|---|
| Stem Cells | Experimental | Intravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations. Receives: Biological: Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in Functional Rating Scale - via questionnaire
Measured: 6 months
Secondary outcomes (3)
Change in Appel Scale
Measured: 4 months
Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)
Measured: 4 months
Change in Forced Vital Capacity (FVC)
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2017
- Study startSep 2016
- Primary completionJan 2019 (estimated)
- Study completionAug 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Motor Neuron DiseaseStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stem Cells Arabia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03067857
- Sponsor's protocol ID
- SCA-MND1
- Status last verified
- Jun 2018
- First submitted
- 25 Feb 2017
- Last updated
- 6 Jun 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Purified Bone-Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
- ClinicalTrials.gov ID
- NCT03067857
- Acronym / sponsor ID
- SCA-MND1
- Conditions
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Progressive Bulbar Palsies
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Stem Cells ArabiaUniversity, hospital or other
- Study start
- Sep 2016
- Primary completion
- Jan 2019 (estimated)
- Study completion
- Aug 2019 (estimated)
- First posted
- 1 Mar 2017 (actual)
- 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.
Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.
Detailed description
A motor neuron disease (MND) is any of several neurological disorders that selectively affect motor neurons, the cells that control voluntary muscles of the body. They include amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), progressive bulbar palsy (PBP) and pseudobulbar palsy; spinal muscular atrophies are also sometimes included in the group.They are neurodegenerative diseases that cause increasing disability and eventually, death.
In spite of intensive research, motor neuron diseases (MNDs) are still incurable. Some of the key factors in this failure to find a cure have been the lack of human cell-based disease models for molecular analysis and drug screening and the difficulty of obtaining homogeneous populations of specific cell types for clinical applications. Stem cell biology has the potential to transform our understanding of disease processes and to revolutionize our approach to develop novel therapies for MNDs.
In this study, the investigators suggest a protocol for the treatment of MND by transplantation of purified autologous bone marrow-derived stem cells via the intrathecal and intravenous route.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem Cells | Experimental | Biological: Stem CellsIntravenous and Intrathecal thecal transplantation via interventional radiology of purified autologous bone-marrow derived specific stem cell populations. |
Who may qualify
- Ages
- 24 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients age 24-20 years old
- Patients with the diagnosis of definite Motor Neuron Disease
Key exclusion criteria (11 listed)
- HIV/HBV/HCV
- Malignancies
- Bleeding
- Pneumonia
- Renal failure
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 |
|---|---|---|
| Stem CellsAutologous bone marrow-derived stem cell populations injected via the intravenous and intrathecal routes. | Biological | Stem Cells |
Primary outcome
Changes in Functional Rating Scale - via questionnaire
Time frame: 6 months
3 secondary outcomes
Change in Appel Scale
Time frame: 4 months
Change in SF-36 (The Short Form (36) Health Survey is a 36 Item)
Time frame: 4 months
Change in Forced Vital Capacity (FVC)
Time frame: 4 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2018.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Stem Cells in people with Motor Neuron Disease and Amyotrophic Lateral Sclerosis, run by Stem Cells Arabia.
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 2 inclusion and 11 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 6 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Changes in Functional Rating Scale - via questionnaire (time frame: 6 months). 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 6 Jun 2018.