Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]Phase 1/2NCT02855112
Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
A Phase 1/2 study testing Adipose derived mesenchymal stem cell in people with Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman], run by Tehran University of Medical Sciences at 1 site in Iran.
Trial summary
Spinal Muscular Atrophy (SMA) is an autosomal recessive disease of motor neurons. In the early 1980s, Werdnig from Vienna University and Hoffman from Heidelberg University described this disorder. So SMA type 1 was named Werdnig- Hoffman disease. Read more about this study
- Who is it for?
- Ages 5 to 12 months
- Conditions the study is for
- Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
- What is being tested?
- Adipose derived mesenchymal stem cell
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteIran
- Study dates
- Jun 2015to Jul 2017
- 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 5 to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age under 12 month, Weak muscle tone, Weakness in mobility, Patients sitting without full conduction of nerve Existence of home senses, Normal Brain function
Who cannot join
- Age beyound 12 month, Brain abnormality, Loss of sensory functions Malignancies
Trial locations and contacts
1 site in 1 country.
Iran1 site, 1 recruiting
- Recruiting
Children's Medical Center
Tehran, Tehran Province, 14194
Rashin Mohseni, PhD, +989123430627, rashin_mohseni@yahoo.com
About this study
Official title: The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients
Spinal Muscular Atrophy (SMA) is an autosomal recessive disease of motor neurons. In the early 1980s, Werdnig from Vienna University and Hoffman from Heidelberg University described this disorder. So SMA type 1 was named Werdnig- Hoffman disease. This is the first genetic disorder that cause death after cystic fibrosis in infants with the prevalence of 1 in 6000 birth. Mutation in the SMN1 gene (Survival Motor Neuron) is the reason for the disease that cause decrease in the SMN protein production. So the alpha motor neurons in the spinal cord ventricle horn will be destroyed and it cause progressive paralysis and defenite death.No specific therapy is yet available for the treatment of Werdnig-Hoffmann disease. Treatment is not disease-modifying and just is supportive. SMA type 1 is diagnosed within the early 6 month after birth and accompanied with breath disorders and definite death in 2 years. The affected infants have a weak muscle tone and they couldn't even hold their head up. Perhaps the only open way for these patients is the application of stem cells that could deliver trophic factor to the apoptotic cells. So this study focuses on the effectivness of cell therapy via adipose derived mesenchymal stem cells on the probable phenotypic changes in these patients.
- Being tested
- Adipose derived mesenchymal stem cell
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adipose derived mesenchymal stem cellBiological
Also called Cell Therapy
Allogeneic Adipose derived Mesenchymal Stem cell transplant
| Study group | Role | What they receive |
|---|---|---|
| Control | No intervention | A group of 10 patients only will be subjected to electro-myogram test every 3 month and then follow up for their survival time without any cell therapy intervention. |
| Adipose derived Mesenchymal Stem cell | Experimental | A group of 10 patients will be take stem cells intra-thecally Dose: 1 million cells/kg for three times Intervals: Every 3 weeks. Receives: Biological: Adipose derived mesenchymal stem cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in action potential of muscles on ElectroMyoGram (EMG) test
Measure the electrical activity of muscles by Electromyography
Measured: Change from Baseline of intervention at 3 month
Secondary outcomes (1)
Changes in Motility on Modified Barthel Index Score
Measure any phenotypic changes in patients motion by direct Observation on Modified Barthel Index Score
Measured: Change from Baseline of intervention at 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2016 (estimated)
- Study startJun 2015
- Primary completionDec 2016 (estimated)
- Study completionJul 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]Studies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tehran University of Medical Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amir Ali Hamidieh, Tehran University of Medical Sciences
- Investigators
- Mahmoode Reza Ashrafi, MD, Principal investigator, Children's Medical Hospital, Tehran University of Medical SciencesAmir Ali Hamidieh, MD, Study chair, Hematology-Oncology & Stem cell Transplant Research center, Tehran University of Medical SciencesRashin Mohseni, PhD, Study director, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02855112
- Sponsor's protocol ID
- 94-01-87-28524
- Status last verified
- Aug 2016
- First submitted
- 22 Jul 2016
- Last updated
- 4 Aug 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 4 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients
- ClinicalTrials.gov ID
- NCT02855112
- Acronym / sponsor ID
- 94-01-87-28524
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Tehran University of Medical SciencesUniversity, hospital or other
- Study start
- Jun 2015
- Primary completion
- Dec 2016 (estimated)
- Study completion
- Jul 2017 (estimated)
- First posted
- 4 Aug 2016 (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.
Spinal Muscular Atrophy (SMA) is an autosomal recessive disease of motor neurons. In the early 1980s, Werdnig from Vienna University and Hoffman from Heidelberg University described this disorder. So SMA type 1 was named Werdnig- Hoffman disease. This is the first genetic disorder that cause death after cystic fibrosis in infants with the prevalence of 1 in 6000 birth. Mutation in the SMN1 gene (Survival Motor Neuron) is the reason for the disease that cause decrease in the SMN protein production. So the alpha motor neurons in the spinal cord ventricle horn will be destroyed and it cause progressive paralysis and defenite death.No specific therapy is yet available for the treatment of Werdnig-Hoffmann disease. Treatment is not disease-modifying and just is supportive. SMA type 1 is diagnosed within the early 6 month after birth and accompanied with breath disorders and definite death in 2 years. The affected infants have a weak muscle tone and they couldn't even hold their head up. Perhaps the only open way for these patients is the application of stem cells that could deliver trophic factor to the apoptotic cells. So this study focuses on the effectivness of cell therapy via adipose derived mesenchymal stem cells on the probable phenotypic changes in these patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control | No intervention | –A group of 10 patients only will be subjected to electro-myogram test every 3 month and then follow up for their survival time without any cell therapy intervention. |
| Adipose derived Mesenchymal Stem cell | Experimental | Biological: Adipose derived mesenchymal stem cellA group of 10 patients will be take stem cells intra-thecally Dose: 1 million cells/kg for three times Intervals: Every 3 weeks. |
Who may qualify
- Ages
- 5 to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Age under 12 month, Weak muscle tone, Weakness in mobility, Patients sitting without full conduction of nerve Existence of home senses, Normal Brain function
Key exclusion criteria (1 listed)
- Age beyound 12 month, Brain abnormality, Loss of sensory functions Malignancies
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 |
|---|---|---|
| Adipose derived mesenchymal stem cellAlso: Cell TherapyAllogeneic Adipose derived Mesenchymal Stem cell transplant | Biological | Adipose derived Mesenchymal Stem cell |
Primary outcome
Changes in action potential of muscles on ElectroMyoGram (EMG) test
Time frame: Change from Baseline of intervention at 3 month
Measure the electrical activity of muscles by Electromyography
1 secondary outcome
Changes in Motility on Modified Barthel Index Score
Time frame: Change from Baseline of intervention at 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Aug 2016.
- Individual participant dataSharing planned: Undecided
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.
Mahmoode Reza Ashrafi, MD principal investigator
Children's Medical Hospital, Tehran University of Medical Sciences
Amir Ali Hamidieh, MD study chair
Hematology-Oncology & Stem cell Transplant Research center, Tehran University of Medical Sciences
Rashin Mohseni, PhD study director
School of Advanced Technologies in Medicine, Tehran University of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Adipose derived mesenchymal stem cell in people with Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman], run by Tehran University of Medical Sciences at 1 site in Iran.
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 1 inclusion and 1 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 4 Aug 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 Iran. 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 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?
Contact Rashin Mohseni, PhD (+989123230627, rashin_mohseni@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in action potential of muscles on ElectroMyoGram (EMG) test (time frame: Change from Baseline of intervention at 3 month). 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 4 Aug 2016.