Hereditary AtaxiaPhase 1/2NCT01360164
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
A Phase 1/2 study testing human umbilical cord mesenchymal stem cells in people with Hereditary Ataxia, run by Shenzhen Beike Bio-Technology Co., Ltd. at 1 site in China.
Trial summary
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- Conditions the study is for
- Hereditary Ataxia
- What is being tested?
- human umbilical cord mesenchymal stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Jan 2010to Dec 2013
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged 16-65 years.
- Harding Diagnosis of SCAs, gene type confirmed.
- Candidates who did not receive any stem cell therapy in past 6months.
- sign the consent form and follow the clinic trail procedure.
Who cannot join
- Cardiac insufficiency; Renal insufficiency; hepatic insufficiency; Total bilirubin higher than 1.5 times of upper limit of normal value; AST /ALT higher than 2.5 times of upper limit of normal value；
- Routine Blood Test: WBC count <3.0×109/ L; PLT count <5×109/L ; or Hemoglobin <100g/L；
- Combined Pneumonia or other Severe systemic bacteria infection；
- Severe drug allergic history or anaphylaxis to 2 or more food or medicine；
- Other brain organic disease (eg. Brain cancer)；
- HIV+, Tumor Markers + ；
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- Severe psychotic patients, cognitive dysfunction， or can not understand or sign the Consent Form；
- Other severe systemic or organic disease；
- Uncontrolled hypertension，blood pressure≥180mmHg/110 mmHg after treatment；
- Pregnancy；
- Enrollment in other trials in the last 3 months；
- Other criteria the investigator consider improper for inclusion.
Trial locations and contacts
1 site in 1 country.
Yun Xu contact
China1 site, 1 recruiting
- Recruiting
Nanjing University Medical College Affiliated Drum Tower Hospital
Nanjing, Jiangsu
Yun Xu, xuyun20042001@yahoo.com.cn
About this study
Official title: Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Current treatments for Hereditary Ataxias are mainly pharmacological, rehabilitative, or psychological treatments，while no effective treatment available. Stem Cell therapy is a novel and promising therapeutic strategy for Hereditary Ataxias treatment. In this study, the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation will be evaluated in patients with Hereditary Ataxias.
Read the detailed description
This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.
- Conditions
- Hereditary Ataxia
- Being tested
- human umbilical cord mesenchymal stem cells
- Keywords
- Human Umbilical Cord Mesenchymal Stem Cells
- Medical subject headings
- Spinocerebellar Degenerations
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human umbilical cord mesenchymal stem cellsBiological
Also called Intervention Group
Participants will be given hUC-MSCs transplantation.
| Study group | Role | What they receive |
|---|---|---|
| Human umbilical cord mesenchymal stem cells transplantation | Experimental | Participants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up. Receives: Biological: human umbilical cord mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale
Measured: 1 year after treatment
the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
Measured: 1 year after treatment
Secondary outcomes (2)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Measured: 1 year after treatment
Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability
Measured: 1 year after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2011 (estimated)
- Study startJan 2010
- Primary completionDec 2012 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hereditary AtaxiaStudies 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
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Nanjing Medical University, Nanjing University Medical College Affiliated Wuxi Second Hospital, Xuzhou Medical University, The Second Hospital of Nanjing Medical University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01360164
- Sponsor's protocol ID
- BKCR-HA-1.0(2010)
- Status last verified
- Nov 2012
- First submitted
- 23 May 2011
- Last updated
- 28 Nov 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Jin JL, Liu Z, Lu ZJ, Guan DN, Wang C, Chen ZB, Zhang J, Zhang WY, Wu JY, Xu Y. Safety and efficacy of umbilical cord mesenchymal stem cell therapy in hereditary spinocerebellar ataxia. Curr Neurovasc Res. 2013 Feb;10(1):11-20. doi: 10.2174/156720213804805936.
PubMed 23151076
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Hereditary Ataxia
- ClinicalTrials.gov ID
- NCT01360164
- Acronym / sponsor ID
- BKCR-HA-1.0(2010)
- Conditions
- Hereditary Ataxia
- Keywords
- Human Umbilical Cord Mesenchymal Stem Cells
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Nanjing Medical University, Nanjing University Medical College Affiliated Wuxi Second Hospital, Xuzhou Medical University, The Second Hospital of Nanjing Medical University
- Study start
- Jan 2010
- Primary completion
- Dec 2012 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 25 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.
The Hereditary Ataxias are a group of genetic disorders characterized by slowly progressive incoordination of gait and often associated with poor coordination of hands, speech, and eye movements. Current treatments for Hereditary Ataxias are mainly pharmacological, rehabilitative, or psychological treatments，while no effective treatment available. Stem Cell therapy is a novel and promising therapeutic strategy for Hereditary Ataxias treatment. In this study, the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation will be evaluated in patients with Hereditary Ataxias.
Detailed description
This Study is designed to evaluate the the safety and efficacy of Human Umbilical Cord Mesenchymal Stem Cells transplantation in patients with Hereditary Ataxias.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Human umbilical cord mesenchymal stem cells transplantation | Experimental | Biological: human umbilical cord mesenchymal stem cellsParticipants will be given umbilical cord mesenchymal stem cells transplantation with a 1 year follow-up. |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Aged 16-65 years.
- Harding Diagnosis of SCAs, gene type confirmed.
- Candidates who did not receive any stem cell therapy in past 6months.
- sign the consent form and follow the clinic trail procedure.
Key exclusion criteria (12 listed)
- Cardiac insufficiency; Renal insufficiency; hepatic insufficiency; Total bilirubin higher than 1.5 times of upper limit of normal value; AST /ALT higher than 2.5 times of upper limit of normal value；
- Routine Blood Test: WBC count <3.0×109/ L; PLT count <5×109/L ; or Hemoglobin <100g/L；
- Combined Pneumonia or other Severe systemic bacteria infection；
- Severe drug allergic history or anaphylaxis to 2 or more food or medicine；
- Other brain organic disease (eg. Brain cancer)；
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 |
|---|---|---|
| human umbilical cord mesenchymal stem cellsAlso: Intervention GroupParticipants will be given hUC-MSCs transplantation. | Biological | Human umbilical cord mesenchymal stem cells transplantation |
Primary outcome
scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale
Time frame: 1 year after treatment
the volume of Cerebellum of Brain Magnetic Resonance Imaging （MRI） Scan
Time frame: 1 year after treatment
2 secondary outcomes
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: 1 year after treatment
Number of Participants with Serious Adverse Events as a Measure of Safety and Tolerability
Time frame: 1 year after treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2012.
- Derived from this study: PubMed 23151076Jin JL, Liu Z, Lu ZJ, Guan DN, Wang C, Chen ZB, Zhang J, Zhang WY, Wu JY, Xu Y. Safety and efficacy of umbilical cord mesenchymal stem cell therapy in hereditary spinocerebellar ataxia. Curr Neurovasc Res. 2013 Feb;10(1):11-20. doi: 10.2174/156720213804805936.
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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.
Yun Xu contact
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 human umbilical cord mesenchymal stem cells in people with Hereditary Ataxia, run by Shenzhen Beike Bio-Technology Co., Ltd. at 1 site in China.
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 4 inclusion and 12 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 28 Nov 2012. 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 China. 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 Yun Xu (xuyun20042001@yahoo.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
scores of International Cooperative Ataxia Rating Scale （ICARS） scale and Berg Scale (time frame: 1 year after treatment). The study lists 2 primary outcomes in all. 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 NCT01360164, which also lists 1 linked publication.
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 28 Nov 2012.