Hereditary Cerebellar Ataxia.Phase 2SCANCT01489267

A New Method to Treat Hereditary Cerebellar Ataxia - Umbilical Cord Mesenchymal Stem Cells Transplantation

A Phase 2 study testing stem cell transplantation in people with Hereditary Cerebellar Ataxia., run by General Hospital of Chinese Armed Police Forces at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 to 65 years
Conditions the study is for: Hereditary Cerebellar Ataxia.
What is being tested?: stem cell transplantation
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 1 siteChina
Study dates: Dec 2011to Jul 2014
Phase: Phase 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 18 to 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

18-65 years of age
Molecularly diagnosed SCA1

Who cannot join

Cognitively impaired individuals
Schizophrenics
Patients with severe kidney and liver disease, epilepsy, heart disease, pulmonary disease, cardiac arrhythmia, diabetes insipidus, leukemia and some other central neural disease (such as Parkinson disease, accidence of brain vascular et al)
Age less than 18 years, age greater than 65 years

Trial locations and contacts

1 site in 1 country.

China1 site

Yihua An
Beijing

Other trials in China

About this study

Official title: The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients

Hereditary cerebellar ataxia is a type of autosomal dominant genetic disease, lesions mainly involving the cerebellum, but the spinal cord and cranial nerves may also be some involvement. A total of 20 molecularly diagnosed SCA1 patients divided in two groups. One group accepted for the treatment of stem cell transplantation,the other group will be the control. Purpose of this project to prove that allogeneic umbilical cord mesenchymal stem cells are applied to clinical safely, and in the treatment of hereditary cerebellar ataxia is valid.

Read the detailed description

After admission the patients accepted transplantation would receive physical examination, blood and urine tests, electrocardiogram, electrophysiologic study;evaluation of SCA1 symptoms (balance, walking, dexterity, tremor, memory, mood and concentration),then stem cell therapy：after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The control only receive evaluation of SCA1 symptoms .

Conditions: Hereditary Cerebellar Ataxia.
Being tested: stem cell transplantation
Keywords: cerebellar ataxia, hereditary disease, stem cell
Medical subject headings: Spinocerebellar Degenerations, Cerebellar Ataxia, Genetic Diseases, Inborn

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

stem cell transplantationOther
Also called Cell therapy.
The patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments and the dose is about 2 ml(including 1×10'7 cells).

Study groups in NCT01489267
Study group	Role	What they receive
Control group	No intervention	Ten patients in the group only receive nerve functional evaluation and electrophysiology examination before and 1,3,6,12 months after recruit. They will not accept cell therapy.
Stem cell transplantation	Experimental	10 patients in the group accept stem cell transplantation. Receives: Other: stem cell transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Measured: within one week before transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test
2. ICARS score
3. Modified Falls Efficacy Scale
4. Berg balance assessment
5. Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
1. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Measured: within one week before transplantation
blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Measured: 1 month after transplantation
blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Measured: 3 months after transplantation
blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Measured: 6 months after transplantation
blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Measured: 12 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test
2. ICARS score
3. Modified Falls Efficacy Scale
4. Berg balance assessment
5. Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
1. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Measured: 1 month after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test
2. ICARS score
3. Modified Falls Efficacy Scale
4. Berg balance assessment
5. Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
1. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Measured: 3 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test
2. ICARS score
3. Modified Falls Efficacy Scale
4. Berg balance assessment
5. Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
1. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Measured: 6 months after transplantation
Nerve functional evaluation
1. Timed-up-and-go test ,5 minutes down-and-up test
2. ICARS score
3. Modified Falls Efficacy Scale
4. Berg balance assessment
5. Tremor Rating Scale:
- Part A - Tremor location/severity rating
- Part B -Handwriting and drawings
- Part C - Functional disabilities resulting from tremor
1. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Measured: 12 months after transplantation

Secondary outcomes (10)

Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Measured: within one week before transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Measured: within one week before transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Measured: 1 month after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Measured: 3 months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Measured: 6 months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin
Measured: 12 months after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Measured: 1 month after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Measured: 3 months after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Measured: 6 months after transplantation
Electrophysiology examination
somatosensory evoked potential(SEP)、brain stem auditory evoked potential(BAEP)
Measured: 12 months after transplantation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2011Jul 2014

First posted9 Dec 2011 (estimated)
Study startDec 2011
Primary completionJul 2013 (estimated)
Study completionJul 2014 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Hereditary Cerebellar Ataxia.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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 20 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01489267
Sponsor's protocol ID: 20111031SCA
Other IDs: 20111031limin
Acronym: SCA
Status last verified: Jun 2012
First submitted: 30 Oct 2011
Last updated: 20 Jun 2012
Results posted: No
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)
Spinocerebellar Ataxia

All Hereditary Cerebellar Ataxia. trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Jun 2012. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: The Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cells Transplantation in Hereditary Cerebellar Ataxia Patients
ClinicalTrials.gov ID: NCT01489267
Acronym / sponsor ID: SCA / 20111031SCA
Conditions: Hereditary Cerebellar Ataxia.
Keywords: cerebellar ataxia, hereditary disease, stem cell
Phase: Phase 2
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 20 participants (estimated)
Lead sponsor: General Hospital of Chinese Armed Police ForcesUniversity, hospital or other
Study start: Dec 2011
Primary completion: Jul 2013 (estimated)
Study completion: Jul 2014 (estimated)
First posted: 9 Dec 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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Control group	No intervention	–Ten patients in the group only receive nerve functional evaluation and electrophysiology examination before and 1,3,6,12 months after recruit. They will not accept cell therapy.
Stem cell transplantation	Experimental	Other: stem cell transplantation10 patients in the group accept stem cell transplantation.

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (2 listed)

18-65 years of age
Molecularly diagnosed SCA1

Key exclusion criteria (4 listed)

Cognitively impaired individuals
Schizophrenics
Patients with severe kidney and liver disease, epilepsy, heart disease, pulmonary disease, cardiac arrhythmia, diabetes insipidus, leukemia and some other central neural disease (such as Parkinson disease, accidence of brain vascular et al)
Age less than 18 years, age greater than 65 years

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 cell transplantationAlso: Cell therapy.The patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments and the dose is about 2 ml(including 1×10'7 cells).	Other	Stem cell transplantation

Primary outcome

blood test
Time frame: within one week before transplantation
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Nerve functional evaluation
Time frame: within one week before transplantation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
blood test
Time frame: 1 month after transplantation
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
blood test
Time frame: 3 months after transplantation
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
blood test
Time frame: 6 months after transplantation
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
blood test
Time frame: 12 months after transplantation
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers(AFP,CEA,CA125,CA15-3,CA19-9,CA72-4CY21-1,NSE) ,Lymphocytes classification
Nerve functional evaluation
Time frame: 1 month after transplantation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Time frame: 3 months after transplantation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Time frame: 6 months after transplantation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)
Nerve functional evaluation
Time frame: 12 months after transplantation
1. Timed-up-and-go test ,5 minutes down-and-up test 2. ICARS score 3. Modified Falls Efficacy Scale 4. Berg balance assessment 5. Tremor Rating Scale: * Part A - Tremor location/severity rating * Part B -Handwriting and drawings * Part C - Functional disabilities resulting from tremor 6. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI), alertness (Computerized Continuous Performing Tasks) and memory (Mini Mental State Examination)

10 secondary outcomes

Urinal test
Time frame: within one week before transplantation
Electrophysiology examination
Time frame: within one week before transplantation
Urinal test
Time frame: 1 month after transplantation
Urinal test
Time frame: 3 months after transplantation
Urinal test
Time frame: 6 months after transplantation
Urinal test
Time frame: 12 months after transplantation
Electrophysiology examination
Time frame: 1 month after transplantation
Electrophysiology examination
Time frame: 3 months after transplantation
Electrophysiology examination
Time frame: 6 months after transplantation
Electrophysiology examination
Time frame: 12 months after transplantation

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Jun 2012.

Related trials for Hereditary Cerebellar Ataxia.

RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
RecruitingObservational study
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Inherited Disease, Rare Diseases
Active, not recruiting
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
Friedreich Ataxia
Recruiting
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Friedreich's Ataxia, Motor Disorders
Active, not recruitingObservational study
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)
Spinocerebellar Ataxia

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.

Yihua An, doctor study director

director of neural stem cell transplantation department in general hospital of chinese armed police forces

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing stem cell transplantation in people with Hereditary Cerebellar Ataxia., run by General Hospital of Chinese Armed Police Forces 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 2 inclusion and 4 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 20 Jun 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 phase mean?

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?

blood test (time frame: within one week before transplantation). The study lists 10 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 NCT01489267.

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 20 Jun 2012.