Anemia, AplasticPhase 1/2NCT00399971
Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia
A Phase 1/2 study testing Ex Vivo Immunotherapy in people with Anemia, Aplastic, run by Shenzhen Zhongxing Yangfan Biotech Co. Ltd. at 1 site in China.
Trial summary
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation. Read more about this study
- Who is it for?
- Ages 10 to 80 years
- Conditions the study is for
- Anemia, Aplastic
- What is being tested?
- Ex Vivo Immunotherapy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteChina
- Study dates
- Nov 2002to Dec 2009
- 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 10 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Clinical Diagnosis of Idiopathic Aplastic Anemia
- Not Eligible for Bone Marrow Transplantation
- Not Responsive to Conventional Immunosuppressive Therapy
Who cannot join
- Leukemia or MDS or PNH or Fanconi's Disease
- pregnancy
- Allergic to Blood Product
- Severe Hypertension or Heart Disease
- Liver or Kidney Disease
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology, Liu Hua Qiao Hospital
Guangzhou, Guangdong, 50010
Yang Xiao, MD, PhD, 86 1390 221 3175
Yang Gao, MD, 86 1355 618 0868
About this study
Official title: Phase I/II Study of An Ex Vivo Immunotherapy for Treatment of Idiopathic Aplastic Anemia
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation. Additionally, these cells travel and home to bone marrow as well as spleen and liver involved in hematopoietic activities, where direct cell-cell contact may be beneficial.
Read the detailed description
Patients will be required to stay in our hospitals to receive the immunotherapy. Allogeneic peripheral blood mononuclear cells from healthy donors and the autologous peripheral mononuclear cells will alternately used for the treatment. Patients are expected to spend at least 6 to 12 months in the hospital and won't be released until they have improved significantly.
- Conditions
- Anemia, Aplastic
- Being tested
- Ex Vivo Immunotherapy
- Keywords
- Idiopathic Aplastic Anemia, Has Failed Conventional Immunosuppressive Therapy, Not Eligible For Bone Marrow Transplantation, C15.378.071.085
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ex Vivo ImmunotherapyProcedure or surgery
Also called Aplastic Anemia, ex vivo Immunotherapy
Allogeneic and autologous ex vivo activated immune cells are given intravenously at least once a week
Ex vivo immunotherapyDrug
Also called Aplastic Anemia, ex vivo Immunotherapy
i.v. infusions, once a week, at least 6 to 12 months
| Study group | Role | What they receive |
|---|---|---|
| Hemathera | Experimental | Patients will receive cell-based immunotherapy. Receives: Procedure: Ex Vivo Immunotherapy; Drug: Ex vivo immunotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Survival
Measured: 2 years
Secondary outcomes (3)
Hematological Parameters
Measured: 2 years
Hematopoietic Activities in Bone Marrow
Measured: 2 years
Quality of Life
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2006 (estimated)
- Study startNov 2002
- Primary completionJun 2009 (estimated)
- Study completionDec 2009 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Anemia, AplasticStudies 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
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The 12th Guangzhou Municipal Hospital, Liu Hua Qiao Hospital
- Investigators
- Demao Yang, PhD, Study chair, Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00399971
- Sponsor's protocol ID
- AAFocus
- Status last verified
- Nov 2008
- First submitted
- 14 Nov 2006
- Last updated
- 11 Nov 2008
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Chen J, Liu W, Wang X, Chen H, Wu J, Yang Y, Wu L, Yang D. Ex vivo immunotherapy for patients with benzene-induced aplastic anemia. J Hematother Stem Cell Res. 2003 Oct;12(5):505-14. doi: 10.1089/152581603322448213.
PubMed 14594507Li G, Wang X, Wu L, Zhang W, Chen H, Xie Y, Yang D. Ex vivo activated immune cells promote survival and stimulate multilineage hematopoietic recovery in myelosuppressed mice. J Immunother. 2005 Jul-Aug;28(4):420-5. doi: 10.1097/01.cji.0000170360.99714.3f.
PubMed 16000962
Study at a glance
From the registry record, last updated by the sponsor on 11 Nov 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of An Ex Vivo Immunotherapy for Treatment of Idiopathic Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00399971
- Acronym / sponsor ID
- AAFocus
- Conditions
- Anemia, Aplastic
- Keywords
- Idiopathic Aplastic Anemia, Has Failed Conventional Immunosuppressive Therapy, Not Eligible For Bone Marrow Transplantation, C15.378.071.085
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Shenzhen Zhongxing Yangfan Biotech Co. Ltd.Industry (pharma, biotech, devices)
- Collaborators
- The 12th Guangzhou Municipal Hospital, Liu Hua Qiao Hospital
- Study start
- Nov 2002
- Primary completion
- Jun 2009 (estimated)
- Study completion
- Dec 2009 (estimated)
- First posted
- 15 Nov 2006 (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.
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation. Additionally, these cells travel and home to bone marrow as well as spleen and liver involved in hematopoietic activities, where direct cell-cell contact may be beneficial.
Detailed description
Patients will be required to stay in our hospitals to receive the immunotherapy. Allogeneic peripheral blood mononuclear cells from healthy donors and the autologous peripheral mononuclear cells will alternately used for the treatment. Patients are expected to spend at least 6 to 12 months in the hospital and won't be released until they have improved significantly.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemathera | Experimental | Procedure: Ex Vivo Immunotherapy, Drug: Ex vivo immunotherapyPatients will receive cell-based immunotherapy. |
Who may qualify
- Ages
- 10 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical Diagnosis of Idiopathic Aplastic Anemia
- Not Eligible for Bone Marrow Transplantation
- Not Responsive to Conventional Immunosuppressive Therapy
Key exclusion criteria (5 listed)
- Leukemia or MDS or PNH or Fanconi's Disease
- pregnancy
- Allergic to Blood Product
- Severe Hypertension or Heart Disease
- Liver or Kidney Disease
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 |
|---|---|---|
| Ex Vivo ImmunotherapyAlso: Aplastic Anemia, ex vivo ImmunotherapyAllogeneic and autologous ex vivo activated immune cells are given intravenously at least once a week | Procedure or surgery | Hemathera |
| Ex vivo immunotherapyAlso: Aplastic Anemia, ex vivo Immunotherapyi.v. infusions, once a week, at least 6 to 12 months | Drug | Hemathera |
Primary outcome
Survival
Time frame: 2 years
3 secondary outcomes
Hematological Parameters
Time frame: 2 years
Hematopoietic Activities in Bone Marrow
Time frame: 2 years
Quality of Life
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Nov 2008.
- Background: PubMed 14594507Chen J, Liu W, Wang X, Chen H, Wu J, Yang Y, Wu L, Yang D. Ex vivo immunotherapy for patients with benzene-induced aplastic anemia. J Hematother Stem Cell Res. 2003 Oct;12(5):505-14. doi: 10.1089/152581603322448213.
- Background: PubMed 16000962Li G, Wang X, Wu L, Zhang W, Chen H, Xie Y, Yang D. Ex vivo activated immune cells promote survival and stimulate multilineage hematopoietic recovery in myelosuppressed mice. J Immunother. 2005 Jul-Aug;28(4):420-5. doi: 10.1097/01.cji.0000170360.99714.3f.
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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.
Demao Yang, PhD study chair
Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
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 Ex Vivo Immunotherapy in people with Anemia, Aplastic, run by Shenzhen Zhongxing Yangfan Biotech 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 3 inclusion and 5 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 11 Nov 2008. 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 Demao Yang, PhD (86 755 2603 1580, dyang@yangfan-biotech.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survival (time frame: 2 years). 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 NCT00399971, which also lists 2 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 11 Nov 2008.