Severe Aplastic AnemiaNCT01364363
Unrelated Donor Stem Cell Transplantation
A study testing Allogeneic transplantation in people with Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by Scripps Health at 1 site in United States.
Trial summary
The purpose of this study is to provide an opportunity for patients with malignancies or bone marrow failure states who lack a suitable sibling donor to undergo allogeneic hematopoietic progenitor cell transplantation using cells from unrelated individuals or cord blood registries. Read more about this study
- Who is it for?
- Ages 18 to 69 years
- Conditions the study is for
- Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia
- What is being tested?
- Allogeneic transplantation
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2005to Jul 2015
- Phase
- Interventional
- 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 69 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- > 18 years of Age
- < 70 years
- ECOG performance status 0, 1 or 2
- Left Ventricular Ejection Fraction > 30%
- Creatinine clearance > 40ml/min
- Transaminases < 2X normal
Show all 11Show fewer
- Total bilirubin < 2X normal
- HIV seronegativity
- Weight < 70kg for cord blood transplantation
- Ability to cover the cost of the transplant, necessary medications, and transportation/housing.
- Caregiver must be available while outpatient
Who cannot join
- Greater than one antigen mismatch at HLA-A, B, C or DR plus one allele mismatch at HLA-A, B, C or DR
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Allogeneic Hematopoietic Progenitor Cell Transplantation From Unrelated Donors
The purpose of this study is to provide an opportunity for patients with malignancies or bone marrow failure states who lack a suitable sibling donor to undergo allogeneic hematopoietic progenitor cell transplantation using cells from unrelated individuals or cord blood registries.
Read the detailed description
same
- Conditions
- Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndromes
- Being tested
- Allogeneic transplantation
- Keywords
- Transplant, Allogeneic, Allogeneic transplant, Unrelated donor transplant, MUD, HSCT, Stem cell transplant
- Medical subject headings
- Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Hodgkin Disease, Lymphoma, Non-Hodgkin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic transplantationProcedure or surgery
Also called MUD transplant
Allogeneic transplantation with matched unrelated donors
| Study group | Role | What they receive |
|---|---|---|
| Total Body Irradiation/VP16 | Other | Acute Leukemias, Myelodysplastic syndromes Receives: Procedure: Allogeneic transplantation |
| Cytoxan/Total Body Irradiation | Other | Chronic Leukemias, Bone Marrow Failure States, Lymphomas, Hodgkin's Disease Receives: Procedure: Allogeneic transplantation |
| Busulfan/Cytoxan | Other | Acute Leukemia, Myelodysplastic syndromes, Chronic Leukemias, Bone Marrow Failure states Receives: Procedure: Allogeneic transplantation |
| BEAM (BCNU, etoposide, Ara-C, melphalan) | Other | Lymphomas, Hodgkin's Disease Receives: Procedure: Allogeneic transplantation |
| Total Lymphoid Irradiation | Other | For patients with Multiple Myeloma, or prior autologous transplantation, or age in excess of 55 Receives: Procedure: Allogeneic transplantation |
| Cladribine/Melphalan | Other | For patients with Multiple Myeloma, or prior autologous transplantation, or age in excess of 55 Receives: Procedure: Allogeneic transplantation |
| FLAG (fludarabine, Ara-C, G-CSF) | Other | For patients undergoing a second allogeneic transplant Receives: Procedure: Allogeneic transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone Marrow and Peripheral Blood Chimerism
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Measured: 30 days post-transplant
Bone Marrow and Peripheral Blood Chimerism
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Measured: 100 days post-transplant
Bone Marrow and Peripheral Blood Chimerism
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Measured: 365 days post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2011 (estimated)
- Study startMar 2005
- Primary completionJul 2015
- Study completionJul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scripps Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeffrey W. Andrey, MD, Principal investigator, Scripps Clinic Medical Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01364363
- Sponsor's protocol ID
- SIRB#13-6190
- Status last verified
- Nov 2022
- First submitted
- 4 Apr 2011
- Last updated
- 23 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Progenitor Cell Transplantation From Unrelated Donors
- ClinicalTrials.gov ID
- NCT01364363
- Acronym / sponsor ID
- SIRB#13-6190
- Conditions
- Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndromes
- Keywords
- Transplant, Allogeneic, Allogeneic transplant, Unrelated donor transplant, MUD, HSCT, Stem cell transplant
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Scripps HealthUniversity, hospital or other
- Study start
- Mar 2005
- Primary completion
- Jul 2015 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 2 Jun 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 purpose of this study is to provide an opportunity for patients with malignancies or bone marrow failure states who lack a suitable sibling donor to undergo allogeneic hematopoietic progenitor cell transplantation using cells from unrelated individuals or cord blood registries.
Detailed description
same
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Total Body Irradiation/VP16 | Other | Procedure: Allogeneic transplantationAcute Leukemias, Myelodysplastic syndromes |
| Cytoxan/Total Body Irradiation | Other | Procedure: Allogeneic transplantationChronic Leukemias, Bone Marrow Failure States, Lymphomas, Hodgkin's Disease |
| Busulfan/Cytoxan | Other | Procedure: Allogeneic transplantationAcute Leukemia, Myelodysplastic syndromes, Chronic Leukemias, Bone Marrow Failure states |
| BEAM (BCNU, etoposide, Ara-C, melphalan) | Other | Procedure: Allogeneic transplantationLymphomas, Hodgkin's Disease |
| Total Lymphoid Irradiation | Other | Procedure: Allogeneic transplantationFor patients with Multiple Myeloma, or prior autologous transplantation, or age in excess of 55 |
| Cladribine/Melphalan | Other | Procedure: Allogeneic transplantationFor patients with Multiple Myeloma, or prior autologous transplantation, or age in excess of 55 |
| FLAG (fludarabine, Ara-C, G-CSF) | Other | Procedure: Allogeneic transplantationFor patients undergoing a second allogeneic transplant |
Who may qualify
- Ages
- 18 to 69 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- > 18 years of Age
- < 70 years
- ECOG performance status 0, 1 or 2
- Left Ventricular Ejection Fraction > 30%
- Creatinine clearance > 40ml/min
Key exclusion criteria (1 listed)
- Greater than one antigen mismatch at HLA-A, B, C or DR plus one allele mismatch at HLA-A, B, C or DR
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 |
|---|---|---|
| Allogeneic transplantationAlso: MUD transplantAllogeneic transplantation with matched unrelated donors | Procedure or surgery | BEAM (BCNU, etoposide, Ara-C, melphalan), Busulfan/Cytoxan, Cladribine/Melphalan, Cytoxan/Total Body Irradiation, FLAG (fludarabine, Ara-C, G-CSF), Total Body Irradiation/VP16, Total Lymphoid Irradiation |
Primary outcome
Bone Marrow and Peripheral Blood Chimerism
Time frame: 30 days post-transplant
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Bone Marrow and Peripheral Blood Chimerism
Time frame: 100 days post-transplant
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Bone Marrow and Peripheral Blood Chimerism
Time frame: 365 days post-transplant
Percent of donor and host cells in peripheral blood and bone marrow will be measured by short tandem repeat (STR) testing and/or standard cytogenetic testing.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2022.
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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.
Jeffrey W. Andrey, MD principal investigator
Scripps Clinic Medical Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Allogeneic transplantation in people with Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by Scripps Health at 1 site in United States.
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 11 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: finished, as of the sponsor's last update on 23 Nov 2022. 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 United States. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Bone Marrow and Peripheral Blood Chimerism (time frame: 30 days post-transplant). The study lists 3 primary outcomes in all. 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 23 Nov 2022.