Severe Aplastic AnemiaPhase 2NCT01383434
Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia
A Phase 2 study testing Bone Marrow Transplant in people with Severe Aplastic Anemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. Read more about this study
- Who is it for?
- Ages 6 months to 70 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Bone Marrow Transplant
- Type of treatment
- Surgery or procedure
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2011to Aug 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 6 months to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens.
- Age 6 months - 70 years
- Patients must meet medical criteria for myeloablative BMT
- Patients or their parents/guardians and donors must be able to sign consent forms.
- Patients must be geographically accessible and willing to participate in all stages of treatment.
Who cannot join
- Poor cardiac function: left ventricular ejection fraction <45% as determined by MUGA or ECHO.
- Poor pulmonary function: FEV1 and FVC <50% predicted.
- Poor renal function
- Positive leukocytotoxic crossmatch
- Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception
- Uncontrolled viral, bacterial, or fungal infections
Trial locations and contacts
1 site in 1 country.
United States1 site
The Sidney Kimmel Comprehensive Cancer Center
Baltimore, Maryland, 21031
About this study
Official title: A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients.
Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure.
This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Bone Marrow Transplant
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone Marrow TransplantProcedure or surgery
Matched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy
What is being measured
The primary outcome is the main question the study is designed to answer.
To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
Measured: 1 year
To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2011 (estimated)
- Study startJun 2011
- Primary completionAug 2014
- Study completionAug 2014
Why it stopped: New revised SAA BMT study opened to take over this study
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.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Robert A Brodsky, MD, Principal investigator, The Johns Hopkins UniversityAmy Dezern, MD, Principal investigator, The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01383434
- Sponsor's protocol ID
- J10117
- Other IDs
- NA_00042991
- Status last verified
- Oct 2016
- First submitted
- 22 Jun 2011
- Last updated
- 13 Oct 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT01383434
- Acronym / sponsor ID
- J10117
- Conditions
- Severe Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: New revised SAA BMT study opened to take over this study
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Study start
- Jun 2011
- Primary completion
- Aug 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 28 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.
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients.
Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure.
This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 6 months to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens.
- Age 6 months - 70 years
- Patients must meet medical criteria for myeloablative BMT
- Patients or their parents/guardians and donors must be able to sign consent forms.
- Patients must be geographically accessible and willing to participate in all stages of treatment.
Key exclusion criteria (6 listed)
- Poor cardiac function: left ventricular ejection fraction <45% as determined by MUGA or ECHO.
- Poor pulmonary function: FEV1 and FVC <50% predicted.
- Poor renal function
- Positive leukocytotoxic crossmatch
- Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception
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 |
|---|---|---|
| Bone Marrow TransplantMatched sibling, haploidentical or matched unrelated donor bone marrow transplant following chemotherapy | Procedure or surgery | – |
Primary outcome
To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
Time frame: 1 year
To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
Time frame: 1 year
Results and safety
The study was stopped early (reason given: New revised SAA BMT study opened to take over this study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2016.
Related trials for Severe Aplastic Anemia
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes, Aplastic Anemias
Active, not recruitingPhase 2
Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
Aplastic Anemia, TPO Receptor Agonists
RecruitingObservational study
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Severe Aplastic Anemia, Telomere Biology Disorders
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
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.
Robert A Brodsky, MD principal investigator
The Johns Hopkins University
Amy Dezern, MD principal investigator
The JOhns Hopkins Sydney Kimmel Comprehensive Cancer Center
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 Bone Marrow Transplant in people with Severe Aplastic Anemia, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 5 inclusion and 6 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: stopped early, as of the sponsor's last update on 13 Oct 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 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.
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?
The study was stopped early (reason given: New revised SAA BMT study opened to take over 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?
To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia (time frame: 1 year). 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?
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 13 Oct 2016.