Anemia, AplasticPhase 1NCT00578266
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
A Phase 1 study testing Cyclophosphamide,Campath IH and TBI in people with Anemia, Aplastic, run by Mayo Clinic at 1 site in United States.
Trial summary
For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched… Read more about this study
- Who is it for?
- Ages Up to 60 years
- Conditions the study is for
- Anemia, Aplastic
- What is being tested?
- Cyclophosphamide,Campath IH and TBI
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2007to 16 Aug 2016
- Phase
- Phase 1
- 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 Up to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
- A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 matched related donor available after high resolution typing.
Who cannot join
- Patients with Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
- HIV seropositive patients
- Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
- Patient greater than 60 years of age.
- Women who are pregnant or nursing.
- Patients with active hepatitis
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- Patients with severe cardiac dysfunction defined as shortening fraction <25%.
- Patients with severe renal dysfunction defined as creatinine clearance <40ml/mim/1.73m2.
- Patient with severe pulmonary dysfunction with FEV1, FVC and DLCO 40% of predicted or 3 SD below normal.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :
- Patient survival probability at 100 days, 1 year and 2 years.
- Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
- Engraftment at 6 months, 1 year and 2 years
Read the detailed description
The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results. For this initial study we plan to enroll up to 24 patients.
- Conditions
- Anemia, Aplastic
- Being tested
- Cyclophosphamide,Campath IH and TBI
- Keywords
- Severe Aplastic Anemia (SAA), Transplant for Severe Aplastic Anemia (SAA)
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphamide,Campath IH and TBIDrug
DAY 5 TREATMENT
- 6 5 CYTOXAN 50 mg/kg WITH MESNA
- 5 CYTOXAN 50 mg/kg WITH MESNA;
- 4 CYTOXAN 50 mg/kgWITH MESNA; CAMPATH 3-10 mg
- 3 CYTOXAN 50 mg/kg WITH MESNA; CAMPATH;
- 2 TBI; CAMPATH; TACROLIMUS
- 1 TBI (second fraction); CAMPATH (am) 0 STEM CELL INFUSION (pm)
| Study group | Role | What they receive |
|---|---|---|
| No Arms | Other | Not described Receives: Drug: Cyclophosphamide,Campath IH and TBI |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years
Measured: 2 years
Secondary outcomes (1)
Patient survival probability at 100 days, 1 year and 2 years.
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 posted21 Dec 2007 (estimated)
- Study startFeb 2007
- Primary completion16 Aug 2016
- Study completion16 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, AplasticStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shakila Khan, Mayo Clinic
- Investigators
- Shakila P. Khan, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00578266
- Sponsor's protocol ID
- 06-006216
- Status last verified
- Mar 2017
- First submitted
- 17 Dec 2007
- Last updated
- 24 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
- ClinicalTrials.gov ID
- NCT00578266
- Acronym / sponsor ID
- 06-006216
- Conditions
- Anemia, Aplastic
- Keywords
- Severe Aplastic Anemia (SAA), Transplant for Severe Aplastic Anemia (SAA)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Feb 2007
- Primary completion
- 16 Aug 2016 (actual)
- Study completion
- 16 Aug 2016 (actual)
- First posted
- 21 Dec 2007 (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.
For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :
- Patient survival probability at 100 days, 1 year and 2 years.
- Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
- Engraftment at 6 months, 1 year and 2 years
Detailed description
The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results. For this initial study we plan to enroll up to 24 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| No Arms | Other | Drug: Cyclophosphamide,Campath IH and TBI |
Who may qualify
- Ages
- Up to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
- A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 matched related donor available after high resolution typing.
Key exclusion criteria (9 listed)
- Patients with Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
- HIV seropositive patients
- Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
- Patient greater than 60 years of age.
- Women who are pregnant or nursing.
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 |
|---|---|---|
| Cyclophosphamide,Campath IH and TBIDAY 5 TREATMENT * 6 5 CYTOXAN 50 mg/kg WITH MESNA * 5 CYTOXAN 50 mg/kg WITH MESNA; * 4 CYTOXAN 50 mg/kgWITH MESNA; CAMPATH 3-10 mg * 3 CYTOXAN 50 mg/kg WITH MESNA; CAMPATH; * 2 TBI; CAMPATH; TACROLIMUS * 1 TBI (second fraction); CAMPATH (am) 0 STEM CELL INFUSION (pm) | Drug | No Arms |
Primary outcome
1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years
Time frame: 2 years
1 secondary outcome
Patient survival probability at 100 days, 1 year and 2 years.
Time frame: 2 years
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 24 Mar 2017.
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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.
Shakila P. Khan, M.D. principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cyclophosphamide,Campath IH and TBI in people with Anemia, Aplastic, run by Mayo Clinic 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 2 inclusion and 9 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 24 Mar 2017. 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 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.
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 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?
1. Engraftment at 6 months, 1 year and 2 years 2.Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years (time frame: 2 years). 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 24 Mar 2017.