Fanconi AnemiaPhase 2FANCT01071239
Hematopoietic Stem Cell Transplant for Fanconi Anemia
A Phase 2 study testing CliniMACs device, Busulfan and Fludarabine in people with Fanconi Anemia, run by Medical College of Wisconsin at 1 site in United States.
Trial summary
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- CliniMACs device, Busulfan and Fludarabine
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2009to 30 Aug 2016
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia
- Karnofsky or Lansy performance scale > or = to 70%.
- Must have adequate cardiac, hepatic, renal and pulmonary function.
- Must have 7/8 or 8/8 available unrelated donor.
Who cannot join
- Pregnant or breastfeeding.
- Active CNS leukemic involvement
- Active uncontrolled viral, bacterial or fungal infection
- Positive for HIV.
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
About this study
Official title: A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
Read the detailed description
We are currently recruiting patients.
- Conditions
- Fanconi Anemia
- Being tested
- CliniMACs device, Busulfan, Fludarabine, Cyclophosphamide, ATG
- Keywords
- Fanconi Anemia, Hematopoietic stem cell transplant
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CliniMACs deviceDevice
Also called Milteny Biotec CliniMACS device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device
BusulfanDrug
Chemotherapy administered as a part of the HSCT conditioning regimen.
FludarabineDrug
Chemotherapy administered as a part of the HSCT conditioning regimen.
CyclophosphamideDrug
Chemotherapy administered as a part of the HSCT conditioning regimen.
ATGDrug
Chemotherapy administered as a part of the HSCT conditioning regimen.
| Study group | Role | What they receive |
|---|---|---|
| Bone marrow processing | Experimental | Bone Marrow processing using the CliniMACs device Receives: Device: CliniMACs device; Drug: Busulfan; Drug: Fludarabine; Drug: Cyclophosphamide; Drug: ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Measured: 1, 3, 6 and 12 months post transplant date
Secondary outcomes (1)
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
Measured: weekly for the first 30 days and then 3, 6, and 12 months post transplant date
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2010 (estimated)
- Study startApr 2009
- Primary completion30 Aug 2016
- Study completion30 Aug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Medical College of WisconsinThe 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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical College of Wisconsin
- Sponsor type
- University, hospital or other
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Responsible party
- Principal investigator, David A. Margolis, Medical College of Wisconsin
- Investigators
- David A Margolis, MD, Principal investigator, Medical College of Wisconsin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01071239
- Sponsor's protocol ID
- FA 08/89
- Acronym
- FA
- Status last verified
- Sep 2019
- First submitted
- 20 May 2009
- Last updated
- 11 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
- ClinicalTrials.gov ID
- NCT01071239
- Acronym / sponsor ID
- FA / FA 08/89
- Conditions
- Fanconi Anemia
- Keywords
- Fanconi Anemia, Hematopoietic stem cell transplant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Medical College of WisconsinUniversity, hospital or other
- Collaborators
- Memorial Sloan Kettering Cancer Center
- Study start
- Apr 2009
- Primary completion
- 30 Aug 2016 (actual)
- Study completion
- 30 Aug 2016 (actual)
- First posted
- 19 Feb 2010 (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 trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
Detailed description
We are currently recruiting patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bone marrow processing | Experimental | Device: CliniMACs device, Drug: Busulfan, Drug: Fludarabine, Drug: Cyclophosphamide, Drug: ATGBone Marrow processing using the CliniMACs device |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia
- Karnofsky or Lansy performance scale > or = to 70%.
- Must have adequate cardiac, hepatic, renal and pulmonary function.
- Must have 7/8 or 8/8 available unrelated donor.
Key exclusion criteria (4 listed)
- Pregnant or breastfeeding.
- Active CNS leukemic involvement
- Active uncontrolled viral, bacterial or fungal infection
- Positive for HIV.
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 |
|---|---|---|
| CliniMACs deviceAlso: Milteny Biotec CliniMACS deviceDonor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device | Device | Bone marrow processing |
| BusulfanChemotherapy administered as a part of the HSCT conditioning regimen. | Drug | Bone marrow processing |
| FludarabineChemotherapy administered as a part of the HSCT conditioning regimen. | Drug | Bone marrow processing |
| CyclophosphamideChemotherapy administered as a part of the HSCT conditioning regimen. | Drug | Bone marrow processing |
| ATGChemotherapy administered as a part of the HSCT conditioning regimen. | Drug | Bone marrow processing |
Primary outcome
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Time frame: 1, 3, 6 and 12 months post transplant date
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
1 secondary outcome
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
Time frame: weekly for the first 30 days and then 3, 6, and 12 months post transplant date
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 11 Sep 2019.
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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.
David A Margolis, MD principal investigator
Medical College of Wisconsin
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 CliniMACs device, Busulfan and Fludarabine in people with Fanconi Anemia, run by Medical College of Wisconsin 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 4 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: finished, as of the sponsor's last update on 11 Sep 2019. 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 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?
To measure the incidence and quality of engraftment and hematopoietic reconstitution. (time frame: 1, 3, 6 and 12 months post transplant date). 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 11 Sep 2019.