ANTIPHOSPHOLIPID SYNDROMEPhase 1NCT00278616
Hematopoietic Stem Cell Transplantation in Patients With Antiphospholipid Syndrome
A Phase 1 study testing Stem Cell Transplantation in people with ANTIPHOSPHOLIPID SYNDROME, run by Richard Burt, MD at 1 site in United States.
Trial summary
Antiphospholipid syndrome is disease believed to be due to immune cells, cells which normally protect the body, but are now producing the protein which leads to abnormal clotting in the body. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- ANTIPHOSPHOLIPID SYNDROME
- What is being tested?
- Stem Cell Transplantation
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2005to Oct 2011
- 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 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age > 18 years< 55 years at the time of pretransplant evaluation
- A or 6.12.B:
- An established diagnosis of a definite primary APS by Sapporo criteria as follows:
- Positive LA and/or ACLA IgG or IgM on two separate measurements, AND
- Arterial, venous or small vessel thrombosis (confirmed by imaging or doppler studies or histopathology, with the exception of superficial venous thrombosis) OR pregnancy morbidity (defined as three or more embryonic losses, OR one or more premature birth due to preeclampsia or growth retardation, OR one or more fetal death)
- APLA-positive Sneddon syndrome defined as an association of ischemic cerebrovascular events and a widespread livedo reticularis
- 3. Patients failed treatment with anticoagulation including warfarin, heparin/LMWH in the presence of positive LA and/or ACLA IgG, or IgM. Failure is defined as any of above described thromboembolic events (6.12.A-2 or 6.12.B) except for pregnancy morbidity while receiving therapeutic anticoagulation. Therapeutic anticoagulation is defined as at least 5000 U of regular heparin SQ BID, OR unfractionated IV heparin adjusted for therapeutic PTT, OR at least 40 mg of lovenox SQ QD (or equivalent LMWH), OR coumadin adjusted for INR of at least 2.0, documented within 1 month of a refractory event or within 3 months if patient was known to be previously stable PLUS in the opinion of the investigator the individual has been receiving adequate anticoagulation.
Who cannot join
- Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is due to the disease itself and considered to be reversible.
- Significant end organ damage such as (not caused by APS):
- LVEF<40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
- Untreated life-threatening arrhythmia.
- Active ischemic heart disease or heart failure.
- DLCO<40% or FEV1/FEV < 50%.
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- Serum creatinine >2.5 or creatinine clearance <30ml/min.
- Liver cirrhosis, transaminases >3x of normal limits or bilirubin >2.0 unless due to Gilbert disease.
- 3. HIV positive.
- 4.Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
- 5. Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
- 6. Positive pregnancy test, inability or unable to pursue effective means of birth control, failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
- 7. Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
- 8. Inability to give informed consent.
- 9. Major hematological abnormalities such as platelet count less than 100,000/ul, ANC less than 1000/ul unless due to APS.
- 10. Failure to collect at least 2.0 x 106 CD34+ / kg cells.
- 11. Patients who are already in a clinical trial for APS treatment
Trial locations and contacts
1 site in 1 country.
United States1 site
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, 60611
About this study
Official title: High Dose Cyclophosphamide & CAMPATH-1H With Hematopoietic Stem Cell Transplantation in Patients With Refractory Antiphospholipid Syndrome (APS): A Phase I Trial
Antiphospholipid syndrome is disease believed to be due to immune cells, cells which normally protect the body, but are now producing the protein which leads to abnormal clotting in the body. This study is designed to examine whether treating patients with high dose cyclophosphamide together with CAMPATH (drugs which reduce the function of the immune system), followed by return of the previously collected stem cells will result in improvement in the disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in the immune system which may be causing the disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack the body. The study purpose is to examine whether this treatment will result in improvement in the disease. The drugs used in this study treatment are drugs for commonly used for immune suppression.
- Conditions
- ANTIPHOSPHOLIPID SYNDROME
- Being tested
- Stem Cell Transplantation
- Medical subject headings
- Antiphospholipid Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantationBiological
Autologous Stem Cell Transplantation
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity; Survival;Disease improvement;Time to disease progression;
Measured: 5 years after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2006 (estimated)
- Study startAug 2005
- Primary completionOct 2011
- Study completionOct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ANTIPHOSPHOLIPID SYNDROMEStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Richard Burt, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Richard Burt, MD, Northwestern University
- Investigators
- Richard Burt, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00278616
- Sponsor's protocol ID
- NU APS AUTO 2004
- Status last verified
- Apr 2012
- First submitted
- 16 Jan 2006
- Last updated
- 11 Apr 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Statkute L, Traynor A, Oyama Y, Yaung K, Verda L, Krosnjar N, Burt RK. Antiphospholipid syndrome in patients with systemic lupus erythematosus treated by autologous hematopoietic stem cell transplantation. Blood. 2005 Oct 15;106(8):2700-9. doi: 10.1182/blood-2005-01-0330. Epub 2005 May 3.
PubMed 15870182
Study at a glance
From the registry record, last updated by the sponsor on 11 Apr 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High Dose Cyclophosphamide & CAMPATH-1H With Hematopoietic Stem Cell Transplantation in Patients With Refractory Antiphospholipid Syndrome (APS): A Phase I Trial
- ClinicalTrials.gov ID
- NCT00278616
- Acronym / sponsor ID
- NU APS AUTO 2004
- Conditions
- ANTIPHOSPHOLIPID SYNDROME
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Richard Burt, MDUniversity, hospital or other
- Study start
- Aug 2005
- Primary completion
- Oct 2011 (actual)
- Study completion
- Oct 2011 (actual)
- First posted
- 18 Jan 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.
Antiphospholipid syndrome is disease believed to be due to immune cells, cells which normally protect the body, but are now producing the protein which leads to abnormal clotting in the body. This study is designed to examine whether treating patients with high dose cyclophosphamide together with CAMPATH (drugs which reduce the function of the immune system), followed by return of the previously collected stem cells will result in improvement in the disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in the immune system which may be causing the disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack the body. The study purpose is to examine whether this treatment will result in improvement in the disease. The drugs used in this study treatment are drugs for commonly used for immune suppression.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Age > 18 years< 55 years at the time of pretransplant evaluation
- A or 6.12.B:
- An established diagnosis of a definite primary APS by Sapporo criteria as follows:
- Positive LA and/or ACLA IgG or IgM on two separate measurements, AND
- Arterial, venous or small vessel thrombosis (confirmed by imaging or doppler studies or histopathology, with the exception of superficial venous thrombosis) OR pregnancy morbidity (defined as three or more embryonic losses, OR one or more premature birth due to preeclampsia or growth retardation, OR one or more fetal death)
Key exclusion criteria (17 listed)
- Poor performance (PS) status (ECOG >2) at the time of entry, unless decline of PS is due to the disease itself and considered to be reversible.
- Significant end organ damage such as (not caused by APS):
- 3. HIV positive.
- 4.Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
- 5. Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
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 |
|---|---|---|
| Stem Cell TransplantationAutologous Stem Cell Transplantation | Biological | – |
Primary outcome
Toxicity; Survival;Disease improvement;Time to disease progression;
Time frame: 5 years after transplant
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Apr 2012.
- Background: PubMed 15870182Statkute L, Traynor A, Oyama Y, Yaung K, Verda L, Krosnjar N, Burt RK. Antiphospholipid syndrome in patients with systemic lupus erythematosus treated by autologous hematopoietic stem cell transplantation. Blood. 2005 Oct 15;106(8):2700-9. doi: 10.1182/blood-2005-01-0330. Epub 2005 May 3.
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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.
Richard Burt, MD principal investigator
Northwestern University
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 Stem Cell Transplantation in people with ANTIPHOSPHOLIPID SYNDROME, run by Richard Burt, MD 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 7 inclusion and 17 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: withdrawn, as of the sponsor's last update on 11 Apr 2012. 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 was withdrawn before enrolling participants, so there are no results.
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?
Toxicity; Survival;Disease improvement;Time to disease progression; (time frame: 5 years after transplant). 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 NCT00278616, which also lists 1 linked publication.
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 Apr 2012.