Sickle Cell AnemiaPhase 1/2NCT00004485
Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
A Phase 1/2 study testing cyclosporine, fludarabine and mycophenolate mofetil in people with Sickle Cell Anemia, run by Fred Hutchinson Cancer Center at 2 sites in United States.
Trial summary
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Read more about this study
- Who is it for?
- Ages Up to 16 years
- Conditions the study is for
- Sickle Cell Anemia
- What is being tested?
- cyclosporine, fludarabine and mycophenolate mofetil
- Type of treatment
- Surgery or procedure
- How many people
- 50 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 1999to Aug 2007
- Phase
- Phase 1/2
- 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 Up to 16 years
- All sexes
- No healthy volunteers
- Child
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by: Recurrent painful events (at least 2 painful events in past year) which cannot be explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2 years that required hospitalization, oxygen, and RBC transfusion Any combination of painful events and ACS episodes that total 2 events within the past year Abnormal cerebral MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing performance
No stage III or IV sickle cell lung disease
Genotypically HLA identical sibling donor available
--Prior/Concurrent Therapy--
No prior transfusions with greater than 5 units RBC
--Patient Characteristics--
Performance status: Karnofsky 70-100%
Hepatic:
- No active hepatitis
- No moderate/severe portal fibrosis
Renal: Glomerular filtration rate at least 30% predicted for age
Neurologic:
- No severe residual functional neurologic impairment
- Hemiplegia alone allowed
Other:
- HIV negative
- Not pregnant or nursing
- Fertile patients must use effective contraception
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Hospital of Oakland
Oakland, California, 94609
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
About this study
Official title: Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.
PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.
Read the detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.
Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.
- Conditions
- Sickle Cell Anemia
- Being tested
- cyclosporine, fludarabine, mycophenolate mofetil, Bone Marrow Transplantation
- Keywords
- genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, sickle cell anemia
- Medical subject headings
- Anemia, Sickle Cell, Genetic Diseases, Inborn, Hematologic Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclosporineDrug
fludarabineDrug
Bone Marrow TransplantationProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startDec 1999
- Primary completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe 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 1/2
- Primary purpose
- Treatment
- Enrolment
- 50 participants
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Investigators
- Mark Walters, Study chair, Children's Hospital of Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004485
- Sponsor's protocol ID
- 199/14243
- Other IDs
- FHCRC-1373.00
- Status last verified
- Sep 2008
- First submitted
- 18 Oct 1999
- Last updated
- 10 Sep 2008
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT00004485
- Acronym / sponsor ID
- 199/14243
- Conditions
- Sickle Cell Anemia
- Keywords
- genetic diseases and dysmorphic syndromes, hematologic disorders, rare disease, sickle cell anemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Study start
- Dec 1999
- Primary completion
- Aug 2007 (actual)
- First posted
- 19 Oct 1999 (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.
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.
PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.
Detailed description
PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.
Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| cyclosporine | Drug | – |
| fludarabine | Drug | – |
| mycophenolate mofetil | Drug | – |
| Bone Marrow Transplantation | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
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 10 Sep 2008.
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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.
Mark Walters study chair
Children's Hospital of Oakland
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing cyclosporine, fludarabine and mycophenolate mofetil in people with Sickle Cell Anemia, run by Fred Hutchinson Cancer Center at 2 sites 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?
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 10 Sep 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 combined 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.
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.
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 10 Sep 2008.