Ignite Creation Date:
2024-05-05 @ 4:47 PM
Last Modification Date:
2024-10-26 @ 9:24 AM
Study NCT ID:
NCT00319878
Status:
UNKNOWN
Last Update Posted:
2008-10-07
First Post:
2006-04-28
Brief Title:
Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
Sponsor:
Office of Rare Diseases ORD
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
A Phase III Trial of Sirolimus Rapamune and Cyclosporine in Patients With Refractory Aplastic Anemia
Status:
UNKNOWN
Status Verified Date:
2008-10
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Aplastic anemia is a rare autoimmune disorder in which the bone marrow production of blood cells is greatly decreased or absent Symptoms include fatigue weakness tiny reddish-purple marks on the skin abnormal bruising and bleeding from the gums nose or intestine While some cases of aplastic anemia are caused by medications toxic exposures or inherited genes most often the cause remains unknown The purpose of this study is to determine the safety and efficacy of combining two drugs sirolimus and cyclosporine for treating individuals with aplastic anemia that has not responded to other treatments
Detailed Description:
The most successful treatment for aplastic anemia is bone marrow transplantation However few patients are eligible for this procedure For others treatment usually consists of immunosuppressive agents such as antithymocyte globulin ATG and cyclosporine Unfortunately even with immunosuppressive therapy relapse is common New combinations of medications may offer alternative and more effective treatment options Sirolimus and cyclosporine are two drugs routinely used to suppress the immune system and prevent rejection in patients who have received organ transplants While cyclosporine has been proven effective for treating aplastic anemia sirolimus has not been tested for this disease This study will evaluate the safety and efficacy of sirolimus in combination with cyclosporine for treating individuals with aplastic anemia that has not responded to other treatments
This study will last at least 6 months Participants will first be screened to verify diagnosis of aplastic anemia The screening will include a physical examination blood test bone marrow biopsy from the pelvic bone and review of medications and medical history Individuals who are eligible will then start the first treatment period Participants will receive two medications cyclosporine will be taken twice a day and sirolimus will be taken once a day Depending on side effects the doses of either drug may be temporarily stopped or lowered On Day 1 blood will be drawn and females will undergo a pregnancy test Subsequent study visits will occur weekly for the first month every 2 weeks for 2 months and then once a month for the remainder of the study Each visit will include a physical examination vital sign assessment and review of side effects and medications Blood tests will be performed weekly for the first 3 weeks and then every 2 weeks
After 6 months of treatment if a participant has shown improvements in disease status without major side effects the treatment will continue Over time the doses may be lowered If a participant has not improved while on the study medication treatment will stop at 6 months Whenever treatment is discontinued the participant will again undergo a physical examination blood tests and bone marrow biopsy
This study will last at least 6 months Participants will first be screened to verify diagnosis of aplastic anemia The screening will include a physical examination blood test bone marrow biopsy from the pelvic bone and review of medications and medical history Individuals who are eligible will then start the first treatment period Participants will receive two medications cyclosporine will be taken twice a day and sirolimus will be taken once a day Depending on side effects the doses of either drug may be temporarily stopped or lowered On Day 1 blood will be drawn and females will undergo a pregnancy test Subsequent study visits will occur weekly for the first month every 2 weeks for 2 months and then once a month for the remainder of the study Each visit will include a physical examination vital sign assessment and review of side effects and medications Blood tests will be performed weekly for the first 3 weeks and then every 2 weeks
After 6 months of treatment if a participant has shown improvements in disease status without major side effects the treatment will continue Over time the doses may be lowered If a participant has not improved while on the study medication treatment will stop at 6 months Whenever treatment is discontinued the participant will again undergo a physical examination blood tests and bone marrow biopsy
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| BMF 5403 | US NIH GrantContract | None | httpsreporternihgovquickSearchU54RR019397-01 |
| U54RR019397-01 | NIH | None | None |