Ignite Creation Date:
2024-05-05 @ 12:07 PM
Last Modification Date:
2024-10-26 @ 9:20 AM
Study NCT ID:
NCT00243399
Status:
COMPLETED
Last Update Posted:
2011-07-15
First Post:
2005-10-20
Brief Title:
Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia
Sponsor:
Childrens Hospital Medical Center Cincinnati
Organization:
Childrens Hospital Medical Center Cincinnati
Study Overview
Official Title:
A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia
Status:
COMPLETED
Status Verified Date:
2011-07
Last Known Status:
None
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:
The purpose of this study is to evaluate the safety of the drug oxandrolone a type of androgen steroid in patients with Fanconi anemia FA and to determine if this drug can help in the treatment of bone marrow failure in these patients Androgen steroids are male hormones that can stimulate the production of red blood cells the cells which carry oxygen in the blood and platelets cells that help blood clot
Detailed Description:
The primary purpose of this study is to evaluate the safety of the drug oxandrolone in patients with Fanconi anemia FA and secondarily to determine if this drug can help in the treatment of bone marrow failure in these patients It is hoped that oxandrolone will have less side effects than oxymetholone the androgen used most frequently in the short-term treatment of bone marrow failure in FA patients Subjects will be enrolled for approximately 18 to 30 months 12 - 24 months of treatment and 6 months additional monitoring The oxandrolone starting dose is 004mgkgday Study monitoring includes weekly complete blood counts monthly serum chemistry labs quarterly physical examinations including virilization exams and liver ultrasounds Semi-annually hand radiographs are obtained for bone maturation and behavioral assessments are conducted to detect any aggressive behavior or mood changes If no improvement n the subjects blood counts are noted after 4 months of therapy the dose will be increased to 008mgkgday for a period of 4 more months If no improvement is noted after a total of eight months oxandrolone will be discontinued If the blood counts show improvement then the drug will continue for a minimum of twelve months Subjects may remain on study and receive a total of 24 months of therapy if they have a response in their blood counts without unacceptable side effects Post-treatment monitoring includes blood work and ultrasound every three months and hand radiograph at six months
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 |
|---|---|---|---|
| FD-R-002539-01 | None | None | None |