Ignite Creation Date:
2024-05-05 @ 9:57 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00006136
Status:
COMPLETED
Last Update Posted:
2005-11-08
First Post:
2000-08-03
Brief Title:
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
Sponsor:
Boston University
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2004-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
OBJECTIVES I Determine whether arginine butyrate with or without epoetin alfa can stimulate gamma-globin chain production to a degree that decreases anemia and results in hematologic improvement in patients with thalassemia intermedia
II Determine whether a proportional increase in gamma-globin synthesis and mRNA and an improvement in nonalfa and alfaglobin chain imbalance by at least 10 over baseline correlate with improved hematologic response in these patients when treated with this regimen
III Determine whether a decrease in hemolysis as assayed by a decrease in LDH compared to baseline levels correlates with improved hematologic response in these patients when treated with this regimen
IV Determine whether any particular genotypes are more responsive than others to this therapy in these patients
V Determine whether baseline epoetin alfa levels gender andor baseline reticulocyte counts or percent circulating nucleated erythroblasts correlate with improved hematologic response in these patients when treated with this regimen
II Determine whether a proportional increase in gamma-globin synthesis and mRNA and an improvement in nonalfa and alfaglobin chain imbalance by at least 10 over baseline correlate with improved hematologic response in these patients when treated with this regimen
III Determine whether a decrease in hemolysis as assayed by a decrease in LDH compared to baseline levels correlates with improved hematologic response in these patients when treated with this regimen
IV Determine whether any particular genotypes are more responsive than others to this therapy in these patients
V Determine whether baseline epoetin alfa levels gender andor baseline reticulocyte counts or percent circulating nucleated erythroblasts correlate with improved hematologic response in these patients when treated with this regimen
Detailed Description:
PROTOCOL OUTLINE This is a multicenter study Patients receive arginine butyrate IV over 6-14 hours on days 1-5 of weeks 1-4 and 7-10 Patients then receive maintenance arginine butyrate IV over 6-14 hours on days 1-4 of weeks 13 15 17 19 21 23 and 25
Patients who have no medical contraindications eg paraspinal extramedullary hematopoiesis hypertension or poorly controlled congestive heart failure may continue therapy Patients receive arginine butyrate IV over 6-14 hours on days 1-4 of weeks 27 29 31 33 35 37 and 39 and epoetin alfa intramuscularly IM or subcutaneously SC three times weekly on weeks 27-40
Patients may continue to receive epoetin alfa IM or SC alone three times weekly on weeks 41-52 Patients with severe anemia hemoglobin less than 7 gdL may receive epoetin alfa alone on weeks 1-12 before arginine butyrate induction therapy
Patients who complete therapy at week 26 are followed every 2 weeks for 2 months Patients who complete therapy at week 40 are followed monthly for 2 months
Patients who have no medical contraindications eg paraspinal extramedullary hematopoiesis hypertension or poorly controlled congestive heart failure may continue therapy Patients receive arginine butyrate IV over 6-14 hours on days 1-4 of weeks 27 29 31 33 35 37 and 39 and epoetin alfa intramuscularly IM or subcutaneously SC three times weekly on weeks 27-40
Patients may continue to receive epoetin alfa IM or SC alone three times weekly on weeks 41-52 Patients with severe anemia hemoglobin less than 7 gdL may receive epoetin alfa alone on weeks 1-12 before arginine butyrate induction therapy
Patients who complete therapy at week 26 are followed every 2 weeks for 2 months Patients who complete therapy at week 40 are followed monthly for 2 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 |
|---|---|---|---|
| BUSM-4839 | None | None | None |