Ignite Creation Date:
2024-05-05 @ 5:02 PM
Last Modification Date:
2024-10-26 @ 9:27 AM
Study NCT ID:
NCT00377416
Status:
COMPLETED
Last Update Posted:
2020-04-10
First Post:
2006-09-14
Brief Title:
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
Sponsor:
University of Massachusetts Worcester
Organization:
University of Massachusetts Worcester
Study Overview
Official Title:
Preclinical Phase III Trials of AAV-AAT Vectors Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin rAAV2-CB-hAAT Gene Vector to AAT-Deficient Adults
Status:
COMPLETED
Status Verified Date:
2020-04
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:
Individuals with a deficiency of the Alpha 1-antitrypsin AAT protein are at risk for developing emphysema and liver damage Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency
Detailed Description:
AAT deficiency is a genetic disorder in which individuals have inadequate levels of the AAT protein AAT protects the lungs from white blood cell enzymes that can damage air sacs within the lungs potentially leading to emphysema Experimental gene transfer procedures in which normal copies of genes are inserted into cells are being developed to treat many genetic diseases including AAT deficiency In this study a modified virus adeno-associated virus AAV has been genetically engineered to contain a normal copy of the AAT gene When AAV is combined with the AAT gene the resulting agent rAAV2-CB-hAAT is able to carry normal copies of the AAT gene into muscle cells to produce additional AAT The purpose of this study is to evaluate the safety of injecting rAAV2-CB-hAAT into individuals with AAT deficiency
This 13-month study will enroll individuals with AAT deficiency Participants currently using AAT protein replacement will discontinue its use for 15 weeks during the study Participants will first attend a baseline study visit which will include a medical history review a physical examination an electrocardiogram ECG to record heart activity blood urine and semen collection pulmonary function tests and chest and arm scans Participants will then attend a 5-day inpatient visit during which they will receive a series of injections consisting of one of four different doses of rAAV2-CB-hAAT Physical examinations will occur on all 5 inpatient days pulmonary function testing arm circumference measurements and collection of blood urine and semen will occur on selected days of the inpatient stay Follow-up study visits with possible overnight stays will occur on Days 14 and 90 On Days 30 45 60 75 180 270 and 365 participants will have blood drawn at a local clinic On these same days study staff will contact participants by telephone to review their medical history and symptoms Unused blood and semen samples will be frozen and stored for future research purposes Participants will have yearly follow-up evaluations by either telephone or mail for a total of 15 years
This 13-month study will enroll individuals with AAT deficiency Participants currently using AAT protein replacement will discontinue its use for 15 weeks during the study Participants will first attend a baseline study visit which will include a medical history review a physical examination an electrocardiogram ECG to record heart activity blood urine and semen collection pulmonary function tests and chest and arm scans Participants will then attend a 5-day inpatient visit during which they will receive a series of injections consisting of one of four different doses of rAAV2-CB-hAAT Physical examinations will occur on all 5 inpatient days pulmonary function testing arm circumference measurements and collection of blood urine and semen will occur on selected days of the inpatient stay Follow-up study visits with possible overnight stays will occur on Days 14 and 90 On Days 30 45 60 75 180 270 and 365 participants will have blood drawn at a local clinic On these same days study staff will contact participants by telephone to review their medical history and symptoms Unused blood and semen samples will be frozen and stored for future research purposes Participants will have yearly follow-up evaluations by either telephone or mail for a total of 15 years
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
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 |
|---|---|---|---|
| IRB 306-03 | Other Identifier | University of Florida | httpsreporternihgovquickSearchR01HL069877 |
| R01HL069877 | NIH | None | None |
| NIH Protocol 30104-465 | OTHER | None | None |
| UF GCRC 567 | OTHER | None | None |
| UF IBC RD 2101 | None | None | None |
| UF GTC TRF AAV001 | None | None | None |