Ignite Creation Date:
2024-05-06 @ 12:54 PM
Last Modification Date:
2024-10-26 @ 1:05 PM
Study NCT ID:
NCT03878069
Status:
COMPLETED
Last Update Posted:
2023-04-27
First Post:
2019-03-12
Brief Title:
Registry Study of Revcovi Treatment in Patients With ADA-SCID
Sponsor:
Chiesi Farmaceutici SpA
Organization:
Chiesi Farmaceutici SpA
Study Overview
Official Title:
Single Arm Open-Label Multicenter Registry Study of Revcovi Elapegademase-lvlr Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
Status:
COMPLETED
Status Verified Date:
2023-04
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:
This registry is conducted in patients with adenosine deaminase severe combined immune deficiency ADA-SCID treated with Revcovi to collect periodic clinical and biochemical data on safety and dose adjustment
Detailed Description:
Patients with ADA-SCID who require treatment with Revcovi as Enzyme Replacement Therapy ERT will be followed until the last enrolled patient has reached a minimum of 24 months of Revcovi treatment or until undergoing HSCT or HSCGT whichever occurs first
Patients undergoing HSCT or HSC-GT will be followed one month after last Revcovi dose and again at six months to assess adverse events AEs and survival Throughout the duration of the study patients will be assessed continually for AEs
PatientsParentsCaregivers will self-administer weekly intramuscular IM doses of Revcovi and will be followed according to the Suggested Schedule of Assessments for trough dAXP and ADA activity Treatment dosing and monitoring will be individualized per provider and patient characteristics in adherence with each study sites standards of care
Patients in the Phase 3 Revcovi study STP-2279-002 will be given the opportunity to enroll in this registry study and proceed to the Treatment Month 6 Visit per the Suggested Schedule of Assessments for Adagen-Transitioning Patients
Patients undergoing HSCT or HSC-GT will be followed one month after last Revcovi dose and again at six months to assess adverse events AEs and survival Throughout the duration of the study patients will be assessed continually for AEs
PatientsParentsCaregivers will self-administer weekly intramuscular IM doses of Revcovi and will be followed according to the Suggested Schedule of Assessments for trough dAXP and ADA activity Treatment dosing and monitoring will be individualized per provider and patient characteristics in adherence with each study sites standards of care
Patients in the Phase 3 Revcovi study STP-2279-002 will be given the opportunity to enroll in this registry study and proceed to the Treatment Month 6 Visit per the Suggested Schedule of Assessments for Adagen-Transitioning Patients
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?:
False
Is an FDA AA801 Violation?:
None