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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT03878069
Date of registration: 12/03/2019
Prospective Registration: Yes
Primary sponsor: Leadiant Biosciences, Inc.
Public title: Registry Study of Revcovi Treatment in Patients With ADA-SCID
Scientific title: Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
Date of first enrolment: June 25, 2019
Target sample size: 20
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03878069
Study type:  Observational [Patient Registry]
Study design:   
Phase: 
Countries of recruitment
United States
Contacts
Name:     Joseph M Wiley, MD
Address: 
Telephone: 301-670-2182
Email: Joseph.Wiley@leadiant.com
Affiliation: 
Name:     Joseph M Wiley, MD
Address: 
Telephone:
Email:
Affiliation:  Leadiant Biosciences, Inc.
Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with diagnosis of ADA-SCID who require ERT with ADA as judged by the treating
physicians, based on the medical history, biochemical test or genotyping.

- Understanding and willing to comply with the Registry recommendations via signed and
dated written informed consent/assent.

- ADA-SCID patient requiring Revcovi as an ERT.

Exclusion Criteria:

- Any condition that, in the opinion of the Investigator, makes the patient unsuitable
for the study.



Age minimum: N/A
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Adenosine Deaminase Deficiency
Severe Combined Immunodeficiency
Intervention(s)
Biological: elapegademase-lvlr
Primary Outcome(s)
ADA activity [Time Frame: Month 24]
Deoxyadenosine nucleotides (dAXP) activity [Time Frame: Month 24]
Secondary Outcome(s)
Clinical status [Time Frame: Month 24]
Immune status (SSA/PI) [Time Frame: Month 24]
Safety assessed by determining adverse events (AEs), serious adverse events (SAEs) [Time Frame: Month 24]
Secondary ID(s)
LBI-2279-004
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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