Summary

Eligibility
for people ages 0 months to 65 years (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Morna Dorsey, MD
Headshot of Morna Dorsey
Morna Dorsey

Description

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.

Official Title

Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy

Details

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. Patients/Parents/Caregivers will self-administer weekly intramuscular (IM) dose(s) 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.

Keywords

Adenosine Deaminase Deficiency Severe Combined Immunodeficiency Elapegademase elapegademase-lvlr

Eligibility

You can join if…

Open to people ages 0 months to 65 years

  • Patients currently receiving chronic ERT with Adagen® and transitioned/transitioning to Revcovi;
  • Infants diagnosed via newborn screening and definitive testing for ADA deficiency prescribed Revcovi;
  • Patients receiving Revcovi while preparing for Hematopoietic Stem Cell Transplant (HSCT) or Hematopoietic Stem Cell Gene Therapy (HSCGT)
  • Patients who decline, are ineligible or do not respond to HSCT or HSC-GT and resume/start Revcovi.

You CAN'T join if...

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

Locations

  • UCSF - University of California accepting new patients
    San Francisco California 94158 United States
  • University of California Los Angeles accepting new patients
    Los Angeles California 90095-1752 United States

Lead Scientist at UCSF

  • Morna Dorsey, MD
    My research interests lie in three main focus areas: 1. Optimizing diagnosis and care of patients with primary immunodeficiency 2. Investigating the psychosocial impact of primary immunodeficiency on patients and families 3. Establishing treatment for patients with food allergy and eosinophilic disorders

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Chiesi Farmaceutici S.p.A.
ID
NCT03878069
Study Type
Observational [Patient Registry]
Participants
Expecting 34 study participants
Last Updated