Summary

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

Description

Summary

The objective of this study is to develop a registry of patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ that contains clinical and biochemical assessments for safety and dose adjustment based on adenosine deaminase (ADA) activity and erythrocyte deoxyadenosine nucleotide (dAXP) levels as well as immunologic monitoring.

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 enrolled during a 24-month period and each will be followed for 24 months after starting Revcovi or until undergoing hematopoietic stem cell transplant (HSCT) or hematopoietic stem cell gene therapy (HSC-GT), 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. Participants in the STP-2279-002 trial 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. An interim analysis will be performed approximately two years after study initiation.

Keywords

Adenosine Deaminase Deficiency Severe Combined Immunodeficiency elapegademase-lvlr

Eligibility

You can join if…

Open to people ages up to 65 years

  • 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.

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 not yet accepting 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

Details

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