Registry Study of Revcovi Treatment in Patients With ADA-SCID

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Brief Title

Registry Study of Revcovi Treatment in Patients With ADA-SCID

Official Title

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

Brief 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.
    

Detailed Description

      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.
    


Study Type

Observational [Patient Registry]


Primary Outcome

Deoxyadenosine nucleotides (dAXP) activity

Secondary Outcome

 Immune status (SSA/PI)

Condition

Adenosine Deaminase Deficiency

Intervention

elapegademase-lvlr

Study Arms / Comparison Groups

 ERT with ADA
Description:  Patients with diagnosis of ADA-SCID treated with ERT with Revcovi or transitioning to Revcovi from Adagen

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

20

Start Date

June 25, 2019

Completion Date

July 2023

Primary Completion Date

June 2023

Eligibility 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.
      

Gender

All

Ages

N/A - 65 Years

Accepts Healthy Volunteers

No

Contacts

Joseph M Wiley, MD, 301-670-2182, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03878069

Organization ID

LBI-2279-004


Responsible Party

Sponsor

Study Sponsor

Leadiant Biosciences, Inc.


Study Sponsor

Joseph M Wiley, MD, Study Director, Leadiant Biosciences, Inc.


Verification Date

November 2019