Registry Study of Revcovi Treatment in Patients With ADA-SCID

Related Clinical Trial
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus cliniMACs HUD for T Cell Depletion Cord Blood Stem Cell Transplantation Study (COBLT) Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency Genetic Basis of Immunodeficiency IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency Sirolimus Prophylaxis for aGVHD in TME SCID Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft Influences on Female Adolescents’ Decisions Regarding Testing for Carrier Status of XSCID Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant EZN-2279 in Patients With ADA-SCID AMG191 Conditioning/CD34+CD90 Stem Cell Transplant Study for SCID Patients Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) Patients Treated for SCID (1968-Present) Clinical Characteristics and Genetic Profiles of Severe Combined Immunodeficiency in China Gene Therapy for ADA-SCID MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim Natural History Study of SCID Disorders Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency SCID Bu/Flu/ATG Study With T Cell Depletion Generalized Neonatal Screening of Severe Combined Immunodeficiencies Neonatal Screening of Severe Combined Immunodeficiencies Gene Therapy for X-linked Severe Combined Immunodeficiency An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) Registry Study of Revcovi Treatment in Patients With ADA-SCID Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

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

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)


Adenosine Deaminase Deficiency



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


* 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


Estimated Enrollment


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.




N/A - 65 Years

Accepts Healthy Volunteers



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

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Chiesi Farmaceutici S.p.A.

Study Sponsor

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

Verification Date

December 2020