Registry Study of Revcovi Treatment in Patients With ADA-SCID
Condition: Adenosine Deaminase Deficiency; Severe Combined Immunodeficiency
MeSH terms: Severe Combined Immunodeficiency
Study type: Observational [Patient Registry]

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.
    

Detailed description: 
      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.
    

Intervention name(s): elapegademase-lvlr
Intervention type(s): Biological
Intervention description(s): Patients transitioning from Adagen: For patients currently receiving Adagen at ≤ 30 U/kg/wk or an unknown Adagen dose, the suggested dosage of Revcovi is 0.2 mg/kg/wk IM. For patients currently receiving Adagen at > 30 U/kg/wk the suggested equivalent Revcovi dosage (mg/kg/wk) is the Adagen dosage in U/kg/wk divided by 150.
At the investigator's discretion, the total weekly dose may be divided and administered in multiple IM injections, increased by 0.033 mg/kg/wk if trough ADA activity is < 30 mmol/hr/L, dAXP is > 0.02 mmol/L, and/or the immune reconstitution is inadequate.
Adagen-naïve patients: The suggested starting Revcovi dosage is 0.2 mg/kg twice weekly IM based on ideal body weight, for a minimum of 12 to 24 weeks until immune reconstitution is achieved. At the investigator's discretion, the dosage may be gradually increased to maintain trough ADA activity > 30 mmol/hr/L, dAXP < 0.02 mmol/L, and/or to maintain adequate immune reconstitution.
Other intervention name(s): Revcovi
Intervention MeSH terms: Elapegademase
Observational model: Cohort
Time perspective: Other
Sampling method: Non-Probability Sample
Study population: 
        There are four types of patients with ADA-SCID who may be eligible for treatment with
        Revcovi, including:

          -  Patients currently receiving chronic ERT with Adagen

          -  Infants diagnosed via newborn screening and/or definitive testing for ADA deficiency

          -  Patients preparing for HSCT or HSC-GT

          -  Patients who decline, are ineligible or do not respond to HSCT or HSC-GT