Registry Study of Revcovi Treatment in Patients With ADA-SCID

Chiesi

Status: Completed

Conditions: Adenosine Deaminase Severe Combined Immunodeficiency

Treatments: Biological: elapegademase-lvlr

Study type: Observational

Funder types: Industry

Identifiers

NCT03878069

CLI-06814AA1-01

Details and patient eligibility

About

This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi. Data on safety and on measures of efficacy are collected.

Full description

Patients with ADA-SCID who require ERT will receive Revcovi on a dosage and schedule determined by the treating physician. They will be followed for safety throughout the study, and will be monitored for the efficacy markers of adenosine deaminase (ADA) activity and deoxyadenosine nucleotide (dAXP) concentration according to a suggested schedule. Some subjects will be new to ERT; some will have transitioned from Adagen, which was the ERT available before Revcovi; and some will have previously participated in an earlier Phase 3 trial of Revcovi (study STP-2279-002). Patients will be followed either until they are able to successfully undergo a stem cell transplant or stem cell gene therapy and thus no longer require ERT treatment, or until all ongoing participants have received a minimum of 24 months of Revcovi treatment.

Note: Due to the nature of this study, all analyses are descriptive and no statistical hypotheses will be tested.

Enrollment: 32 patients

Sex: All

Ages: Under 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female, aged newborn to adult

• In need of ERT treatment due to one of the following circumstances:

  • Waiting to receive a stem cell transplant
  • Previously declined, had been found to be ineligible for, or did not respond to a stem cell transplant
  • Waiting to enrol in a clinical trial on gene therapy, or had been found to be ineligible for or had failed such a trial

• One of the following histories of ERT treatment:

  • Revcovi only
  • Previously on Adagen but had transitioned to Revcovi
  • Not yet on any ERT but about to start on Revcovi

Exclusion criteria

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

Trial design

32 participants in 1 patient group

Patients with ADA-SCID in need of ERT treatment

Description:

All participants will receive Revcovi. For analysis purposes, there will be three groups, who differ with respect to ERT history: * Adagen-naïve: Subjects starting on ERT for the first time * Adagen-transitioning: Subjects switching from Adagen to Revcovi * STP-2279-002 participants: Subjects who had taken part in an earlier Phase III trial of Revcovi (study STP-2279-002) in which they had been switched from Adagen to Revcovi. This last group differs from the Adagen-transitioning group in that they will have been on Revcovi the longest.

Treatment:

Biological: elapegademase-lvlr