Registry Study of Revcovi Treatment in Patients With ADA-SCID

Chiesi Farmaceutici S.p.A.32 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Adenosine Deaminase Severe Combined Immunodeficiency

Interventions

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Revcovi is administered intramuscularly (i.m.). Weekly dosage is calculated in mg/kg of body weight, and can be adjusted over the course of the trial based on ADA activity and dAXP concentration as well as on clinical assessment by the treating physician.

Eligibility

Sex: ALLMin age: 0 MonthsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (14)

University of California Los Angeles

Los Angeles, California, United States

Allergy & Asthma Medical Group and Research Center, A P.C.

San Diego, California, United States

Outcomes

Primary Outcomes

Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement

A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.

Time frame: From enrollment to end of treatment up to Month 24

Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement

An optimal level of ADA plasma activity is defined as a trough value of 30 millimoles per hour per liter (mmol/h/L) or greater. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.

Time frame: From enrollment to end of treatment up to Month 24

Safety of Revcovi

The number of subjects reporting adverse events (AEs). Due to the nature of ADA-SCID, only AEs deemed to be at least possibly related to Revcovi treatment were documented.

Time frame: From enrollment to end of treatment up to Month 24

Data from ClinicalTrials.gov

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