CompletedNot Applicable

Registry Study of Revcovi Treatment in Patients With ADA-SCID

United States32 participantsStarted 2019-09-30

Plain-language summary

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.

Who can participate

Age range0 Months – 65 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

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

Timeframe: From enrollment to end of treatment up to Month 24

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

Timeframe: From enrollment to end of treatment up to Month 24

Safety of Revcovi

Timeframe: From enrollment to end of treatment up to Month 24

Trial details

NCT IDNCT03878069

SponsorChiesi Farmaceutici S.p.A.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-01-18

Results submitted2024-03-28

View on ClinicalTrials.gov More Adenosine Deaminase Severe Combined Immunodeficiency trials

Who can participate

Age range0 Months – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

Timeframe: From enrollment to end of treatment up to Month 24

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

Timeframe: From enrollment to end of treatment up to Month 24

Safety of Revcovi

Timeframe: From enrollment to end of treatment up to Month 24