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 range

0 Months – 65 Years

Sex

ALL

See this in plain English?

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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 range

0 Months – 65 Years

Sex

ALL

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