Clinical trial NCT03878069: Registry Study of Revcovi Treatment in Patients With ADA-SCID
Official title: Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Adenosine Deaminase Severe Combined Immunodeficiency
Keywords: ADA-SCID
Revcovi
ERT
Lead sponsor: Chiesi Farmaceutici S.p.A.
Sponsor class: INDUSTRY
First posted: 2019-03-18
Enrollment: 32
Sex: ALL
Minimum age (years): 0
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with ADA-SCID who require ERT

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: 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
measure: 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
measure: Safety of Revcovi
timeFrame: From enrollment to end of treatment up to Month 24
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: San Diego
country: United States
facility: Allergy & Asthma Medical Group and Research Center, A P.C.
state: California
city: San Francisco
country: United States
facility: UCSF - University of California
state: California
city: Washington
country: United States
facility: Children's National
state: District of Columbia
city: Saint Petersburg
country: United States
facility: University of South Florida Allergy Immunology Clinic
state: Florida
city: New Orleans
country: United States
facility: Childrens Hospital of New Orleans
state: Louisiana
city: Minneapolis
country: United States
facility: Children's Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital - Washington University School of Medicine
state: Missouri
city: Buffalo
country: United States
facility: UBMD Pediatrics Outpatient Center
state: New York
city: Durham
country: United States
facility: Duke University Hospital
state: North Carolina
city: Hershey
country: United States
facility: Penn State Children's Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Memphis
country: United States
facility: Le Bonheur Children's Hospital
state: Tennessee
city: Seattle
country: United States
facility: Seattle Children's
state: Washington
Study officials:
References: