Registry Study of Revcovi Treatment in Patients With ADA-SCID

Severe Combined Immunodeficiency Clinical Trial

Official title:

Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03878069
• Other study ID #: CLI-06814AA1-01
• Status: Completed
• Start date: June 25, 2019
• Est. completion date: January 18, 2023

Study information

• Verified date: April 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

This registry is conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ to collect periodic clinical and biochemical data on safety and dose adjustment.

Description:

Patients with ADA-SCID who require treatment with Revcovi as Enzyme Replacement Therapy (ERT) will be followed until the last enrolled patient has reached a minimum of 24 months of Revcovi treatment or until undergoing HSCT or HSCGT, whichever occurs first.

Patients undergoing HSCT or HSC-GT will be followed one month after last Revcovi dose and again at six months to assess adverse events (AEs) and survival. Throughout the duration of the study, patients will be assessed continually for AEs.

Patients/Parents/Caregivers will self-administer weekly intramuscular (IM) dose(s) of Revcovi and will be followed according to the Suggested Schedule of Assessments for trough dAXP and ADA activity. Treatment dosing and monitoring will be individualized per provider and patient characteristics in adherence with each study sites' standards of care.

Patients in the Phase 3 Revcovi™ study (STP-2279-002) will be given the opportunity to enroll in this registry study and proceed to the Treatment Month 6 Visit per the Suggested Schedule of Assessments for Adagen-Transitioning Patients.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: January 18, 2023
• Est. primary completion date: January 18, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Months to 65 Years

Eligibility

Inclusion Criteria:

• Patients currently receiving chronic ERT with Adagen® and transitioned/transitioning to Revcovi;

• Infants diagnosed via newborn screening and definitive testing for ADA deficiency prescribed Revcovi;

• Patients receiving Revcovi while preparing for Hematopoietic Stem Cell Transplant (HSCT) or Hematopoietic Stem Cell Gene Therapy (HSCGT)

• Patients who decline, are ineligible or do not respond to HSCT or HSC-GT and resume/start Revcovi.

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Adenosine Deaminase Deficiency
• Severe Combined Immunodeficiency

Intervention

Biological:
• elapegademase-lvlr
Patients transitioning from Adagen: For patients currently receiving Adagen at = 30 U/kg/wk or an unknown Adagen dose, the suggested dosage of Revcovi is 0.2 mg/kg/wk IM. For patients currently receiving Adagen at > 30 U/kg/wk the suggested equivalent Revcovi dosage (mg/kg/wk) is the Adagen dosage in U/kg/wk divided by 150. At the investigator's discretion, the total weekly dose may be divided and administered in multiple IM injections, increased by 0.033 mg/kg/wk if trough ADA activity is < 30 mmol/hr/L, dAXP is > 0.02 mmol/L, and/or the immune reconstitution is inadequate. Adagen-naïve patients: The suggested starting Revcovi dosage is 0.2 mg/kg twice weekly IM based on ideal body weight, for a minimum of 12 to 24 weeks until immune reconstitution is achieved. At the investigator's discretion, the dosage may be gradually increased to maintain trough ADA activity > 30 mmol/hr/L, dAXP < 0.02 mmol/L, and/or to maintain adequate immune reconstitution.

Locations

Country	Name	City	State
United States	UBMD Pediatrics Outpatient Center	Buffalo	New York
United States	Duke University Hospital	Durham	North Carolina
United States	Penn State Children's Hospital	Hershey	Pennsylvania
United States	University of California Los Angeles	Los Angeles	California
United States	Le Bonheur Children's Hospital	Memphis	Tennessee
United States	Children's Minnesota	Minneapolis	Minnesota
United States	Childrens Hospital of New Orleans	New Orleans	Louisiana
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	St. Louis Children's Hospital - Washington University School of Medicine	Saint Louis	Missouri
United States	University of South Florida Allergy Immunology Clinic	Saint Petersburg	Florida
United States	Allergy & Asthma Medical Group and Research Center, A P.C.	San Diego	California
United States	UCSF - University of California	San Francisco	California
United States	Seattle Children's	Seattle	Washington
United States	Children's National	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Deoxyadenosine nucleotides (dAXP) activity	Total trough erythrocyte dAXP activity	Month 24
Primary	ADA activity	Trough plasma ADA activity	Month 24
Secondary	Immune status (SSA/PI)	Absolute lymphocyte count and subset B, T, and NK analysis.; Immunoglobulin (Ig) concentrations (IgG, IgA, and IgM).; Measurement of immune response at Investigator discretion.	Month 24
Secondary	Clinical status	Infections (clinically or microbiologically documented) Incidence and duration of hospitalizations Growth for patients < 18 years old Overall survival through the end of study	Month 24
Secondary	Safety assessed by determining adverse events (AEs), serious adverse events (SAEs)	Assessed by determining adverse events (AEs), serious adverse events (SAEs), clinical signs and symptoms from physical examinations, and laboratory examinations	Month 24