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Registry Study of Revcovi Treatment in Patients With ADA-SCID

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ClinicalTrials.gov Identifier: NCT03878069
Recruitment Status : Not yet recruiting
First Posted : March 18, 2019
Last Update Posted : March 18, 2019
Sponsor:
Information provided by (Responsible Party):
Leadiant Biosciences, Inc.

Brief Summary:
The objective of this study is to develop a registry of patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) treated with Revcovi™ that contains clinical and biochemical assessments for safety and dose adjustment based on adenosine deaminase (ADA) activity and erythrocyte deoxyadenosine nucleotide (dAXP) levels as well as immunologic monitoring.

Condition or disease Intervention/treatment
Adenosine Deaminase Deficiency Severe Combined Immunodeficiency Biological: elapegademase-lvlr

Detailed Description:

Patients with ADA-SCID who require treatment with Revcovi as Enzyme Replacement Therapy (ERT) will be enrolled during a 24-month period and each will be followed for 24 months after starting Revcovi or until undergoing hematopoietic stem cell transplant (HSCT) or hematopoietic stem cell gene therapy (HSC-GT), 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.

Participants in the STP-2279-002 trial 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.

An interim analysis will be performed approximately two years after study initiation.


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Study Type : Observational [Patient Registry]
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 2 Years
Official Title: Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
Estimated Study Start Date : June 2019
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023


Group/Cohort Intervention/treatment
ERT with ADA
Patients with diagnosis of ADA-SCID treated with ERT with Revcovi or transitioning to Revcovi from Adagen
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.

Other Name: Revcovi




Primary Outcome Measures :
  1. Deoxyadenosine nucleotides (dAXP) activity [ Time Frame: Month 24 ]
    Total trough erythrocyte dAXP activity

  2. ADA activity [ Time Frame: Month 24 ]
    Trough plasma ADA activity


Secondary Outcome Measures :
  1. Immune status (SSA/PI) [ Time Frame: Month 24 ]

    Absolute lymphocyte count and subset B, T, and NK analysis.

    Immunoglobulin (Ig) concentrations (IgG, IgA, and IgM).

    Measurement of immune response at Investigator discretion.


  2. Clinical status [ Time Frame: Month 24 ]
    Infections (clinically or microbiologically documented) Incidence and duration of hospitalizations Growth for patients < 18 years old Overall survival through the end of study

  3. Safety assessed by determining adverse events (AEs), serious adverse events (SAEs) [ Time Frame: Month 24 ]
    Assessed by determining adverse events (AEs), serious adverse events (SAEs), clinical signs and symptoms from physical examinations, and laboratory examinations



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Ages Eligible for Study:   up to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients who receive at least one dose of study drug and have at least one safety evaluation.
Criteria

Inclusion Criteria:

  • Patients with diagnosis of ADA-SCID who require ERT with ADA as judged by the treating physicians, based on the medical history, biochemical test or genotyping.
  • Understanding and willing to comply with the Registry recommendations via signed and dated written informed consent/assent.
  • ADA-SCID patient requiring Revcovi as an ERT.

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03878069


Contacts
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Contact: Joseph M Wiley, MD 301-670-2182 Joseph.Wiley@leadiant.com
Contact: Elizabeth Walsh 301-670-5447 Elizabeth.Walsh@leadiant.com

Sponsors and Collaborators
Leadiant Biosciences, Inc.

Additional Information:

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Responsible Party: Leadiant Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT03878069     History of Changes
Other Study ID Numbers: LBI-2279-004
First Posted: March 18, 2019    Key Record Dates
Last Update Posted: March 18, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Agammaglobulinemia
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Blood Protein Disorders
Hematologic Diseases
Lymphoproliferative Disorders
Lymphatic Diseases