Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases

NCT03939533 | Completed Phase 3 Results FDA Drug

• 1 other identifier: SCGAM-06
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 18

Brief Summary

CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES

Conditions

Primary Immune Deficiency Disorder

Outcome Measures

Primary Outcomes (1)

• IgG Trough Levels From Baseline to End of Study (28 Weeks)

  Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study (28 weeks) every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline through study completion (28 weeks)

  Through study completion, up to 28 weeks

Secondary Outcomes (4)

• Serious Bacterial Infection Rates

  Through study completion, up to 28 weeks

• Time to Resolution of Infections

  Through study completion, 28 weeks

• Antibiotic Usage

  Through study completion, up to 28 weeks

• Number of Antibiotic Treatment Episodes Annualized

  Through study completion, up to 28 weeks

Study Arms (3)

Increased Volume Cohort - Cohort 1

EXPERIMENTAL

Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks

Drug: CUTAQUIG

Increased Infusion Rate Cohort - Cohort 2

EXPERIMENTAL

Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

Drug: CUTAQUIG

Every Other Week Dosing Cohort - Cohort 3

EXPERIMENTAL

Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose

Drug: CUTAQUIG

Interventions

CUTAQUIG DRUG

Human normal immunoglobulin

Every Other Week Dosing Cohort - Cohort 3; Increased Infusion Rate Cohort - Cohort 2; Increased Volume Cohort - Cohort 1

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥2 years and ≤75 years.
• Confirmed diagnosis of primary immunodeficiency (PI) disease as defined by the European Society for Immunodeficiencies and Pan American Group for Immunodeficiency and requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. Note: The exact type of PI disease will be recorded.
• Established on a consistent or stable mg/kg dose of any SCIG treatment for a minimum of 3 months prior to Screening. Note: patients entering Cohort 3 must be on weekly SCIG infusions for a minimum of 12 weeks.
• Availability of the Immunoglobulin G (IgG) trough levels of 2 previous SCIG infusions within 1 year of Screening, with 1 trough level obtained within 3 months prior to enrollment, and maintenance of trough serum IgG levels
• ≥5.0 g/L in 2 previous infusions. Patients with no prior IgG trough level within 3 months prior to enrollment may use the Screening IgG trough level as their 2nd reading.
• Voluntarily given, fully informed signed informed consent. For patients under the legal age of consent, voluntarily given, fully-informed, signed informed consent will be provided by patient's parent or legal guardian, and assent will be provided by patient (per age-appropriate Institutional Review Board \[IRB\] requirements).
• Females of childbearing potential, who are not nursing and have no plans for pregnancy during the course of the study, have been using at least 1 acceptable form of birth control for a minimum of 30 days prior to the Screening visit and must agree to use at least 1 acceptable method of contraception for 30 days after the last dose of CUTAQUIG. Acceptable methods include: intrauterine device (IUD), hormonal contraception, male or female condom, spermicide gel, diaphragm, sponge, cervical cap, or abstinence.
• For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening visit.
• Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

You may not qualify if:

• Evidence of active infection within 4 weeks of Screening or during the Screening Period.
• Current or clinically-significant history of any cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurological, immunological (excluding PI), hematologic, and/or psychiatric disorder(s), or a history of any other illness that, in the opinion of the Investigator, might confound the results of the study, or pose additional risk to the patient by participation in the study.
• Known history of adverse reactions to immunoglobulin A (IgA) in other products.
• Body mass index (BMI) \>40 kg/m2 for patients entering Cohort 2 or Cohort 3. There are no BMI restrictions for Cohort 1.
• Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as Polysorbate 80).
• Requirement of any routine premedication for IgG administration.
• History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
• Severe liver function impairment (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] \>3 times above upper limit of normal).
• Known protein-losing enteropathies or clinically significant proteinuria.
• Presence of renal function impairment (creatine \>120 μM/L or creatinine \>1.35 mg/dL), or predisposition for acute renal failure (eg, any degree of preexisting renal insufficiency or routine treatment with known nephritic drugs).
• Treatment with oral or parenteral steroids for ≥30 days, or when given intermittently or as bolus at daily doses ≥0.15 mg/kg when taken within 30 days of Screening. Note: Short or intermittent courses of steroids (ie, a steroid burst) of \>0.15 mg/kg/day is allowed for treatment of a short-term condition such as an asthma exacerbation.
• Treatment with immunosuppressive or immunomodulatory drugs (except Omalizumab).
• Use of HYQVIA (Immune Globulin Infusion 10% \[Human\] with Recombinant Human Hyaluronidase) within 3 months prior to first CUTAQUIG infusion.
• Live viral vaccination (such as measles, rubella, mumps, and varicella) within 2 months prior to first CUTAQUIG infusion.
• Exposure to blood or any blood product or derivative, other than subcutaneous IgG used for regular PI disease treatment, within 3 months before the first CUTAQUIG infusion.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (18)

Octapharma Research Site

Scottsdale, Arizona, 85251, United States

Location

Octapharma Research Site

Irvine, California, 92697, United States

Location

Octapharma Research Site

Santa Barbara, California, 93105, United States

Location

Octapharma Research Site

Centennial, Colorado, 80112, United States

Location

Octapharma Research Site

North Palm Beach, Florida, 33408, United States

Location

Octapharma Research Site

St. Petersburg, Florida, 33701, United States

Location

Octapharma Research Site

Albany, Georgia, 31707, United States

Location

Octapharma Research Site

Louisville, Kentucky, 40215, United States

Location

Octapharma Research Site

Chevy Chase, Maryland, 20815, United States

Location

Octapharma Research Site

St Louis, Missouri, 63141, United States

Location

Octapharma Research Site

Papillion, Nebraska, 68046, United States

Location

Octapharma Research Site

Asheville, North Carolina, 28801, United States

Location

Octapharma Research Site

Cincinnati, Ohio, 45231, United States

Location

Octapharma Research Site

Mayfield, Ohio, 44124, United States

Location

Octapharma Research Site

Toledo, Ohio, 43617, United States

Location

Octapharma Research Site

Pittsburgh, Pennsylvania, 15241, United States

Location

Octapharma Research Site

Dallas, Texas, 75225, United States

Location

Octapharma Research Site

Bellingham, Washington, 98225, United States

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Limitations and Caveats

For outcome #3, 'Mean' is correct. The values presented are indeed calculated as means using the usual formula (only for the CIs a compound Poisson process model is used to account for the intra-patient correlation in incidents).For each patient, the annual infection rate was calculated as # of infections/observation period.

Results Point of Contact

• Title: Patrick Murphy
• Organization: CRMG

p.murphy@crmg-usa.com

413-821-0022

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2019
• First Posted: May 6, 2019
• Study Start: October 17, 2019
• Primary Completion: January 3, 2022
• Study Completion: January 3, 2022
• Last Updated: November 7, 2023
• Results First Posted: November 7, 2023

Record last verified: 2023-11

Locations

US (18)