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

NCT ID: NCT03939533

Last Updated: 2023-11-07

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

64 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-10-17

Study Completion Date

2022-01-03

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Primary Immune Deficiency Disorder

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Increased Volume Cohort - Cohort 1

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

Group Type EXPERIMENTAL

CUTAQUIG

Intervention Type DRUG

Human normal immunoglobulin

Increased Infusion Rate Cohort - Cohort 2

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

Group Type EXPERIMENTAL

CUTAQUIG

Intervention Type DRUG

Human normal immunoglobulin

Every Other Week Dosing Cohort - Cohort 3

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

Group Type EXPERIMENTAL

CUTAQUIG

Intervention Type DRUG

Human normal immunoglobulin

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

CUTAQUIG

Human normal immunoglobulin

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Age ≥2 years and ≤75 years.
2. 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.
3. 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.
4. 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.
5. 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).
6. 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.
7. For female patients of child-bearing potential, a negative result in a urine pregnancy test conducted at the Screening visit.
8. Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.

Exclusion Criteria

1. Evidence of active infection within 4 weeks of Screening or during the Screening Period.
2. 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.
3. Known history of adverse reactions to immunoglobulin A (IgA) in other products.
4. Body mass index (BMI) \>40 kg/m2 for patients entering Cohort 2 or Cohort 3. There are no BMI restrictions for Cohort 1.
5. Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product (such as Polysorbate 80).
6. Requirement of any routine premedication for IgG administration.
7. History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
8. Severe liver function impairment (aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] \>3 times above upper limit of normal).
9. Known protein-losing enteropathies or clinically significant proteinuria.
10. 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).
11. 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.
12. Treatment with immunosuppressive or immunomodulatory drugs (except Omalizumab).
13. Use of HYQVIA (Immune Globulin Infusion 10% \[Human\] with Recombinant Human Hyaluronidase) within 3 months prior to first CUTAQUIG infusion.
14. Live viral vaccination (such as measles, rubella, mumps, and varicella) within 2 months prior to first CUTAQUIG infusion.
15. 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.
16. Treatment with any investigational medicinal product within 3 months prior to first CUTAQUIG infusion. Note: Patients participating in Study SCGAM-03 will be allowed to enter this study without the 3-month waiting period for an Investigational Product. Patients receiving another SCIG product within 3 months prior to the first CUTAQUIG infusion may be considered for enrollment after Sponsor approval.
17. Presence of any condition that is likely to interfere with the evaluation of CUTAQUIG or satisfactory conduct of the trial.
18. Known or suspected to abuse alcohol, drugs, psychotropic agents, or other chemicals within the past 12 months prior to first CUTAQUIG infusion.
19. Known active or chronic hepatitis B, hepatitis C, or HIV infection. Past hepatitis B or hepatitis C infection that has been cured is allowed.
Minimum Eligible Age

2 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Octapharma

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Octapharma Research Site

Scottsdale, Arizona, United States

Site Status

Octapharma Research Site

Irvine, California, United States

Site Status

Octapharma Research Site

Santa Barbara, California, United States

Site Status

Octapharma Research Site

Centennial, Colorado, United States

Site Status

Octapharma Research Site

North Palm Beach, Florida, United States

Site Status

Octapharma Research Site

St. Petersburg, Florida, United States

Site Status

Octapharma Research Site

Albany, Georgia, United States

Site Status

Octapharma Research Site

Louisville, Kentucky, United States

Site Status

Octapharma Research Site

Chevy Chase, Maryland, United States

Site Status

Octapharma Research Site

St Louis, Missouri, United States

Site Status

Octapharma Research Site

Papillion, Nebraska, United States

Site Status

Octapharma Research Site

Asheville, North Carolina, United States

Site Status

Octapharma Research Site

Cincinnati, Ohio, United States

Site Status

Octapharma Research Site

Mayfield, Ohio, United States

Site Status

Octapharma Research Site

Toledo, Ohio, United States

Site Status

Octapharma Research Site

Pittsburgh, Pennsylvania, United States

Site Status

Octapharma Research Site

Dallas, Texas, United States

Site Status

Octapharma Research Site

Bellingham, Washington, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

SCGAM-06

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Phase 2/3 Study of IGSC, 20% in PIDD
NCT01218438 COMPLETED PHASE2/PHASE3
Immune Globulin Subcutaenous (Human), 20%
NCT01412385 COMPLETED PHASE2/PHASE3