A Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions

IMMUNOe Research Centers

Status and phase: Completed; Phase 4

Conditions: CVI - Common Variable Immunodeficiency

Treatments: Biological: C1-esterase inhibitor [recombinant] (C1-INH-R)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03576469

IIS201702-CVID

Details and patient eligibility

About

Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency and neurologic conditions may experience adverse drug reactions (ADRs). The mechanism of the ADR is unknown. Currently, the standard practice for these patients is to change from IV to subcutaneous IG (SCIG) but because of the need of immunomodulation or patient preference, SCIG may not be an option. Data has shown that some levels of complement decrease from pre- to post-infusion of IVIG. This study is to determine if replacing this complement protein may ameliorate ADRs.

Full description

This is a single-site, pilot study conducted in the US to determine the benefit of human C1-esterase inhibitor [recombinant] (C1-INH-R) therapy to ameliorate ADRs in subjects receiving IVIG therapy experience ADRs post-infusion. Subjects who are currently receiving IVIG for immunodeficiency or neurologic conditions and experience ADRs will be enrolled.

In part 1, subjects will continue to receive IVIG for two infusions. Complement proteins will be measured pre- and post-infusion. Quality of life and other questionnaires will be administered.

In part 2, subjects will receive C1-INH-R infusion prior to receiving IVIG infusion for three infusions. Complement proteins will be measured pre- and post- infusion. Quality of life and other questionnaires will be administered.

Enrollment: 20 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age 18 years and older experiencing ADRs related to IVIG infusions
• Stable dose of IVIG for 3 months
• Willing to comply with all aspects of the protocol, including blood draws
• Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception. Acceptable forms of contraception are defined as those with a failure rate < 1% when properly applied and include: a combination oral pill, some intra-uterine devices, and a sterilized partner in a stable relationship. Female patients must not be pregnant, planning to become pregnant, or be actively breastfeeding through the entire study period.

Exclusion criteria

• Receiving treatment for HAE, either prophylactic or acute therapy
• Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
• Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant.
• Patients who, in the investigator's opinion, might not be suitable for the trial

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

C1-esterase inhibitor [recombinant] (C1-INH-R)

Experimental group

Description:

Single-site, open-label arm to evaluate the benefit of C1-INH-R in subjects on IVIG therapy who experience ADRs. The study will have 2 periods: * 6 - 8 weeks - subjects will receive 2 infusions of IVIG * 9 - 12 weeks - subjects will receive 3 infusions of C1-INH-R prior to IVIG infusion

Treatment:

Biological: C1-esterase inhibitor [recombinant] (C1-INH-R)