A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects Who Experience ADRs Related to IVIG Infusions

IMMUNOe Research Centers1 site in 1 country20 target enrollmentStarted: June 13, 2018Last updated: February 15, 2021

Conditions

Interventions

Trial Snapshot

Phase: Phase 4
Status: Completed
Sponsor: IMMUNOe Research Centers
Enrollment: 20
Locations: 1
Primary Endpoint: The change in Headache Impact Scale (HIT-6)

Study Overview Study Design Eligibility Criteria Arms & Interventions Outcomes Investigators Sites Records & Identifiers Similar Trials

Study Overview

Brief Summary

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.

Detailed 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.

Study Design

Study Type: Interventional
Allocation: Na
Intervention Model: Single Group
Primary Purpose: Prevention
Masking: None

Eligibility Criteria

Ages: 18 Years to — (Adult, Older Adult)
Sex: All
Accepts Healthy Volunteers: No

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

Arms & Interventions

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

Experimental

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

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