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

NCT ID: NCT03576469

Last Updated: 2021-02-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-06-13
• Study Completion Date: 2020-03-01

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.

Conditions

CVI - Common Variable Immunodeficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

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

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

Group Type: EXPERIMENTAL

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

Intervention Type: BIOLOGICAL

C1-INH-R is FDA approved and indicated for the treatment of acute attacks of angioedema in adolescent and adult patients with Hereditary Angioedema (HAE) as a replacement for low levels of C1-esterase inhibitor or low function of C1-esterase inhibitor

Interventions

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

C1-INH-R is FDA approved and indicated for the treatment of acute attacks of angioedema in adolescent and adult patients with Hereditary Angioedema (HAE) as a replacement for low levels of C1-esterase inhibitor or low function of C1-esterase inhibitor

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

IMMUNOe Research Centers

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Isaac Melamed, MD

Role: PRINCIPAL_INVESTIGATOR

IMMUNOe Research Centers

Locations

IMMUNOe Research Centers

Centennial, Colorado, United States

Countries

United States

References

Melamed IR, Miranda H, Heffron M, Harper JR. Recombinant Human C1 Esterase Inhibitor for the Management of Adverse Events Related to Intravenous Immunoglobulin Infusion in Patients With Common Variable Immunodeficiency or Polyneuropathy: A Pilot Open-Label Study. Front Immunol. 2021 Mar 2;12:632744. doi: 10.3389/fimmu.2021.632744. eCollection 2021.

Reference Type: DERIVED

PMID: 33737935 (View on PubMed)

Other Identifiers

IIS201702-CVID

Identifier Type: -

Identifier Source: org_study_id