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Completed

NCT Number: NCT03576469

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

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.

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Key information

Conditions

CVI - Common Variable Immunodeficiency Angioedema Angioedemas, Hereditary Cardiovascular Diseases Chemically-Induced Disorders Common Variable Immunodeficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Drug-Related Side Effects and Adverse Reactions Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

IMMUNOe Research Centers

Centennial, Colorado, 80112, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

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

Primary outcomes

  1. The change in Modified Fatigue Severity Scale (mFSS)

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated Rasch-built 7-item modified fatigue survey scale. Scale rated 0 - 3 (3 = agree, 0 = less agree)

  2. The change in Modified Fatigue Impact Scale (MFIS)

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated 24 item questionnaire measuring the impact of fatigue rated 0 - 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always)

  3. The change in Migraine Disability Assessment (MIDAS)

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Headache severity measurement of number of days affected after infusion

  4. The change in Headache Impact Scale (HIT-6)

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated measurement of the impact headaches have on the ability to function on the job, at school, at home and in social situations. 6 item questionnaire rated from never (6 points each), rarely (8 points each), sometimes (10 points each), very often (11 points each), always (13 points each). The higher number the more the impact.

  5. The change in Activities of Daily Living Sliding Scale

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated 10 point scale measuring level of activity with 1 being the worst (least) and 10 being the best (greatest)

  6. The change in Activities of Daily Living Questionnaire

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Calculates the number of days missed from work, school/daycare/activities, housework, and regular exercise

  7. The change in Energy Sliding Scale

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated10 point scale measuring energy level with 1 being the worst (Lowest) and 10 being the best (highest)

  8. The change in Infection Questionnaire

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated 7 item infection questionnaire measured from 1 to 10 with 1 being the least affected and 10 being the most affected.

  9. The change in Perceived Deficits Questionnaire - cognitive assessment

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Subject-rated 20 item questionnaire measuring memory, attention and concentration rated 0 to 4 (0 = never, 1 = rarely, 2 = sometimes, 3 = often, 4 = almost always) with the lower score being least impact.

  10. The change in 36 item short form survey (SF-36)

    Time frame: Measurement prior to infusion 3 (week 9 - 12) compared to end of study (week 19 - 25)

    Health Survey asks 36 questions to measure functional health and well-being from the patient's point of view.

  11. Change in the number of ADRs

    Time frame: Measured at each infusion (every 3 - 4 weeks)

    Adverse reactions to infusions

Secondary outcomes

  1. Change in levels of C1-INH pre- and post-infusion

    Time frame: Measurement at each infusion (every 3 - 4 weeks)

    Laboratory levels of C1-INH total and functional

Sponsors and collaborators

Lead sponsor

IMMUNOe Research Centers

Industry

Registry information

Official study title

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

Important dates

Study start
2018
Primary completion
2019
Study completion
2020
First posted
Jul 3, 2018
Registry last updated
Feb 15, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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