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

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ClinicalTrials.gov Identifier: NCT03576469
Recruitment Status : Recruiting
First Posted : July 3, 2018
Last Update Posted : July 3, 2018
Sponsor:
Information provided by (Responsible Party):
IMMUNOe Research Centers

Brief Summary:
Patients receiving intravenous immunoglobulin (IVIG) therapy for primary immunodeficiency, specifically common variable immunodeficiency (CVID), 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.

Condition or disease Intervention/treatment Phase
CVI - Common Variable Immunodeficiency Biological: C1-esterase inhibitor [recombinant] (C1-INH-R) Phase 4

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 for CVID for experience ADRs post-infusion. CVID subjects who are currently receiving IVIG every 3 to 4 weeks for immunodeficiency 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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Single Group
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: A Single-site, Open-Label, Pilot Study to Evaluate the Benefit of RUCONEST® in Subjects With CVID Who Experience ADRs Related to IVIG Infusions
Actual Study Start Date : June 13, 2018
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : September 2019


Arm Intervention/treatment
Experimental: C1-esterase inhibitor [recombinant] (C1-INH-R)

Single-site, open-label arm to evaluate the benefit of C1-INH-R in subjects with CVID who experience ADRs related to IVIG. The study will have 2 periods:

  • 6 - 8 weeks - subjects will receive 2 infusions of IVIG every 3 - 4 weeks
  • 9 - 12 weeks - subjects will receive 3 infusions of C1-INH-R prior to IVIG infusion every 3 - 4 weeks
Biological: 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




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 years and older with a diagnosis of CVID experiencing ADRs related to IVIG infusions
  • Stable dose of IVIG for 3 months on a cycle of 21±3 or 28±3 days
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03576469


Contacts
Contact: Melinda Heffron, BA 303-771-9000 mheffron@immunoeresearch.com

Locations
United States, Colorado
IMMUNOe Research Centers Recruiting
Centennial, Colorado, United States, 80112
Contact: Melinda Heffron, BA    303-771-9000 ext 1251    mheffron@immunoeresearch.com   
Principal Investigator: Isaac Melamed, MD         
Sponsors and Collaborators
IMMUNOe Research Centers
Investigators
Principal Investigator: Isaac Melamed, MD IMMUNOe Research Centers

Responsible Party: IMMUNOe Research Centers
ClinicalTrials.gov Identifier: NCT03576469     History of Changes
Other Study ID Numbers: IIS201702-CVID
First Posted: July 3, 2018    Key Record Dates
Last Update Posted: July 3, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by IMMUNOe Research Centers:
Intravenous Immunoglobulin
C1-esterase inhibitor
Adverse drug reaction (ADR)

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Common Variable Immunodeficiency
Immune System Diseases
Complement C1s
Complement C1 Inhibitor Protein
Complement C1 Inactivator Proteins
Immunologic Factors
Physiological Effects of Drugs
Complement Inactivating Agents
Immunosuppressive Agents