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A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02247739
Recruitment Status : Completed
First Posted : September 25, 2014
Results First Posted : December 8, 2017
Last Update Posted : December 8, 2017
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Tracking Information
First Submitted Date  ICMJE September 16, 2014
First Posted Date  ICMJE September 25, 2014
Results First Submitted Date  ICMJE July 26, 2017
Results First Posted Date  ICMJE December 8, 2017
Last Update Posted Date December 8, 2017
Study Start Date  ICMJE December 2014
Actual Primary Completion Date May 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 7, 2017)
Number of HAE Attacks [ Time Frame: 28 days ]
Average number of HAE attacks normalized to a 28 day period
Original Primary Outcome Measures  ICMJE
 (submitted: September 19, 2014)
Number of HAE Attacks [ Time Frame: 28 days ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 7, 2017)
  • Number of Participants With Adverse Events [ Time Frame: 20 weeks ]
    Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
  • Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks [ Time Frame: 28 days ]
    Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Original Secondary Outcome Measures  ICMJE
 (submitted: September 19, 2014)
Adverse events [ Time Frame: 20 weeks ]
Current Other Pre-specified Outcome Measures
 (submitted: November 7, 2017)
Immunogenicity [ Time Frame: 20 weeks ]
Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies
Original Other Pre-specified Outcome Measures
 (submitted: September 19, 2014)
Immunogenicity [ Time Frame: 20 weeks ]
anti-C1INH and anti-host related impurities (HRI) antibodies
 
Descriptive Information
Brief Title  ICMJE A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
Official Title  ICMJE A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, 3-Period Crossover Study to Evaluate the Efficacy and Safety of Recombinant Human C1 Inhibitor in the Prophylaxis of Angioedema Attacks in Patients With Hereditary Angioedema (HAE)
Brief Summary

Primary Objective:

To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE

Secondary Objective:

To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE

Detailed Description

Study Design:

This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.

Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Condition  ICMJE Hereditary Angioedema
Intervention  ICMJE
  • Biological: Recombinant human C1 inhibitor
    Other Names:
    • rhC1INH
    • Ruconest
    • Conestat alfa
  • Other: Placebo
    Other Name: Saline
Study Arms  ICMJE
  • Experimental: rhC1INH twice weekly
    rhC1INH administered twice weekly
    Intervention: Biological: Recombinant human C1 inhibitor
  • Experimental: rhC1INH once weekly
    rhC1INH administered once weekly
    Intervention: Biological: Recombinant human C1 inhibitor
  • Placebo Comparator: Placebo (Saline) twice weekly
    Placebo (Saline) administered twice weekly
    Intervention: Other: Placebo
Publications * Riedl MA, Grivcheva-Panovska V, Moldovan D, Baker J, Yang WH, Giannetti BM, Reshef A, Andrejevic S, Lockey RF, Hakl R, Kivity S, Harper JR, Relan A, Cicardi M. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595-1602. doi: 10.1016/S0140-6736(17)31963-3. Epub 2017 Jul 25.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 15, 2016)
32
Original Estimated Enrollment  ICMJE
 (submitted: September 19, 2014)
30
Actual Study Completion Date  ICMJE September 2016
Actual Primary Completion Date May 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Patients fulfilling the following criteria at Screening are eligible for participation in the study:

  1. Age 13 years or older
  2. Laboratory confirmed diagnosis of HAE
  3. A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
  4. Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
  5. Provided written informed consent (and written assent for minors)
  6. Willingness and ability to comply with all protocol procedures

Exclusion Criteria:

Patients who meet any of the following criteria at Screening are to be excluded from study participation:

  1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
  2. Diagnosis of acquired angioedema (AAE)
  3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
  4. Treatment with any investigational drug in the past 30 days
  5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
  6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Czechia,   Italy,   Macedonia, The Former Yugoslav Republic of,   Romania,   Serbia,   United States
Removed Location Countries Czech Republic,   Netherlands
 
Administrative Information
NCT Number  ICMJE NCT02247739
Other Study ID Numbers  ICMJE C1 3201
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Pharming Technologies B.V.
Study Sponsor  ICMJE Pharming Technologies B.V.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Pharming Technologies B.V.
Verification Date November 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP