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Trial record 75 of 9032 for:    Genetic Diseases, Inborn AND genetic disorder

A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)

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: NCT00851409
Recruitment Status : Completed
First Posted : February 26, 2009
Results First Posted : January 31, 2013
Last Update Posted : June 26, 2018
Sponsor:
Information provided by (Responsible Party):
Pharming Technologies B.V.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Genetic Disorders
Hereditary Angioedema
Intervention Drug: Recombinant Human C1 Inhibitor
Enrollment 25
Recruitment Details Patients with a history of frequent attacks, defined as "HAE" attacks occurring at least every two weeks were included in the study.
Pre-assignment Details  
Arm/Group Title Recombinant Human C1 Inhibitor
Hide Arm/Group Description

Weekly administration of 50 IU/kg recombinant human C1 inhibitor

Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Period Title: Overall Study
Started 25
Completed 25
Not Completed 0
Arm/Group Title Recombinant Human C1 Inhibitor
Hide Arm/Group Description

Weekly administration of 50 IU/kg recombinant human C1 inhibitor

Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Overall Number of Baseline Participants 25
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants
<=18 years
0
   0.0%
Between 18 and 65 years
25
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 25 participants
Female
20
  80.0%
Male
5
  20.0%
1.Primary Outcome
Title HAE Attacks/Week
Hide Description Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Recombinant Human C1 Inhibitor
Hide Arm/Group Description:
Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.
Overall Number of Participants Analyzed 25
Mean (95% Confidence Interval)
Unit of Measure: attacks/week
Historical
0.9
(0.51 to 1.30)
Prophylaxis
0.4
(0.27 to 0.56)
2.Secondary Outcome
Title The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.
Hide Description "PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4)
Time Frame 8 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Recombinant Human C1 Inhibitor
Hide Arm/Group Description:

Weekly administration of 50 IU/kg recombinant human C1 inhibitor

Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Overall Number of Participants Analyzed 25
Geometric Mean (95% Confidence Interval)
Unit of Measure: ratio
Functional C1 Inhibitor level
1.06
(0.98 to 1.14)
Antigenic C1 Inhibitor level
1.02
(0.91 to 1.15)
C4 level
1.00
(0.74 to 1.33)
Time Frame Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
Adverse Event Reporting Description All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
 
Arm/Group Title Recombinant Human C1 Inhibitor
Hide Arm/Group Description

Weekly administration of 50 IU/kg recombinant human C1 inhibitor

Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

All-Cause Mortality
Recombinant Human C1 Inhibitor
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Recombinant Human C1 Inhibitor
Affected / at Risk (%) # Events
Total   2/25 (8.00%)    
Infections and infestations   
Appendicitis  1  1/25 (4.00%)  1
Respiratory, thoracic and mediastinal disorders   
Laryngeal edema  1  1/25 (4.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (Unspecified)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
Recombinant Human C1 Inhibitor
Affected / at Risk (%) # Events
Total   2/25 (8.00%)    
Blood and lymphatic system disorders   
Anaemia-mild  1  1/25 (4.00%)  1
Anaemia-moderate  1  1/25 (4.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (Unspecified)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to its submission or disclosure. The sponsor may request to delete information identified by sponsor as confidential information prior to submitting such manuscript and/or abstract for publication. For a multi-center study, the investigator must wait (i) at least 24 months after the study is completed at all sites or (ii) until after the multi-center publication.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Affairs Department
Organization: Pharming Technologies BV
Phone: +31715247400
EMail: medicalinfo@pharming.com
Layout table for additonal information
Responsible Party: Pharming Technologies B.V.
ClinicalTrials.gov Identifier: NCT00851409     History of Changes
Other Study ID Numbers: C1 1207
First Submitted: February 25, 2009
First Posted: February 26, 2009
Results First Submitted: November 9, 2012
Results First Posted: January 31, 2013
Last Update Posted: June 26, 2018