Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients
This study is currently recruiting participants.
Verified February 2013 by Pharming Technologies B.V.
Sponsor:
Pharming Technologies B.V.
Information provided by (Responsible Party):
Pharming Technologies B.V.
ClinicalTrials.gov Identifier:
NCT01359969
First received: May 19, 2011
Last updated: February 20, 2013
Last verified: February 2013
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Purpose
This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.
| Condition | Intervention | Phase |
|---|---|---|
|
Hereditary Angioedema |
Drug: rhC1INH |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Open-label, Phase II, Single Arm Study to Evaluate the Safety, Immunogenicity, Pharmacokinetics and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Pediatric Patients With Hereditary Angioedema, From 2 up to and Including 13 Years of Age |
Resource links provided by NLM:
Genetics Home Reference related topics:
hereditary angioedema
Drug Information available for:
Conestat alfa
U.S. FDA Resources
Further study details as provided by Pharming Technologies B.V.:
Primary Outcome Measures:
- The primary objective is the assessment of safety and tolerability (adverse events, physical examination, vital signs, immunological and routine laboratory analyses et cetera) [ Time Frame: 90 Days ] [ Designated as safety issue: Yes ]
- Immunogenicity by assessing antibodies against recombinant human C1INH (IgG and IgM) anti-rhC1INH) [ Time Frame: 90 Days ] [ Designated as safety issue: Yes ]
- Immunogenicity by assessing antibodies against host related impurities (anti-HRI) [ Time Frame: 90 Days ] [ Designated as safety issue: Yes ]
- Immunogenicity by assessing IgE antibodies against rabbit epithelium [ Time Frame: 28 Days ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Beginning of relief [ Time Frame: 24 Hours ] [ Designated as safety issue: No ]Time to beginning of relief assessed by using the overall severity visual analog scale(VAS), defined as the first time point with a decrease of at least 20 mm with respect to baseline at any eligible location, with persistence at the next time point
- Minimal symptoms [ Time Frame: 24 Hours ] [ Designated as safety issue: No ]Time to minimal symptoms assessed by using the overall severity VAS, defined as the first time point at which the overall severity VAS falls below 20 mm for each assessed location
- Complete resolution [ Time Frame: 28 Days (diary recording) ] [ Designated as safety issue: No ]Patient-recorded time at which all angioedema symptoms at all locations have resolved
- Pharmacokinetic parameters for first attack [ Time Frame: 4 Hours ] [ Designated as safety issue: No ]C1INH activity
- Pharmacodynamic parameters for first attack [ Time Frame: 4 Hours ] [ Designated as safety issue: No ]C4 levels
| Estimated Enrollment: | 20 |
| Study Start Date: | December 2011 |
| Estimated Study Completion Date: | January 2014 |
| Estimated Primary Completion Date: | January 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Recombinant Human C1 Inhibitor |
Drug: rhC1INH
Patients up to 84 kg will receive one i.v. injection of Ruconest at a dose of 50 U/kg. The reconstituted solution should be administered as a slow i.v. injection over approximately 5 minutes. Patients of 84 kg body weight or greater will receive one i.v. injection of Ruconest at the dose of 4200 U (2 vials).
Other Name: Ruconest
|
Eligibility| Ages Eligible for Study: | 2 Years to 13 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- From 2 up to and including 13 years of age
- Clinical and laboratory confirmed diagnosis of HAE (baseline C1INH activity <50% of normal)
- Signed written informed consent (parental permission) signed by the legal guardian(s)
- Clinical symptoms of an acute HAE attack
- Onset of eligible symptoms within 5 hours from the moment at which medical evaluation to determine eligibility has occurred
- Attack severity moderate or greater, as rated by the investigator
Exclusion Criteria:
- A diagnosis of acquired C1INH deficiency (AAE)
- A medical history of allergy to rabbits or rabbit-derived products or positive anti-rabbit epithelium (dander) IgE test
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01359969
Contacts
| Contact: Annemarie Kleefstra | +31 71 5247 400 | a.kleefstra@pharming.com |
| Contact: Anurag Relan, MD | a.relan@pharming.com |
Locations
| Germany | |
| Charité Hospital | Recruiting |
| Berlin, Germany | |
| University Hospital Frankfurt | Recruiting |
| Frankfurt, Germany | |
| Klinikum Rechts der Isar, Technical University Munich | Recruiting |
| Munich, Germany | |
| Israel | |
| Bnei Zion Hospital | Recruiting |
| Haifa, Israel | |
| Sheba Medical Center | Recruiting |
| Tel Hashomer, Israel | |
| Souraski Medical Center | Recruiting |
| Tel-Aviv, Israel | |
| Italy | |
| Hospital Luigi Sacco | Recruiting |
| Milan, Italy | |
| Poland | |
| Pediatric Hospital | Recruiting |
| Krakow, Poland | |
| Pediatric Hospital | Recruiting |
| Lublin, Poland | |
| Romania | |
| Mures County Clinical Hospital | Recruiting |
| Targu Mures, Romania | |
Sponsors and Collaborators
Pharming Technologies B.V.
More Information
No publications provided
| Responsible Party: | Pharming Technologies B.V. |
| ClinicalTrials.gov Identifier: | NCT01359969 History of Changes |
| Other Study ID Numbers: | C1 1209, 2011-000987-92 |
| Study First Received: | May 19, 2011 |
| Last Updated: | February 20, 2013 |
| Health Authority: | Italy: The Italian Medicines Agency Germany: Federal Institute for Drugs and Medical Devices Poland: Ministry of Health Romania: National Medicines Agency Israel: Ministry of Health |
Keywords provided by Pharming Technologies B.V.:
|
Hereditary Angioedema HAE Angioedema |
Recombinant C1 Inhibitor rhC1INH Pediatrics |
Additional relevant MeSH terms:
|
Angioedema Angioedemas, Hereditary Vascular Diseases Cardiovascular Diseases Urticaria Skin Diseases, Vascular Skin Diseases Hypersensitivity, Immediate Hypersensitivity |
Immune System Diseases Genetic Diseases, Inborn Complement C1 Inhibitor Protein Complement Inactivating Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 18, 2013