History of Changes for Study: NCT01397864

C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks

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Study Record Versions

Version	Submitted Date	Changes
1	July 19, 2011	None (earliest Version on record)
2	February 15, 2012	Sponsor/Collaborators and Study Status
3	November 9, 2012	Contacts/Locations and Study Status
4	July 10, 2013	Study Status
5	November 15, 2013	Study Status and Study Design
6	July 8, 2014	Contacts/Locations and Study Status
7	January 7, 2015	Study Status
8	July 28, 2015	Study Status
9	November 6, 2015	Study Status
10	February 15, 2016	Contacts/Locations and Study Status
11	October 14, 2016	Study Status
12	April 3, 2018	Study Status and Contacts/Locations
13	March 19, 2019	Outcome Measures, Study Status, Contacts/Locations and Study Description
14	June 18, 2019	Contacts/Locations and Study Status
15	March 4, 2020	Contacts/Locations and Study Status
16	September 15, 2020	Study Status and Contacts/Locations
17	February 6, 2024	Study Status and Contacts/Locations

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	September 15, 2020	February 6, 2024

Study Identification


Unique Protocol ID:	C1 1412	C1 1412
Brief Title:	C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks	C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
Official Title:	C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks	C1 Inhibitor Treatment Registry to Assess the Safety and Immunological Profile of Ruconest in the Treatment of HAE Attacks
Secondary IDs:

Study Status


Record Verification:	September 2020	February 2024
Overall Status:	Recruiting	Recruiting
Study Start:	July 2011	July 2011
Primary Completion:	June 2021 [Anticipated]	January 2025 [Anticipated]
Study Completion:	December 2021 [Anticipated]	January 2025 [Anticipated]

First Submitted:	July 18, 2011	July 18, 2011
First Submitted that Met QC Criteria:	July 19, 2011	July 19, 2011
First Posted:	July 20, 2011 [Estimate]	July 20, 2011 [Estimate]

Last Update Submitted that Met QC Criteria:	September 15, 2020	February 6, 2024
Last Update Posted:	September 16, 2020 [Actual]	February 7, 2024 [Actual]

Sponsor/Collaborators


Sponsor:	Pharming Technologies B.V.	Pharming Technologies B.V.
Responsible Party:	Sponsor	Sponsor
Collaborators:

Oversight


U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:	No	No

Study Description


Brief Summary:	This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.	This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.
Detailed Description:	see below	see below

Conditions


Conditions:	Hereditary Angioedema	Hereditary Angioedema
Keywords:	Hereditary Angioedema HAE Angioedema Recombinant C1 Inhibitor rhC1INH registry	Hereditary Angioedema HAE Angioedema Recombinant C1 Inhibitor rhC1INH registry

Study Design


Study Type:	Observational	Observational
Observational Study Model:	Cohort	Cohort
Time Perspective:	Prospective	Prospective
Biospecimen Retention:
Biospecimen Description:
Enrollment:	300 [Anticipated]	300 [Anticipated]
Number of Groups/Cohorts	1	1

Groups and Interventions

Groups/Cohorts	Interventions
Hereditary Angioedema	Drug: rhC1INH or pdC1INH C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (Ruconest) Other Names: Ruconest

Outcome Measures


Primary Outcome Measures:
1.	The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks [ Time Frame: December 2019 ]	The primary objective is to observe the adverse event profile and insufficient efficacy, following single and repeated treatment with Ruconest or pdC1INH of acute angioedema attacks [ Time Frame: December 2019 ]
Secondary Outcome Measures:
1.	To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies) [ Time Frame: December 2019 ]	To assess the immunological profile of Ruconest (for suspected hypersensitivity or suspected neutralizing antibodies) [ Time Frame: December 2019 ]

Eligibility


Study Population:	The aim is to recruit 300 patients treated with Ruconest. Additionally, the study will continue until 100 patients have been exposed to Ruconest for at least 3 attacks. Enrolment in the pdC1INH arm will be unrestricted.	The aim is to recruit 300 patients treated with Ruconest. Additionally, the study will continue until 100 patients have been exposed to Ruconest for at least 3 attacks. Enrolment in the pdC1INH arm will be unrestricted.
Sampling Method:	Probability Sample	Probability Sample
Minimum Age:
Maximum Age:
Sex:	All	All
Gender Based:
Accepts Healthy Volunteers:	No	No
Criteria:	Inclusion Criteria: Decision to treat the HAE patient with C1 inhibitor (either Ruconest or pdC1INH) Patients must give written informed consent Exclusion Criteria: A diagnosis of acquired C1INH deficiency (AAE)	Inclusion Criteria: Decision to treat the HAE patient with C1 inhibitor (either Ruconest or pdC1INH) Patients must give written informed consent Exclusion Criteria: A diagnosis of acquired C1INH deficiency (AAE)

Contacts/Locations


Central Contact Person:	Miranda Boshuizen Telephone: +31 71 5247 400 Email: m.boshuizen@pharming.com	Miranda Boshuizen Telephone: +31 71 5247 400 Email: m.boshuizen@pharming.com
Study Officials:	Anurag Relan, MD Study Director Pharming Technologies BV	Anurag Relan, MD Study Director Pharming Technologies BV
Locations:	Bulgaria	Bulgaria
	UMHAT Alexandrovska EAD [Recruiting] Sofia, Bulgaria Contact:Contact: Maria Staevska, Prof.	UMHAT Alexandrovska EAD [Recruiting] Sofia, Bulgaria Contact:Contact: Maria Staevska, Prof.
	Croatia	Croatia
	KBC Split [Recruiting] Split, Croatia Contact:Contact: Drasko Cikojevic, Dr.	KBC Split [Recruiting] Split, Croatia Contact:Contact: Drasko Cikojevic, Dr.
	CHC Sestre milosrdnice [Recruiting] Zagreb, Croatia Contact:Contact: Renata Vrsalovic, Dr.	CHC Sestre milosrdnice [Recruiting] Zagreb, Croatia Contact:Contact: Renata Vrsalovic, Dr.
	General Hospital Sibenik [Recruiting] Šibenik, Croatia Contact:Contact: Ljerka Karadza-Lapic, Dr.	General Hospital Sibenik [Recruiting] Šibenik, Croatia Contact:Contact: Ljerka Karadza-Lapic, Dr.
	Czechia	Czechia
	Faculty Hospital by St. Anna Brno [Recruiting] Brno, Czechia Contact:Contact: Roman Hakl, Dr.	Faculty Hospital by St. Anna Brno [Recruiting] Brno, Czechia Contact:Contact: Roman Hakl, Dr.
	France	France
	Hospital A Michallon, CHU Grenoble [Recruiting] La Tronche, France Contact:Contact: Laurence Bouillet, Prof. Dr.	Hospital A Michallon, CHU Grenoble [Recruiting] La Tronche, France Contact:Contact: Laurence Bouillet, Prof. Dr.
	Germany	Germany
	Charite Universitatsmedizin Berlin [Recruiting] Berlin, Germany Contact:Contact: Marcus Maurer, Prof. Contact:Contact: Markus Magerl, Dr.	Charite Universitatsmedizin Berlin [Recruiting] Berlin, Germany Contact:Contact: Marcus Maurer, Prof. Contact:Contact: Markus Magerl, Dr.
	Hungary	Hungary
	Semmelweis University, 3rd Department of Internal Medicine [Recruiting] Budapest, Hungary Contact:Contact: Henriette Farkas, Prof.	Semmelweis University, 3rd Department of Internal Medicine [Recruiting] Budapest, Hungary Contact:Contact: Henriette Farkas, Prof.
	Italy	Italy
	Ospedale Luigi Sacco [Recruiting] Milan, Italy Contact:Contact: Andrea Zanichelli, Dr.	Ospedale Luigi Sacco [Recruiting] Milan, Italy Contact:Contact: Andrea Zanichelli, Dr.
	North Macedonia	North Macedonia
	PHI University Clinic of Dermatology [Recruiting] Skopje, North Macedonia Contact:Contact: Vesna Grivcheva-Panovska, Prof.	PHI University Clinic of Dermatology [Recruiting] Skopje, North Macedonia Contact:Contact: Vesna Grivcheva-Panovska, Prof.
	Norway	Norway
	Alesund Hospital [Recruiting] Alesund, Norway Contact:Contact: Robert Brudevold, Dr.	Alesund Hospital [Completed] Alesund, Norway
	Stavanger University Hospital [Recruiting] Stavanger, Norway Contact:Contact: Ellen Pritzier, Dr.	Stavanger University Hospital [Completed] Stavanger, Norway
	Poland	Poland
	University Hospital Krakow [Completed] Krakow, Poland	University Hospital Krakow [Completed] Krakow, Poland
	Slovakia	Slovakia
	Bratislava University Hospital [Recruiting] Bratislava, Slovakia Contact:Contact: Katarina Hrubiskova, Dr.	Bratislava University Hospital [Recruiting] Bratislava, Slovakia Contact:Contact: Katarina Hrubiskova, Dr.
	University Hospital Martin [Recruiting] Martin, Slovakia Contact:Contact: Milos Jesenak, Prof.	University Hospital Martin [Recruiting] Martin, Slovakia Contact:Contact: Milos Jesenak, Prof.
	Slovenia	Slovenia
	University Clinic Golnik [Recruiting] Golnik, Slovenia Contact:Contact: Mitja Kosnik, Prof.	University Clinic Golnik [Completed] Golnik, Slovenia
	Sweden	Sweden
	Ryhof County Hospital [Recruiting] Jonkoping, Sweden Contact:Contact: Janne Bjorkander, Prof.	Ryhof County Hospital [Completed] Jonkoping, Sweden

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