Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients

NCT01359969 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: C1 12092011-000987-92
• Study Type: interventional
• Target: 57
• Locations: 10 countries
• Sites: 16

Brief Summary

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.

Conditions

Hereditary Angioedema

Keywords

Hereditary Angioedema; HAE; Angioedema; Recombinant C1 Inhibitor; rhC1INH; Pediatrics

Outcome Measures

Primary Outcomes (1)

• Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief.

  Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).

  The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.

Secondary Outcomes (1)

• Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations

  The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.

Study Arms (1)

Recombinant Human C1 Inhibitor

EXPERIMENTAL

Patients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.

Drug: rhC1INH

Interventions

rhC1INH DRUG

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).

Also known as: Ruconest

Recombinant Human C1 Inhibitor

Eligibility Criteria

• Age: 2 Years - 13 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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 Form (ICF)(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

You may not qualify if:

• A diagnosis of acquired C1INH deficiency (AAE)
• A medical history of allergy to rabbits or rabbit-derived products or positive anti-rabbit epithelium (dander) immunoglobuline E (IgE) test

Sponsors & Collaborators

• Pharming Technologies B.V.: lead

Study Sites (16)

Portland Clinical Research/AAIM Care, LLC

Portland, Oregon, 97015, United States

Location

UIA FN Plzen ( Institute of Immunology and Allergology), Faculty Hospital Plzen

Pilsen, Alej Svobody 80, 304 60 Plzen, Czechia

Location

University Hospital Motol, Institute of Immunology

Prague, V Úvalu 84, 150 06 Prague, Czechia

Location

Charité - Universitätsmedizin Berlin

Berlin, Charitéplatz 1, 10117, Germany

Location

Klinikum Rechts der Isar, Technical University Munich

Munich, Germany

Location

Heim Pál Gyermekkórház, II. számú Gyermek Belgyógyászati Osztály

Budapest, Madarász Utca 22-24, 1131, Hungary

Location

Bnei Zion Hospital

Haifa, Israel

Location

Souraski Medical Center

Tel Aviv, Israel

Location

Sheba Medical Center

Tel Litwinsky, Israel

Location

Hospital Luigi Sacco

Milan, Italy

Location

Azienda Ospedaliera Universitaria S. Giovanni di Dio e Ruggi d'Aragona

Salerno, 84131, Italy

Location

University Clinic Of Dermatology Skopje

Skopje, 1000, North Macedonia

Location

Pediatric Hospital

Krakow, Poland

Location

Pediatric Hospital

Lublin, Poland

Location

Mures County Clinical Hospital

Târgu Mureş, Romania

Location

Klinika detí a dorastu, Univerzitna nemocnica Martin

Martin, Kollárova 2, 036 59 Martin, Slovakia

Location

Related Publications (1)

• Reshef A, Grivcheva-Panovska V, Kessel A, Kivity S, Klimaszewska-Rembiasz M, Moldovan D, Farkas H, Gutova V, Fritz S, Relan A, Giannetti B, Magerl M. Recombinant human C1 esterase inhibitor treatment for hereditary angioedema attacks in children. Pediatr Allergy Immunol. 2019 Aug;30(5):562-568. doi: 10.1111/pai.13065. Epub 2019 May 29.

  PMID: 30993784 RESULT

MeSH Terms

Conditions

Angioedemas, Hereditary; Angioedema

Interventions

conestat alfa

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Results Point of Contact

• Title: Anurag Relan, MD, VP Clinical Research & Medical Affairs
• Organization: Pharming Technologies BV

a.relan@pharming.com

+31 715247400

Study Officials

• Anurag Relan, MD

  VP Clinical Research & Medical Affairs at Pharming

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2011
• First Posted: May 25, 2011
• Study Start: January 17, 2012
• Primary Completion: July 17, 2017
• Study Completion: July 17, 2017
• Last Updated: March 29, 2024
• Results First Posted: March 29, 2024

Record last verified: 2024-03

Locations

RO (1); US (1); HU (1); DE (2); CZ (2); IL (3); SL (1); PL (2); NO (1); IT (2)