CSL312 (garadacimab) in the prevention of hereditary angioedema attacks

Phase 1

• Conditions: Hereditary angioedema; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-000570-25-IT
• Lead Sponsor: CSL BEHRING GMBH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-04
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Capable of providing written informed consent
2. Male or female.
3. Aged = 12 years
4. Diagnosed with clinically confirmed C1-INH HAE
5. Experienced = 3 HAE attacks during the 3 months before Screening
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

Concomitant diagnosis of another form of angioedema such as idiopathic or acquired angioedema, recurrent angioedema associated with urticarial or hereditary angioedema type 3

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The main objective is to evaluate the efficacy of sucutaneous administration of CSL312 as prophylaxis to prevent hereditary angioedema attacks in subjects with hereditry angioedema<br>;Secondary Objective: The secondary objectives of the study are: <br>1. To characterize the clinical efficacy of subcutaneous CSL312 in the prophylactic treatment of hereditary angioedema<br>2. to evaluate the safety of subcutaneous CSL312 in the prophylactic treatment of hereditary angioedema<br>;Primary end point(s): The primary endpoint is the time-normalized number of HAE attacks during treatment from Day 1 through Day 182.;Timepoint(s) of evaluation of this end point: up to 6 months<br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - The reduction in the attack rate during the Treatment Period compared to the Run-in Period.<br>- The time-normalized number of HAE attacks requiring on-demand treatment.<br>- The time-normalized number of moderate and / or severe HAE attacks.<br>- The time-normalized number of HAE attacks at various time points during the treatment period.<br>- Subject Global Assessment of Response to Treatment (SGART).<br>- Adverse events (AEs).<br>- Adverse events of special interest (AESIs).<br>- Serious adverse events (SAEs).<br>- CSL312 induced anti-CSL312 antibodies.<br>- Clinically significant abnormalities in laboratory assessments;Timepoint(s) of evaluation of this end point: to 1: up to 6 months<br>to 2: 6 months, and first 3 months and second 3 months<br>to 3: 6 months, and first 3 months and second 3 months<br>to 4: First 3 months and second 3 months and 6 months<br>to 5: 6 months, and first 3 months and second 3 months<br>to 6: Up to 6 months<br>to 7 and 8: Up to 8 months<br>