CSL312_3003 Safety and Pharmacokinetic study in Subjects 2 to 11 Years of Age with Hereditary Angioedema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

CSL Behring LLC

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

10 May 2024 → 19 Nov 2025

Decision date (initial)

2023-10-17

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2022-502386-13-00

ClinicalTrials.gov

NCT05819775

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

Evaluate the safety and pharmacokinetics of SC administration of CSL312 in the prophylactic treatment of pediatric subjects with C1-INH HAE

Secondary objectives 1

1. Evaluate efficacy, pharmacodynamics, and safety of CSL312 in the prophylactic treatment of pediatric subjects with C1-INH HAE

Conditions and MedDRA coding

Hereditary angioedema (HAE)

Study design 1 period

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002726-PIP01-19

Plan to share IPD

Yes

IPD plan description

CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers.  For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at [email protected].

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Male or female
2. Aged 2 to 11 years, inclusive, with body weight ≥ 10th percentile based on age
3. Diagnosed with clinically confirmed C1-INH HAE
4. Experienced ≥ 2 HAE attacks during the 6 months before Screening

Exclusion criteria 6

1. Concomitant diagnosis of another form of angioedema, such as idiopathic or acquired angioedema, recurrent angioedema associated with urticaria, or HAE type III
2. Use of C1-INH products, androgens, antifibrinolytics, approved or future approved medications, or other small molecule medications for routine prophylaxis against HAE attacks
3. Participation in another interventional clinical study
4. Having laboratory clinical abnormalities assessed as clinically significant by the investigator in results of hematology or chemistry assessments performed during Screening
5. Currently receiving a therapy not permitted during the study
6. Being pregnant or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 8

1. Number of subjects with treatment emergent adverse events (TEAEs)
2. Percent of subjects with TEAEs
3. Number of TEAEs
4. TEAE rates per injection
5. TEAE rates per subject year
6. Maximum concentration (Cmax) of CSL312 at steady-state
7. Trough concentration (Ctrough) of CSL312 at steady-state
8. Time to maximum concentration (Tmax) of CSL312 at steady-state

Secondary endpoints 22

1. Time-normalized number of HAE attacks per month and per year
2. Time-normalized number of HAE attacks treated with on-demand treatment per month and per year
3. Time-normalized number of moderate and / or severe HAE attacks per month and per year
4. Percentage reduction in the time-normalized number of HAE attacks
5. The number of subjects experiencing at least ≥ 50%, ≥ 70%, ≥ 90%, or equal to 100% (attack-free) reduction in the time-normalized number of HAE attacks
6. Number of subjects with serious adverse events (SAEs)
7. Percent of subjects with SAEs
8. Number of subjects experiencing death
9. Percent of subjects experiencing death
10. Number of subjects with related TEAEs
11. Percent of subjects with related TEAEs
12. Number of subjects with TEAEs leading to study discontinuation
13. Percent of subjects with TEAEs leading to study discontinuation
14. Number of subjects with TEAEs by severity
15. Percent of subjects with TEAEs by severity
16. Number of subjects with Anti-CSL312 antibodies
17. Percent of subjects with Anti-CSL312 antibodies
18. Number of subjects with adverse events of special interest (AESIs)
19. Percent of subjects with AESIs
20. FXIIa-mediated kallikrein activity
21. Number of subjects with laboratory findings reported as AEs
22. Percent of subjects with laboratory findings reported as AEs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

CSL Behring LLC

Sponsor organisation

CSL Behring LLC

Address

1020 1st Avenue

City

King Of Prussia

Postcode

19406-1310

Country

United States

Scientific contact point

Organisation

CSL Behring LLC

Contact name

Trial Registration Coordinator

Public contact point

Organisation

CSL Behring LLC

Contact name

Trial Registration Coordinator

Third parties 12

Locations

2 EU/EEA countries · 8 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 105 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications