Konfident-Kid

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Kalvista Pharmaceuticals Limited

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

24 Oct 2024 → 16 Jan 2026

Decision date (initial)

2024-09-05

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

KalVista Pharmaceuticals Ltd

External identifiers

EU CT number

2023-507591-44-00

ClinicalTrials.gov

NCT06467084

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Pharmacokinetic, Safety

To evaluate the safety and tolerability of sebetralstat in pediatric patients aged 2 to <12 years of age with HAE Type I or II.

Secondary objectives 2

1. To evaluate the pharmacokinetics (PK) of sebetralstat in pediatric patients with HAE Type I or II.
2. To evaluate the clinical efficacy of sebetralstat for the on-demand treatment of HAE attacks in pediatric patients with HAE Type I or II.

Conditions and MedDRA coding

Hereditary Angioedema Type I or II

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002723-PIP01-19

Plan to share IPD

No

IPD plan description

Data will not be shared until all global regulatory filings are complete.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male or female patients 2 to 11 years of age.
2. Confirmed diagnosis of HAE Type I or II at any time in the medical history.
3. For patients ≥20 kg at screening, patient has had at least 1 documented HAE attack in the last year prior to screening.
4. Caregiver, as assessed by the Investigator, must be able to appropriately store and administer IMP and be able to read, understand, and complete the diary.
5. Investigator believes that the patient and caregiver are willing and able to adhere to all protocol requirements.
6. Parent or LAR provides signed informed consent and patient provides assent (when applicable).

Exclusion criteria 9

1. Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH, idiopathic angioedema, or angioedema associated with urticaria.
2. A clinically significant history of poor response to bradykinin receptor 2 (BR2) blocker, C1-INH therapy, or plasma kallikrein inhibitor therapy for the management of HAE, in the opinion of the Investigator.
3. Patient weighs <9.5 kg.
4. Use of angiotensin-converting enzyme (ECA) inhibitors after the Screening Visit.
5. Any estrogen-containing medications with systemic absorption (such as oral contraceptives including ethinylestradiol or hormonal replacement therapy) within 7 days prior to the Screening Visit.
6. Patients who require sustained use of strong cytochrome CYP3A4 inhibitors or inducers or moderate CYP3A4 inducers.
7. Any clinically significant comorbidity or systemic dysfunction, which in the opinion of the Investigator, would jeopardize the safety of the patient by participating in the trial.
8. Known hypersensitivity to sebetralstat or to any of the excipients.
9. Participation in any interventional investigational clinical trial within 4 weeks of the last dosing of investigational drug prior to the Screening Visit.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The proportion of pediatric patients with HAE Types I or II who take any sebetralstat dose, who experience any AE(s) (including fatal AEs) during the study, irrespective of uses of other medications and sebetralstat discontinuations for any reason.

Secondary endpoints 4

1. CaGI-C: Time to beginning of symptom relief defined as at least “a little better” (2 time points in a row) within 12 hours of the first IMP administration.
2. CaGI-S: Time to first incidence of decrease from baseline (2 time points in a row) within 12 hours of the first IMP administration.
3. CaGI-S: Time to HAE attack resolution defined as “none” within 24 hours of the first IMP Administration
4. Time to use of conventional on-demand treatment within 12 hours of the first IMP administration.

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Kalvista Pharmaceuticals Limited

Sponsor organisation

Kalvista Pharmaceuticals Limited

Address

Porton Science Park, Bybrook Road, Porton Down Bybrook Road Porton Down

City

Salisbury

Postcode

SP4 0BF

Country

United Kingdom

Scientific contact point

Organisation

Kalvista Pharmaceuticals Limited

Contact name

KalVista Clinical

Public contact point

Organisation

Kalvista Pharmaceuticals Limited

Contact name

KalVista Clinical

Third parties 7

Locations

3 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

Documents 137 files

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

Application history

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