Cangrelor safety and pharmacodynamics in paediatric subjects with congenital heart disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Chiesi Farmaceutici S.p.A.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Decision date (initial)

2026-06-08

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Safety

To evaluate the safety profile of the recommended dosing regimen of Cangrelor in the paediatric population.

Conditions and MedDRA coding

Congenital Heart Disease

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001348-PIP01-12

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Males and females aged from birth to less than 18 years
2. Diagnosis of CHD, including, but not limited to, patent ductus arteriosus, coarctation of aorta, atrial septal defect, ventricular septal defects, valvular stenosis
3. Expected to undergo a diagnostic and/or therapeutic percutaneous vascular procedure
4. Female subjects fulfilling one of the following criteria: • Women of non-childbearing potential (WONCBP), defined as physiologically incapable of becoming pregnant; • Women of childbearing potential (WOCBP) fulfilling one of the following criteria: o Sexually active: they and/or their partner must be willing to use a highly effective birth control method from the signature of the informed consent and until the end of the follow-up period (Day 4/discharge) o Non-sexually active (contraception is not required in this case)
5. Male subjects fulfilling one of the following criteria: • Non-fertile male subjects (contraception is not required in this case); • Fertile male subjects without a female partner (contraception is not required in this case); • Fertile male subjects with any WOCBP partner: they must be willing to use male condom from the signature of the informed consent and until the end of the follow-up period (Day 4/discharge); • Fertile male subjects with WONCBP partner (contraception is not required in this case).

Exclusion criteria 5

1. Corrected age of <37 weeks at screening
2. History of intracerebral bleeding or cerebral arteriovenous malformation; or evidence of any prior bleeding with neurological deficit
3. Gastrointestinal or genitourinary bleeding within the last 2 weeks prior to screening, excluding normal menstrual cycles
4. Platelet count less than 150,000 cells/µL or higher than 450,000 cells/µL at screening
5. Known allergy to Cangrelor or known sensitivity to any component of Cangrelor

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 8

1. Percentage of subjects with major, clinically relevant non-major (CRNM) or minor bleeding (as defined by the Scientific and Standardisation Subcommittee [SSC] on Paediatric and Neonatal Thrombosis and Haemostasis from the International Society on Thrombosis and Haemostasis [ISTH]) occurring within 72 hours after the start of Cangrelor infusion
2. Percentage of subjects requiring hemoderivative transfusion related to Cangrelor infusion
3. Percentage of subjects with new onset of dyspnoea, wheezing or respiratory distress, or experiencing worsening of any pre-treatment dyspnoea, wheezing or respiratory distress after the start of Cangrelor infusion
4. Vital signs (including HR, SBP and DBP)
5. Laboratory parameters (including haematology, blood chemistry and urinalysis)
6. Percentage of subjects with an increase in creatinine >2 times the pre-infusion value
7. Percentage of subjects experiencing an AE of decrease in urine output considered related to Cangrelor
8. Treatment-emergent adverse events (TEAEs), adverse drug reactions (ADRs), serious adverse events, severe TEAEs and TEAEs leading to study discontinuation

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Chiesi Farmaceutici S.p.A.

Sponsor organisation

Chiesi Farmaceutici S.p.A.

Address

Via Palermo 26/a

City

Parma

Postcode

43122

Country

Italy

Scientific contact point

Organisation

Chiesi Farmaceutici S.p.A.

Contact name

Clinical Project Manager

Public contact point

Organisation

Chiesi Farmaceutici S.p.A.

Contact name

Clinical Project Manager

Third parties 3

Locations

4 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Results and documents

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

Documents 36 files

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

Application history

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