A prospective randomized single blind multicenter phase II study of organ perfusion with Custodiol-N compared with Custodiol in heart transplantation in children (CL-N-HTX-Paed-II/10/20)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dr. Franz Koehler Chemie GmbH

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

5 Mar 2025 → ongoing

Decision date (initial)

2024-05-24

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2023-510492-57-00

EudraCT number

2022-001795-34

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

The objective of this investigation is to compare the safety of two organ preservation solutions,Custodiol and Custodiol-N, in paediatric patients undergoing heart transplantation
Primary: Safety assessment, i.e. continuous adverse event reporting up to 3 months

Secondary objectives 13

1. Main Secondary: vital parameters up to day 7
2. laboratory tests including cardiac troponin and creatinine kinase (CKMB) up to day 7
3. Haemodynamics from termination of cardiopulmonary bypass until transfer to intensive care unit
4. Death up to 3 months
5. Graft survival up to 12 months
6. Readmission to ICU up to 12 months
7. Length of ICU stay up to 12 months
8. Catecholamine requirement up to day 7
9. Milrinone support up to day 7
10. Need for pacemaker therapy up to day 7
11. Echocardiographic markers of function and rejection up to day 7
12. Cardiac arrhythmias up to day 7
13. Device therapy up to 12 months

Conditions and MedDRA coding

heart transplantation in children

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002735-PIP03-20

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Patient criteria: Age from birth to less than18 years
2. Patient critera: Recipients awaiting their first transplant
3. Patient criteria: Ability of the patients and/ or their legal guardians to understand character and individual consequences of the clinical trial
4. Patient criteria: written informed consent of the patients and/ or their legal guardians (must be available before enrolment in the study)
5. Patient criteria: Patient listed on the waiting list for heart transplantation
6. Donor criteria: deceased donors should fulfil the standard criteria for organ donation (SCD)

Exclusion criteria 9

1. Patients who have participated within 30 days or are still participating in any other interventional studies
2. history of severe organic disease other than concerning the heart
3. history/demonstration of HIV antibodies or AIDS
4. multiorgan transplantation
5. machine-perfused organ
6. the explantation team is affiliated to another clinic than transplantation team
7. Failing Fontan patients
8. Pregnancy and lactation
9. Patients who have been incarcerated or involuntarily institutionalized by court order or by the authorities

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety assessment, i.e. continuous adverse event reporting up to 3 months

Secondary endpoints 15

1. Vital parameters (heart rate, blood pressure, body temperature) up to day 7
2. Laboratory tests including cardiac troponin and creatinine kinase (CK-MB) up to day 7o CK-MB peak value from 4 to 168 hours after release of the aortic cross clamp (measurements 4,8,12,16,20,24± 30min; 32h,40h,48h,56h,64h,72h ± 2 hours; visit 4-7 one time/interval) o CK-MB AUC value visit 1-7 o Troponin T up to 168 hours after release of the aortic cross clamp (measurements 4,8,12,16,20,24hours ± 30 min;32h,40h,48h,56h,64h,72h ± 2hours; visit 4-7 one time/interval) o Troponin AUC value visit 1-7
3. Hematology and clinical chemistry pre-operative and on visit 1,3,7 post-transplantation.
4. Haemodynamics at termination of cardiopulmonary bypass, and after 4h, 8h and 12h after aortic cross clamp release but only until transfer to intensive care unit (SBP, DBP), HR, PAP (if available), CVR (if available)
5. Death up to 3 months
6. Graft survival up to 12 months
7. Readmission to ICU up to 12 months
8. Length of ICU stays up to 12 months
9. Catecholamine requirement (yes/no) up to day 7
10. Antihypertensiva intake (yes/no) up to 3 months
11. Milrinone support (yes/no) up to day 7
12. Need for pacemaker therapy (yes/no) up to day 7 and up to 12 months
13. Device therapy (each yes/no) up to 12 months (including LVAD, RVAD,ECMO, BIVAD or percutaneuos LVAD)
14. Echocardiographic markers of function and rejection up to day 7 (Ejection fraction, enddiastolic and endsystolic ventricle volume)
15. Cardiac arrhythmias up to day 7 (occurrence, severity, type)

Investigational products

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

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dr. Franz Koehler Chemie GmbH

Sponsor organisation

Dr. Franz Koehler Chemie GmbH

Address

Werner-Von-Siemens-Strasse 14- 28

City

Bensheim

Postcode

64625

Country

Germany

Scientific contact point

Organisation

Dr. Franz Koehler Chemie GmbH

Contact name

Christoph Knosalla

Public contact point

Organisation

Dr. Franz Koehler Chemie GmbH

Contact name

Christoph Knosalla

Third parties 1

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Corrective measures 1 · Art. 77 CTR

Results and documents

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

Documents 13 files

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

Application history

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