A study to learn how gadoquatrane moves into, through, and out of the body and how safe it is in children aged 0 to less than 18 years, who will undergo a contrast enhanced MRI

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Bayer AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01]

Trial duration

16 Oct 2023 → 22 Nov 2024

Decision date (initial)

2023-10-04

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Bayer AG, Leverkusen, Germany

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety

To evaluate plasma PK of gadoquatrane after a single intravenous injection of 0.04 mmol Gd/kg bw in pediatric participants undergoing CE-MRI

Secondary objectives 1

1. To evaluate the safety and tolerability of gadoquatrane after a single intravenous injection of 0.04 mmol Gd/kg bw in pediatric participants undergoing CE-MRI

Conditions and MedDRA coding

Contrast enhancement in magnetic resonance imaging in children

Study design 1 period

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002778-PIP01-20

Plan to share IPD

No

IPD plan description

Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Participants from birth to <18 years of age at the time of signing the informed consent form
2. Participants who have a clinical indication to undergo a CE-MRI for any clinical condition in any body region and who can undergo study procedures as per Investigator judgement
3. Participants can be male or female (according to their reproductive organs and functions assigned by chromosomal complement)
4. Female contraception and barriers as well as pregnancy testing is required as appropriate for the age and sexual activity of pediatric participants and as required by local regulations
5. The legal guardian(s) is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
6. When applicable by law for a participant, a legal guardian must be available to help the study-site personnel ensure follow up, accompany the participant to the study site on each assessment day, and consistently and consecutively be available to provide information on the participant during the scheduled study visits

Exclusion criteria 11

1. Body weight <2500 g at screening and baseline
2. The medical condition and/or local allowance for maximum blood draw in the pediatric participant is not suitable for study procedures, including PK and safety blood draws, without compromising any expected clinical care/procedure need
3. Acute kidney injury (i.e., acute renal failure)
4. Age-adjusted renal function is "decreased" (eGFR <80% of age adjusted normal renal function) as evaluated by the investigator based on a serum or plasma creatinine result obtained within 2 weeks prior to study intervention.
5. Considered clinically unstable or has a concurrent/concomitant condition that may not allow participation for the full planned study period, in the judgement of the investigator
6. History of moderate to severe allergic-like reaction to any GBCA
7. Bronchial asthma which is considered unstable or had major therapeutical modification within last 4 weeks.
8. Severe cardiovascular disease, except for cardiac or vascular magnetic resonance (MR), if considered clinically justified by the investigator
9. Planned or expected intervention (e.g., treatment or procedure) or change in treatment (e.g. start of chemotherapy) that may significantly affect study parameters (i.e. safety/AEs [e.g. confounding AEs or safety events due to surgery or chemotherapy], PK parameters) or would prevent the participant from performing study procedures, from the administration of gadoquatrane up to the 24 h ± 4 h follow-up
10. Participants who received or will receive any other contrast agent within 72 hours prior to gadoquatrane injection or up to 72 hours after gadoquatrane injection
11. Contraindications to the administration of GBCAs (depending on local product label), or history of adverse reaction to GBCAs

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Area under the curve (AUC) of gadoquatrane after single administration
2. Plasma clearance normalized to body weight (CL/BW) of gadoquatrane after single administration
3. Apparent volume of distribution at steady state normalized to body weight (Vss/bw)
4. Simulation of plasma concentration at 20 minutes post-injection (C20)

Secondary endpoints 1

1. Number and intensity of treatment-emergent AEs (TEAEs), including number of SAEs, occurring within 24 (±4) hours post-injection or in the post-treatment period of 7 (±1) days after the day of study intervention

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Bayer AG

Sponsor organisation

Bayer AG

City

Leverkusen

Postcode

51368

Country

Germany

Scientific contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Public contact point

Organisation

Bayer AG

Contact name

Therapeutic Area Head

Third parties 2

Locations

6 EU/EEA countries · 18 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 13 files

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

Application history

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