Extension study to evaluate the long-term outcomes of subjects in study 20090

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

Diseases [C] - Eye Diseases [C11]

Trial duration

27 Mar 2020 → 20 Sep 2025

Decision date (initial)

2023-11-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Bayer AG

External identifiers

EU CT number

2023-504207-89-00

EudraCT number

2018-003180-54

ClinicalTrials.gov

NCT04015180

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate long-term safety outcomes and visual function of subjects included in Study 20090 for treatment for retinopathy of prematurity (ROP)

Secondary objectives 1

1. To describe the visual function and overall development of subjects included in Study 20090 for treatment for ROP

Conditions and MedDRA coding

Retinopathy of prematurity

Study design 1 period

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-000236-PIP05-18

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 3

1. Subject was treated in Study 20090
2. Age less than 13 months of chronological age
3. Signed informed consent from parent(s)/legally authorized representative(s), which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol

Exclusion criteria 1

1. Subject has a medical condition preventing participation in the study, or performance of study procedures

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Binocular best-corrected visual acuity in Snellen equivalent score at 5 years of age.

Secondary endpoints 10

1. Proportion of subjects with ocular adverse events (AEs) and serious AEs (SAEs) through 5 years of age
2. Proportion of subjects with systemic AEs and SAEs through 5 years of age
3. Proportion of subjects developing unfavorable ocular structural outcome (retinal detachment, macular dragging, macular fold, retrolental opacity) at 1, 3, and 5 years of age
4. Proportion of subjects with absence of active ROP and unfavorable structural outcomes at 1 year of age
5. Best-corrected visual acuity in each eye at 3 and 5 years of age
6. Refractive spherical equivalent in each eye at 3 and 5 years of age
7. Neurodevelopmental outcomes at 2 and 5 years of age using standardized development tests (eg, Bayley Scales of Infant and Toddler Development, Third Edition [BSID-III], the Differential Ability Scales® II [DAS-II®], the Wechsler Preschool and Primary Scale of Intelligence™, Fourth Edition [WPSSI-IV], Vineland Adaptive Behavior Scales, Second Edition [VABS-II]).
8. Proportion of subjects with recurrence of ROP at 3 and 5 years of age
9. Proportion of subjects requiring treatment for ROP during this extension study
10. Proportion of subjects requiring ophthalmological treatment during this extension study

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

10 EU/EEA countries · 23 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 18 files

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

Application history

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