A Phase 3, Multicenter, Open-label, Basket, Long-term Extension Study of Ustekinumab in Pediatric Clinical Study Participants (2 to <18 Years of Age)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Janssen - Cilag International

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Immune system processes [G12]

Trial duration

30 Nov 2021 → ongoing

Decision date (initial)

2023-04-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2022-501067-40-00

EudraCT number

2020-004457-76

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Others, Safety

Long-term safety data

Conditions and MedDRA coding

Moderately to Severely Active Ulcerative Colitis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Males or females 2 to <18 years of age, inclusive (at the time of the first administration of study intervention during the LTE).
2. Must have completed the dosing planned in the primary pediatric ustekinumab study.
3. Benefit of continued ustekinumab therapy (ie, a clinical response or clinical remission as defined in the primary study at the final efficacy last visit of the primary study).
4. Parent(s) (preferably both if available or as per local requirements), legal guardian(s) or their legally acceptable representative must sign an ICF indicating that he or she understands the purpose of, and procedures required for, the study and is willing to allow the child to participate in the study. Assent is also required of children capable of understanding the nature of the study (typically 7 years of age and older) as described in Informed Consent Process in protocol Appendix 3 (Section 10.3). An adolescent who signs the assent form will be given the opportunity to sign an adult ICF at a later visit when they reach the age of majority during the study to indicate that he or she participate in the study.
5. Please refer to study protocol section 5.1 for full list of inclusion criteria.

Exclusion criteria 7

1. Are pregnant, nursing, or planning pregnancy or fathering a child.
2. Have had ANY of (a) confirmed severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2 [COVID-19]) infection (test positive), OR (b) suspected SARS-CoV-2 infection (clinical features without documented test results), OR (c) close contact with a person with known or suspected SARS-CoV-2 infection
3. Exception: may be included with a documented negative result for a validated SARS-CoV-2 test (i) obtained at least 2 weeks after conditions (a), (b), (c) above (timed from resolution of key clinical features if present, eg, fever, cough, dyspnea) AND (ii) with absence of ALL conditions (a), (b), (c) above during the period between the negative test result and the baseline study visit NOTES on COVID-related exclusion
4. The field of COVID-related testing (for presence of, and immunity to, the SARS-CoV-2 virus) is rapidly evolving. Additional testing may be performed as part of screening and/or during the study if deemed necessary by the investigator and in accordance with current regulations/guidance from authorities/standards of care Precaution: for those who may carry a higher risk for severe COVID-19 illness, follow guidance from local health authorities when weighing the potential benefits and risks of enrolling in the study, and during participation in the study (protocol Appendix 8 [Section 10.8]).
5. Any condition for which, in the opinion of the investigator, participation would not be in the best interest of the participant (eg, compromise the well-being) or that could prevent, limit, or confound the protocol-specified assessments.
6. Participants who receive a live vaccination may be permitted to remain in the study, if approved by the sponsor and study intervention is held for a period of time specified by the sponsor. Receipt of a live SARS CoV- 2 vaccine (against the virus that causes COVID-19) is not automatically an exclusion criterion and must be discussed with the medical monitor.
7. Please refer to study protocol section 5.2 for full list of exclusion criteria.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. AEs, SAEs, AEs leading to discontinuation of study intervention, and AEs of special interest (as determined for each indication).
2. Clinical laboratory hematology and chemistry.
3. Injection-site reactions
4. AEs of worsening of the disease under study.
5. An addition of concomitant therapy due to loss of response.

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Janssen - Cilag International

Sponsor organisation

Janssen - Cilag International

Address

Turnhoutseweg 30

City

Beerse

Postcode

2340

Country

Belgium

Scientific contact point

Organisation

Janssen - Cilag International

Contact name

CTIS Point of Contact

Public contact point

Organisation

Janssen - Cilag International

Contact name

CTIS Point of Contact

Third parties 3

Locations

7 EU/EEA countries · 25 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 45 files

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

Application history

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