Efficacy, Safety, and Pharmacokinetics of Guselkumab in Pediatric Participants with Moderately to Severely Active Ulcerative Colitis

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

Diseases [C] - Immune System Diseases [C20]

Trial duration

14 May 2024 → ongoing

Decision date (initial)

2024-03-05

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: Therapy, Pharmacodynamic, Safety, Efficacy, Dose response

To evaluate the efficacy of guselkumab in pediatric participants with moderately to severely active UC at the end of maintenance therapy among participants who were induction responders.

Conditions and MedDRA coding

Ulcerative Colitis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001523-PIP04-19

Plan to share IPD

Yes

IPD plan description

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1) 2 to <18 years of age, inclusive (at the time of the first administration of study intervention at Week 0).
2. 2) Weight ≥10 kg at the time of consent for screening.
3. 3) A pathology report ‌to support a documented diagnosis of UC must be available in the source documents. There is no maximum duration for which a participant needs to be diagnosed with UC. If the pathology report to support a documented diagnosis of UC is not available in the source documents, the screening endoscopy with biopsies (obtained within 3 weeks before first study intervention administration) needs to support the diagnosis of UC.
4. 4) Moderately to severely active UC, defined by a baseline modified Mayo (without PGA) score of 5 through 9 inclusive, with a screening Mayo endoscopy subscore ≥2 as determined by a central review of the video of the endoscopy.
5. 5) Participant must: a. Have failed an advanced therapy, ie, have received treatment with 1 or more TNFα antagonist, vedolizumab, ozanimod, or JAK inhibitor, and have a documented history of failure to respond to or tolerate such treatment as defined in Appendix 16 and Appendix 19 OR b. Be naïve to advanced therapy (ie, TNFα antagonist, vedolizumab, ozanimod, or JAK inhibitor) or not have demonstrated a history of failure to respond to, or tolerate, advanced therapy and have a prior or current UC medication history that includes at least 1 of the following: 1) Inadequate response to or failure to tolerate current treatment with oral or IV corticosteroids, aminosalicylates, or immunomodulators as defined in Appendix 17 AND/OR 2) History of failure to respond to, or tolerate, at least 1 of the following therapies: oral or IV corticosteroids, aminosalicylates, or immunomodulators AND/OR 3) History of corticosteroid dependence as defined in Appendix 17

Exclusion criteria 5

1. 1) Have severe colitis as evidenced by: a. Investigator judgment that the participant is likely to require a colectomy within 12 weeks of Week 0. OR b. Symptom complex at screening or Week 0 visit that includes at least 4 of the following: 1) Diarrhea with ≥6 bowel movements/day with macroscopic blood in stool 2) Focal severe or rebound abdominal tenderness 3) Persistent fever (≥37.5°C) for more than 5 days 4) Persistent tachycardia for more than 5 days 5) Anemia (hemoglobin <8.5 g/dL)
2. 2) Have UC limited to the rectum only or to <20 cm of the colon.
3. 3) Presence of a stoma.
4. 4) Presence or history of a fistula
5. 5) Have evidence of CD: a. Small intestinal or ileal disease by upper GI small bowel follow-through, ileocolonoscopy with histology, video capsule endoscopy, or magnetic resonance enterography. b. Noncaseating and non-mucin granulomas that are suggestive of a diagnosis of CD on colonoscopy. c. Skip lesions on colonoscopy including absolute rectal sparing, with a normal rectum both endoscopically and histologically. d. Perianal disease.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Clinical remission (modified Mayo) at Week 56

Investigational products

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

Test 4

Placebo 3

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 8

Locations

8 EU/EEA countries · 33 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

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 126 files

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

Application history

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