A study evaluating the effects of filgotinib in children and teenagers with ulcerative colitis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Alfasigma S.p.A.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

15 Jun 2025 → ongoing

Decision date (initial)

2024-11-12

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Alfasigma

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy, Pharmacokinetic

To evaluate the efficacy of filgotinib in induction of remission and maintenance of effect in pediatric subjects with UC

Secondary objectives 5

1. To evaluate the safety and tolerability of filgotinib in pediatric subjects with UC
2. To evaluate the efficacy of a 58-week course of filgotinib to achieve response and remission in pediatric subjects with UC
3. To evaluate the effect of filgotinib on patient-reported outcomes and on health-related quality of life in pediatric subjects with UC
4. To characterize the PK of filgotinib in pediatric subjects with UC
5. To evaluate the acceptability of the filgotinib pediatric film-coated tablet formulation and the adult tablet formulation in pediatric subjects with UC

Conditions and MedDRA coding

Ulcerative colitis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001619-PIP03-16

Plan to share IPD

Yes

IPD plan description

Only after approval of Marketing Authorization for pediatric UC, at Alfasigma's discretion.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Subject must have a minimum BW of 15 kg
2. Subject: a. has documented diagnosis of UC with a minimum duration of 3 months, b. has mMCS of 5 to 9, and an MCS endoscopic score >=2, rectal bleeding >=1, and stool frequency >=1, c. has had an inadequate response, loss of response, intolerance, or has medical contraindications to corticosteroids, immunosuppressants, and/or biologic therapy. This includes subjects who depend on corticosteroids to control their symptoms and who experience worsening of their disease when attempting to wean off corticosteroids.
3. Female or male subjects from 8 to <18 years of age, on the date of signing the ICF.

Exclusion criteria 8

1. Subject has a diagnosis of inflammatory bowel disease (IBD)-unclassified or indeterminate colitis, isolated proctitis, or toxic megacolon
2. Subject has a history of colectomy or extensive small bowel resection.
3. Subject with psychological or cognitive difficulties that might interfere with study participation.
4. Subject has any previous exposure to a Janus kinase (JAK) inhibitor or medication with a similar mode of action (e.g. tofacitinib, baricitinib, upadacitinib).
5. Female subject is pregnant or breast feeding or intending to become pregnant or breastfeed during the study.
6. Subject has an active infection.
7. Subject with a history of complicated herpes zoster infection (with multi-dermatomal, disseminated, ophthalmic, or central nervous system involvement).
8. Currently on any therapy for chronic infection (such as pneumocystis, cytomegalovirus, herpes simplex, herpes zoster, or atypical mycobacteria).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Proportion of subjects achieving clinical remission based on modified Mayo Clinical Score (mMCS) at Week 10
2. Proportion of subjects achieving clinical remission based on mMCS2 at Week 58

Secondary endpoints 16

1. Incidence of treatment-emergent adverse events (TEAEs), adverse events of interest, and laboratory abnormalities through follow-up (Week 62)
2. Change from baseline in body mass index (BMI) at Week 58
3. Change from baseline in height velocity at Week 58
4. Proportion of subjects achieving clinical remission defined by Pediatric Ulcerative Colitis Activity Index (PUCAI) <10 at Week 10
5. Proportion of subjects achieving clinical remission defined by PUCAI <10 at Week 58
6. Proportion of subjects achieving endoscopic remission defined by Mayo endoscopic subscore of 0 at Week 10
7. Proportion of subjects achieving endoscopic remission defined by Mayo endoscopic subscore of 0 at Week 58
8. Proportion of subjects achieving mMCS response defined as mMCS reduction by >=2 points and >=30% from induction baseline, with decrease in rectal bleeding subscore of >=1 or absolute rectal bleeding subscore of 0 or 1 at Week 10
9. Proportion of subjects achieving MCS response defined as mMCS reduction by >=2 points and >=30% from induction baseline, with decrease in rectal bleeding subscore of >=1 or absolute rectal bleeding subscore of 0 or 1 at Week 58
10. Proportion of subjects achieving 6-month corticosteroid-free mMCS remission at Week 58
11. Change from baseline in TUMMY-UC score at Week 10 and Week 58
12. Proportion of subjects with TUMMY-UC remission at Week 10 and Week 58
13. Change from baseline in IMPACT-III score at Week 10 and Week 58
14. PK parameters of filgotinib and its primary metabolite GS-829845 (including maximum observed plasma concentration at steady state [Cmax,ss], area under the plasma concentration-time curve over the dosing interval at steady state [AUC0-24,ss], and area under the plasma concentration-time curve over the dosing interval at steady state for the effective exposure [AUCeff,ss])
15. Acceptability of the age-appropriate pediatric film-coated tablet formulation assessed by Pediatric Oral Medicine Acceptability Questionnaire for Patients (POMAQ-P)
16. Acceptability of the adult tablet formulation assessed by POMAQ-P

Investigational products

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

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Alfasigma S.p.A.

Sponsor organisation

Alfasigma S.p.A.

Address

Via Ragazzi Del '99 5

City

Bologna

Postcode

40133

Country

Italy

Scientific contact point

Organisation

Alfasigma S.p.A.

Contact name

Alexandra Mangili

Public contact point

Organisation

Alfasigma S.p.A.

Contact name

Alexandra Mangili

Third parties 8

Locations

12 EU/EEA countries · 42 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 6 · Art. 38 CTR

Results and documents

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

Documents 475 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