PURSUIT 2: A Study to Assess the Efficacy and Safety of Golimumab in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Sponsor

Janssen Research & Development, LLC (Industry)

Overall Status

Recruiting

CT.gov ID

NCT03596645

Collaborator

(none)

100

Enrollment

Locations

Arms

Anticipated Duration (Months)

1.5

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate efficacy of golimumab in inducing clinical remission as assessed by the Mayo score, in pediatric participants with moderately to severely active ulcerative colitis (UC). In addition, the safety profile of golimumab, in pediatric participants with moderately to severely active UC will be assessed.

Condition or Disease	Intervention/Treatment	Phase
Colitis, Ulcerative	Drug: Golimumab Drug: Infliximab	Phase 3

Detailed Description

This is a multicenter study in pediatric participants aged 2 to 17 years with moderately to severely active UC, defined as a baseline Mayo score of 6 through 12, inclusive, with an endoscopy subscore of greater than or equal to (>=)2. This 54-week study will consist of a 6-week short-term phase and a 48-week long-term phase followed by a study extension (Week 54 to end of study). At Week 58, participants who are eligible will continue receiving golimumab in the study extension. The primary hypothesis is that golimumab is an effective therapy in pediatric UC relative to historical placebo control as assessed by clinical remission based on Mayo score. Safety will be monitored throughout the study.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

100 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 3 Randomized, Open-Label Study to Assess the Efficacy, Safety, and Pharmacokinetics of Golimumab Treatment, a Human Anti-TNFα Monoclonal Antibody, Administered Subcutaneously in Pediatric Participants With Moderately to Severely Active Ulcerative Colitis

Actual Study Start Date :

Oct 29, 2018

Anticipated Primary Completion Date :

Dec 22, 2022

Anticipated Study Completion Date :

Jul 29, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Group 1: Golimumab Participants will receive subcutaneous (SC) golimumab through Week 50. Doses will be based on body surface area. After the Week 54 evaluations, at the discretion of investigator, participants benefiting from continued SC golimumab will continue to receive SC golimumab in the extension until end of study.	Drug: Golimumab Participants receive subcutaneous (SC) golimumab through Week 50, where doses will be based on body surface area. After the Week 54 evaluations, at the discretion of investigator, participants benefiting from continued SC golimumab will continue to receive SC golimumab in the extension until end of study.
Experimental: Group 2: Infliximab Participants will receive infliximab intravenous (IV) through Week 46. Doses will be based on body weight. After the Week 54 evaluations, participants receiving infliximab will be withdrawn from study participation and transition to local standard of care which may include continued commercially available infliximab at the discretion of their physician.	Drug: Infliximab Participants will receive infliximab intravenous (IV) through Week 46. Doses will be based on body weight. After the Week 54 evaluations, participants receiving infliximab will be withdrawn from study participation and transition to local standard of care which may include continued commercially available infliximab at the discretion of their physician.

Arm

Intervention/Treatment

Experimental: Group 1: Golimumab

Participants will receive subcutaneous (SC) golimumab through Week 50. Doses will be based on body surface area. After the Week 54 evaluations, at the discretion of investigator, participants benefiting from continued SC golimumab will continue to receive SC golimumab in the extension until end of study.

Drug: Golimumab

Participants receive subcutaneous (SC) golimumab through Week 50, where doses will be based on body surface area. After the Week 54 evaluations, at the discretion of investigator, participants benefiting from continued SC golimumab will continue to receive SC golimumab in the extension until end of study.

Experimental: Group 2: Infliximab

Participants will receive infliximab intravenous (IV) through Week 46. Doses will be based on body weight. After the Week 54 evaluations, participants receiving infliximab will be withdrawn from study participation and transition to local standard of care which may include continued commercially available infliximab at the discretion of their physician.

Drug: Infliximab

Outcome Measures

Primary Outcome Measures

Clinical Remission at Week 6 as Assessed by the Mayo Score [At Week 6]
Clinical remission is defined as a Mayo score less than or equal to (<=) 2 points, with no individual sub score greater than (>) 1. The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.

Secondary Outcome Measures

Symptomatic Remission at Week 54 [At Week 54]
Symptomatic remission is defined as Mayo stool frequency subscore of 0 or 1 and a rectal bleeding subscore of 0.
Clinical Remission at Week 54 as Assessed by the Mayo score [At Week 54]
Clinical remission is defined as a Mayo score <=2 points, with no individual subscore >1 (based on Mayo endoscopy subscore assigned by the local endoscopist). The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.
Clinical Remission at Week 54 as Assessed by the Pediatric Ulcerative Colitis Activity Index Score (PUCAI) Score [At Week 54]
Clinical remission is defined as a PUCAI score less than (<)10. The PUCAI is a noninvasive measure of UC disease activity. It comprises 6 scales and total score ranges between 0 and 85 points. The scales are: abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI total score is calculated as the sum of the 6 subscores. Higher scores indicate a more severe disease.
Clinical Remission at Week 6 as Assessed by the PUCAI Score [At Week 6]
Clinical remission is defined as a PUCAI score <10. The PUCAI is a noninvasive measure of ulcerative colitis (UC) disease activity. It comprises 6 scales and total score ranges between 0 and 85 points. The scales are: abdominal pain, rectal bleeding, stool consistency, number of stools, nocturnal bowel movement, and activity level. The PUCAI total score is calculated as the sum of the 6 subscores. Higher scores indicate a more severe disease.
Clinical Response at Week 6 as Assessed by the Mayo Score [At Week 6]
Clinical response is defined as a decrease from baseline in the Mayo score of greater than or equal to (>=)30 percent (%) and >=3 points, with either a decrease from baseline in the rectal bleeding subscore of >=1 or a rectal bleeding subscore of 0 or 1. The Mayo score consists of 4 sub scores (stool frequency, rectal bleeding, endoscopy findings, and physician's global assessment), each of which is rated on a scale from 0 to 3, indicating normal to severe activity. The total score is calculated as the sum of the 4 sub scores and values range from 0 to 12. A score of 3 to 5 points indicates mildly active disease; a score of 6 to 10 indicates moderately active disease; and a score of 11 to 12 indicates severe disease.
Endoscopic Healing at Week 6 [At Week 6]
Endoscopic healing is defined as an endoscopy subscore of the Mayo score of 0 (normal or inactive disease) or 1 (mild disease).
Endoscopic Healing at Week 54 [At Week 54]
Endoscopic healing is defined as an endoscopy subscore of the Mayo score of 0 (normal or active disease) or 1 (mild disease).

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Must either be currently receiving treatment with, or have a history of having failed to respond to, or have a medical contraindication to at least 1 of the following therapies: oral or intravenous corticosteroids, 6-mercaptopurine, methotrexate or azathioprine OR must either have or have had a history of corticosteroid dependency (that is an inability to successfully taper corticosteroids without a return of the symptoms of ulcerative colitis [UC]) OR required more than 3 courses of corticosteroids in the past year
Moderately to severely active UC (as defined by baseline Mayo score of 6 through 12 [endoscopy sub score assigned by local endoscopist], inclusive), including a (sigmoidoscopy or colonoscopy) sub score greater than or equal to (>=2)
If receiving enteral nutrition, must have been on a stable regimen for at least 2 weeks prior to the first administration of study intervention at Week 0. Participants who receive parenteral nutrition are not permitted to enroll in the trial
No history of latent or active tuberculosis prior to screening
Must be up to date with all immunizations (that is, measles, mumps, rubella, and varicella) in agreement with current local immunization guidelines for immunosuppressed participants before Week 0

Exclusion Criteria:

History of liver or renal insufficiency; significant cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, rheumatologic, psychiatric (including suicidality), or metabolic disturbances
History of malignancy or macrophage activation syndrome or hemophagocytic lymphohistiocytosis
Have UC limited to the rectum only or to <20 percent (%) of the colon
Presence of a stoma
Presence or history of a fistula
Contraindications to the use of golimumab or infliximab or anti-tumor necrosis factor (TNF-alpha) therapy per local prescribing information

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California San Francisco	San Francisco	California	United States	94158
2	Children's Hospital Colorado and University of Colorado	Aurora	Colorado	United States	80045
3	Rocky Mountain Pediatric Gastroenterology	Lone Tree	Colorado	United States	80124
4	Connecticut Children's Medical Center	Hartford	Connecticut	United States	06106
5	Nemours DuPont Hospital for Children	Wilmington	Delaware	United States	19803
6	Children's Center for Digestive Health Care	Atlanta	Georgia	United States	30342
7	Boston Children's Hospital	Boston	Massachusetts	United States	02115
8	Mayo Clinic	Rochester	Minnesota	United States	55905
9	Columbia University Medical Center	New York	New York	United States	10032
10	University of North Carolina at Chapel Hill	Chapel Hill	North Carolina	United States	27599
11	Digestive Disease Specialists Inc	Oklahoma City	Oklahoma	United States	73112
12	Medical University of South Carolina	Charleston	South Carolina	United States	29425
13	GI For Kids	Knoxville	Tennessee	United States	37922
14	Children's Medical Center of Dallas	Dallas	Texas	United States	75207
15	Cook Childrens Medical Center	Fort Worth	Texas	United States	76104
16	DHAT Research Institute	Garland	Texas	United States	75044
17	Universitair Kinderziekenhuis Koningin Fabiola	Brussel		Belgium	1020
18	Cliniques Universitaires Saint-Luc	Brussel		Belgium	1200
19	UZ Brussel	Jette		Belgium	1090
20	MK Blumenau Pesquisa Clínica	Blumenau		Brazil	89010-506
21	Hospital Pequeno Principe	Curitiba		Brazil	80250-060
22	Hospital da Crianca Santo Antonio de Porto Alegre	Porto Alegre		Brazil	92020-430
23	BR Trials	São Paulo		Brazil	05003-090
24	Hôpital Pellegrin CHU Bordeaux	Bordeaux		France	33000
25	Hopital Femme Mère Enfant	Bron		France	69677
26	Hôpital Necker	Paris		France	75015
27	Rambam Medical Center	Haifa		Israel	3109601
28	Shaare Zedek Medical Center	Jerusalem		Israel	91031
29	Schneider Children's Medical Center	Petah Tikva		Israel	4920235
30	Sheba Medical Center	Ramat Gan		Israel	52621
31	Assaf Harofeh Medical Center	Rishon-Le-Zion		Israel	70300
32	Sourasky Medical Center	Tel-Aviv		Israel	6997801
33	Azienda USL di Bologna - Ospedale Maggiore	Bologna		Italy	40133
34	ASST Spedali Civili Brescia	Brescia		Italy	25123
35	AOU Meyer	Firenze		Italy	50139
36	AOU Policlinico G.Martino	Messina		Italy	98124
37	AOU Policlinico Umberto I	Roma		Italy	00161
38	IRCCS Ospedale Pediatrico Bambino Gesu	Roma		Italy	00165
39	Casa Sollievo della Sofferenza, IRCCS	San Giovanni Rotondo		Italy	71013
40	IRCCS Materno Infantile Burlo Garofolo	Trieste		Italy	34137
41	Kyungpook National University Chilgok Hospital	Daegu		Korea, Republic of	41404
42	Seoul National University Hospital	Seoul		Korea, Republic of	03080
43	Severance Hospital, Yonsei University Health System	Seoul		Korea, Republic of	03722
44	Asan Medical Center	Seoul		Korea, Republic of	05505
45	Samsung Medical Center	Seoul		Korea, Republic of	06351
46	Emma Children's Hospital Academic Medical Center	Amsterdam		Netherlands	1105 AZ
47	Radboudumc Amalia Children's Hospital	Nijmegen		Netherlands	6500 HB
48	Isala Kliniek	Zwolle		Netherlands	8000 GK
49	Szpital Uniwersytecki NR 1 IM. Dr. Antoniego Jurasza	Bydgoszcz		Poland	85-094
50	Szpital im. M. Kopernika	Gdansk		Poland	80-803
51	Uniwersytecki Szpital Dzieciecy w Krakowie	Krakow		Poland	30-663
52	Wojewodzki Specjalistyczny Szpital Dziecięcy im. Prof. Stanislawa Popowskiego	Olsztyn		Poland	10-561
53	Gabinet Lekarski Bartosz Korczowski	Rzeszow		Poland	35-302
54	Centrum Zdrowia Matki, Dziecka i Młodzieży	Warszawa		Poland	00-635
55	Szpital Pomnik Centrum Zdrowia Dziecka	Warszawa		Poland	04-730
56	Hosp. Univ. de Cruces	Barakaldo		Spain	48902
57	Hosp. Sant Joan de Deu	Barcelona		Spain	08950
58	Hosp. Infantil Univ. Niño Jesus	Madrid		Spain	28009
59	Hosp. Gral. Univ. Gregorio Marañon	Madrid		Spain	28036
60	Hosp. Univ. 12 de Octubre	Madrid		Spain	28041
61	Hosp. Regional Univ. de Malaga	Málaga		Spain	29011
62	Hosp. Virgen Del Rocio	Sevilla		Spain	41013
63	National Taiwan University Hospital	Taipei		Taiwan	100
64	Taipei Veterans General Hospital	Taipei		Taiwan	112
65	Chang Gung Memorial Hospital- Linkou	Taoyuan City		Taiwan	33305

Sponsors and Collaborators

Janssen Research & Development, LLC

Investigators

Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Janssen Research & Development, LLC

ClinicalTrials.gov Identifier:

NCT03596645

Other Study ID Numbers:

CR108499
2017-004496-31
CNTO148UCO3003

First Posted:

Jul 24, 2018

Last Update Posted:

Aug 18, 2022

Last Verified:

Aug 1, 2022

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 18, 2022