Trial Outcomes & Findings for Predicting Response to Standardized Pediatric Colitis Therapy (NCT NCT01536535)

Last Updated: 2019-09-20

Results Overview

Week 52 CS-free remission: Number of participants with a PUCAI \< 10 and no corticosteroids (CS) for 28 days without additional therapy or colectomy. Participants in both groups received corticosteroids, biologics, or colectomies, if symptomatic. The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a non-invasive disease activity index. The index measures include abdominal pain, rectal bleeding, stool consistency, number of stools per 24 hours, nocturnal stools, and activity level. A total score less than 10 indicates remission, Mild disease activity is 10-30, Moderate 35-60, Severe disease activity 65-85.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

431 participants

Primary outcome timeframe

52 weeks

Results posted on

2019-09-20

Participant Flow

Recruitment and enrollment began in July 2012 and ended in April 2015. Final study visits were in April 2016 at which time all study patients had a minimum of one year follow up. Patients were recruited from 29 centers in the United States and Canada.

Participants were consented at diagnostic colonoscopy obtaining biological specimens (DNA, serum, rectal tissue, stool for microbiome). After diagnosis of Ulcerative Colitis, they began treatment dictated by initial disease severity and provider/patient choice. Three patients were untreated.

Participant milestones

Participant milestones
Measure	Mild UC Disease Mild = Initiated on mesalazine, or on oral CS with Pediatric Ulcerative Colitis Activity Index (PUCAI) \< 45	Moderate to Severe UC Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45
Overall Study STARTED	178	250
Overall Study COMPLETED	163	237
Overall Study NOT COMPLETED	15	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Mild UC Disease Mild = Initiated on mesalazine, or on oral CS with Pediatric Ulcerative Colitis Activity Index (PUCAI) \< 45	Moderate to Severe UC Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45
Overall Study Withdrawal by Subject	10	5
Overall Study Lost to Follow-up	4	6
Overall Study Physician Decision	1	1
Overall Study Participant moved	0	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mild UC n=178 Participants Mild = Initiated on mesalamine, or on oral CS with PUCAI \< 45	Moderate to Severe UC n=250 Participants Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45	Total n=428 Participants Total of all reporting groups
Age, Continuous	12.6 years STANDARD_DEVIATION 3.4 • n=178 Participants	12.7 years STANDARD_DEVIATION 3.2 • n=250 Participants	12.7 years STANDARD_DEVIATION 3.3 • n=428 Participants
Sex: Female, Male Female	88 Participants n=178 Participants	124 Participants n=250 Participants	212 Participants n=428 Participants
Sex: Female, Male Male	90 Participants n=178 Participants	126 Participants n=250 Participants	216 Participants n=428 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: 52 weeks

Outcome measures

Outcome measures
Measure	Mild UC n=163 Participants Mild = Initiated on mesalazine, or on oral CS with PUCAI \< 45	Moderate to Severe UC n=237 Participants Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45
Number of Participants With Corticosteroid Free Remission (SFR)	80 Participants	70 Participants

PRIMARY outcome

Timeframe: Within 52 weeks

Number of participants who needed additional therapy or colectomy. Additional therapy included Anti-Tumour Necrosis Factor alpha (TNFα), Calcineurin inhibitor, Immunomodulator

Outcome measures

Outcome measures
Measure	Mild UC n=163 Participants Mild = Initiated on mesalazine, or on oral CS with PUCAI \< 45	Moderate to Severe UC n=237 Participants Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45
Number of Participants Who Needed Additional Therapy or Colectomy	46 Participants	151 Participants

SECONDARY outcome

Timeframe: Within 52 weeks

Number of participants who received a colectomy within 52 weeks

Outcome measures

Outcome measures
Measure	Mild UC n=163 Participants Mild = Initiated on mesalazine, or on oral CS with PUCAI \< 45	Moderate to Severe UC n=237 Participants Moderate/Severe = Initiated on IV CS, or oral CS with PUCAI ≥45
Number of Participants Receiving a Colectomy	2 Participants	23 Participants

Adverse Events

Mild UC

Serious events: 21 serious events

Other events: 157 other events

Deaths: 0 deaths

Moderate to Severe UC

Serious events: 79 serious events

Other events: 224 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Mild UC n=178 participants at risk Mild = Initiated on 5-Aminosalicylates (ASA) or on oral CS with PUCAI \< 45	Moderate to Severe UC n=250 participants at risk Moderate/Severe = Initiated on IV CS or oral CS with PUCAI ≥45
Gastrointestinal disorders Hospitalized for UC complications	9.6% 17/178 • Adverse events were collected from Baseline through to the 52 week visit.	27.6% 69/250 • Adverse events were collected from Baseline through to the 52 week visit.
Hepatobiliary disorders Pancreatitus acute	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Renal and urinary disorders Nephrolithiasis	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.80% 2/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Clostridium difficile colitis	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.00% 0/250 • Adverse events were collected from Baseline through to the 52 week visit.
General disorders Dehydration	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.00% 0/250 • Adverse events were collected from Baseline through to the 52 week visit.
Psychiatric disorders Depression	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.00% 0/250 • Adverse events were collected from Baseline through to the 52 week visit.
Nervous system disorders Generalised tonic-clonic seizure	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Gastroenteritis	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Immune system disorders Hypersensitivity to mesalamine severe headache and pleuritic chest pain	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Psychiatric disorders Intentional Overdose	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Intestinal anastomosis	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
General disorders Syncope	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.
Immune system disorders Vasculitic rash	0.00% 0/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.40% 1/250 • Adverse events were collected from Baseline through to the 52 week visit.

Other adverse events

Other adverse events
Measure	Mild UC n=178 participants at risk Mild = Initiated on 5-Aminosalicylates (ASA) or on oral CS with PUCAI \< 45	Moderate to Severe UC n=250 participants at risk Moderate/Severe = Initiated on IV CS or oral CS with PUCAI ≥45
Gastrointestinal disorders Abdominal Pain	31.5% 56/178 • Adverse events were collected from Baseline through to the 52 week visit.	28.4% 71/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Diarrhea	29.2% 52/178 • Adverse events were collected from Baseline through to the 52 week visit.	28.4% 71/250 • Adverse events were collected from Baseline through to the 52 week visit.
General disorders Headache	11.8% 21/178 • Adverse events were collected from Baseline through to the 52 week visit.	9.2% 23/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Nausea	5.6% 10/178 • Adverse events were collected from Baseline through to the 52 week visit.	6.8% 17/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Vomiting	3.9% 7/178 • Adverse events were collected from Baseline through to the 52 week visit.	4.4% 11/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Dyspepsia	2.2% 4/178 • Adverse events were collected from Baseline through to the 52 week visit.	4.4% 11/250 • Adverse events were collected from Baseline through to the 52 week visit.
Skin and subcutaneous tissue disorders Rash	1.7% 3/178 • Adverse events were collected from Baseline through to the 52 week visit.	1.6% 4/250 • Adverse events were collected from Baseline through to the 52 week visit.
Skin and subcutaneous tissue disorders Acne	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	4.4% 11/250 • Adverse events were collected from Baseline through to the 52 week visit.
Gastrointestinal disorders Clostridium difficile	1.1% 2/178 • Adverse events were collected from Baseline through to the 52 week visit.	1.2% 3/250 • Adverse events were collected from Baseline through to the 52 week visit.
Hepatobiliary disorders Pancreatitis	0.56% 1/178 • Adverse events were collected from Baseline through to the 52 week visit.	0.80% 2/250 • Adverse events were collected from Baseline through to the 52 week visit.

Additional Information

Dr. Jeffrey Hyams

Connecticut Childrens

Phone: 8605459532

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place