CT to Assess the Efficacy and Safety of Adding GMA to Infliximab in Paediatric Patients With Steroid-refractory Ulcerative Colitis

Adacyte Therapeutics SL15 enrolled

Overview

The primary endpoint will be evaluated through the following variables: PUCAI score, IFX levels, and steroid treatment. Clinical response to IFX will be evaluated through the PUCAI score. The response will be considered clinically significant if PUCAI points continue maintained below 30 during the study period. The IFX response will also be determined by IFX serum levels. A therapeutic IFX level, i.e. for achieving an adequate clinical response, is established above 6 μg/mL. Finally, the necessity, or not, of a steroid treatment during the study period will also be indicative of successful efficacy with GMA.

The trial has the following objectives: Primary objective (PO): The primary endpoint will be evaluated through the following variables: PUCAI score, IFX levels, and steroid treatment. Clinical response to IFX will be evaluated through the PUCAI score. The response will be considered clinically significant if PUCAI points continue maintained below 30 during the study period. The IFX response will also be determined by IFX serum levels. A therapeutic IFX level, i.e. for achieving an adequate clinical response, is established above 6 μg/mL. Finally, the necessity, or not, of a steroid treatment during the study period will also be indicative of successful efficacy with GMA. Secondary objectives (SO): 1. Quantification of IFX trough and ATI levels, and description of the IFX dose at baseline, 12 and 40 weeks of follow-up. 2. Measurement of PUCAI score at baseline, 12 and 40 weeks of follow-up. 3. Patients maintaining clinical response off steroids, 12 and 40 weeks of follow-up. 4. Patients experiencing flares-ups during the study period. 5. Quantification of fecal calprotectin level at baseline, 12 and 40 weeks of follow-up. Fecal calprotectin is associated with clinical remission with levels higher than 150 μg/g. 6. Measurement of C-reactive protein, erythrocyte sedimentation rate, hemoglobin, albumin, platelet levels at baseline, 12 and 40weeks of follow-up. 7. Quantification of leucocyte counts in peripheral blood at baseline, 12 and 40 weeks of followup. 8. Monitoring of AEs during the study period

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Eligibility

Sex: ALLMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients under 18 years of age and weighing ≥25 kg at the time of study initiation. 2. Patients with diagnosis of UC. 3. Patients who started IFX treatment due to the lack of response to corticosteroids following an UC flare-up (steroid-refractory UC). 4. Patients who have received IFX between 12 and 16 weeks prior to the study initiation. 5. Patients who have showed a clinical response to IFX at the time of study initiation (defined as a reduction of at least 15 points in PUCAI score and being maintained below 30 points). 6. Patients with therapeutic IFX blood levels (above 6 μg/mL) at the time of study initiation. 7. Patient´s legal guardian must be willing and able to give written informed consent, and the patient must be willing to give written informed assent (if applicable as determined by the Ethics Committee) and comply with the Study visit Schedule. Exclusion Criteria: 1. Patients who have received another anti-TNF prior to entry in the study. 2. Patients with a peripheral circulation count of less than 2,000 granulocytes per μL. 3. Pregnant and lactating of childbearing potential patients. 4. Participation in another study or use of any experimental therapy within 30 days before day 1 of Study initiation.

Locations (10)

Hospital Coimbra

Coimbra, Portugal

RECRUITING

Hospital Santa Maria

Lisbon, Portugal

RECRUITING

Hospital Soa Joao

Porto, Portugal

RECRUITING

Hospital H. Sant Joan de Déu

Sant Joan Despí, Barcelona, Spain

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, Spain

RECRUITING

Hospital Infantil Universitario Niño Jesús

Madrid, Spain

RECRUITING

Hospital Materno-Infantil del H.U.R. de Málaga

Málaga, Spain

RECRUITING

Hospital U. Ntra Señora de Candelaria

Santa Cruz de Tenerife, Spain

RECRUITING

Complejo H. Regional Virgen Del Rocío

Seville, Spain

RECRUITING

Hospital Universitari I Politècnic La Fe

Valencia, Spain

RECRUITING

Outcomes

Primary Outcomes

Primary objective:evaluate if Adacolumn® can sustain clinical response to IFX for 12 weeks of follow-up (V.11) or 40 weeks of follow-up (V18). Clinical response to IFX will be evaluated through PUCAI SCORE

Description of PUCAI score as continuous variable at V.11 and at V.18 will be performed. Response to IFX will be clinically significant if PUCAI score continues maintained below 30 during the study (follow-up visits), so the nº and % of patients with a PUCAI score below 30 points in all visits between baseline to V.11 and baseline to V.18 will be described. Furthermore, comparison scores in both visits (baseline vs. V.11 and baseline vs. V.18) will be performed, by means of absolute and relative changes