Clinical trial NCT05430412: CT to Assess the Efficacy and Safety of Adding GMA to Infliximab in Paediatric Patients With Steroid-refractory Ulcerative Colitis
Official title: Clinical Trial to Assess the Efficacy and Safety of Adding GMA to Infliximab in Paediatric Patients With Steroid-refractory Ulcerative Colitis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Ulcerative Colitis
Keywords: Ulcerative Colitis
Lead sponsor: Adacyte Therapeutics SL
Sponsor class: INDUSTRY
Collaborators: name: Adknoma Health Research
First posted: 2022-06-24
Enrollment: 15
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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

Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: 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 IFX-LEVELS
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: 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 STEROIDS USE
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
Secondary outcomes:
measure: To evaluate the IFX and ATI level variations in the treatment regimen during the study period. Quantification of IFX trough and ATI levels, and description of the IFX dose at baseline, 12 and 40 weeks of follow-up
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To measure the PUCAI score variation during the study period. Measurement of PUCAI score at baseline, 12 and 40 weeks of follow-up.
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To determine the percentage of patients maintaining steroid-free response during the study period at 12 and 40 weeks.
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To describe the number of flare-ups during the study period. Patients experiencing flares-ups during the study period.
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To measure faecal calprotectin level during the study period. Quantification of faecal calprotectin level at baseline, 12 and 40 weeks of follow-up. Faecal calprotectin is associated with clinical remission with levels higher than 150 μg/g.
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To monitor the evolution of biochemical markers of inflammatory activity in UC and other laboratory parameters. Measurement of C-reactive protein, ESR, haemoglobin, albumin, platelet levels, PT, granulocytes at baseline, 12(V11) and 40 weeks(V18)
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To determine the evolution of leucocyte counts in peripheral blood during the study period. Quantification of leucocyte counts in peripheral blood at baseline, 12 and 40 weeks of follow-up.
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
measure: To evaluate the safety of Adacolumn® during the study period, all the recorded AEs will be described by:
timeFrame: Baseline visit vs Visit 11 (12 weeks) (interim analysis). Baseline visit vs Visit 18 (40 weeks) (for final analysis)
Other outcomes:
Locations:
city: Coimbra
country: Portugal
facility: Hospital Coimbra
city: Lisboa
country: Portugal
facility: Hospital Santa Maria
city: Oporto
country: Portugal
facility: Hospital Soa Joao
city: Sant Joan Despí
country: Spain
facility: Hospital H. Sant Joan de Déu
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Niño Jesús
city: Málaga
country: Spain
facility: Hospital Materno-Infantil del H.U.R. de Málaga
city: Sevilla
country: Spain
facility: Complejo H. Regional Virgen Del Rocío
city: Tenerife
country: Spain
facility: Hospital U. Ntra Señora de Candelaria
city: Valencia
country: Spain
facility: Hospital Universitari I Politècnic La Fe
Study officials:
affiliation: Hospital San Joan de Deu
name: Francisco Javier Martín de Carpi
role: STUDY_CHAIR
affiliation: CHU Sao Joao
name: Jorge Manuel Bastos Amil Dias
role: STUDY_CHAIR
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