E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Celiac Disease |
Enfermedad celíaca |
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E.1.1.1 | Medical condition in easily understood language |
Celiac disease occurs in genetically predisposed people in whom the ingestion of gluten leads to damage in the small intestine |
La enfermedad celíaca se da en personas genéticamente predispuestas en las que la ingestión de gluten les provoca daño en su intestino delgado |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10007864 |
E.1.2 | Term | Celiac disease |
E.1.2 | System Organ Class | 100000004856 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the efficacy of PRV-015 in attenuating the symptoms of celiac disease in adult patients with non-responsive celiac disease (NRCD) as measured by the Abdominal Symptoms domain of the Celiac Disease Patient Reported Outcome (CeD PRO) questionnaire. |
Evaluar la eficacia de PRV- 015 desde el punto de vista de la atenuación de los síntomas de la enfermedad celíaca en pacientes adultos con CNR (celiaquía no respondedora), de acuerdo con los resultados del dominio de síntomas abdominales del cuestionario sobre resultados percibidos por el paciente en la enfermedad celíaca (RPP -EC). |
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E.2.2 | Secondary objectives of the trial |
• To assess the effect of treatment with PRV-015 on other measures of disease activity
• To assess the safety, tolerability, and pharmacokinetics (PK) of PRV-015 when administered to adult patients with non-responsive celiac disease (NRCD). |
• Evaluar el efecto del tratamiento con PRV-015 sobre otros criterios de valoración de la actividad de la enfermedad.
• Evaluar la seguridad, la tolerabilidad y la farmacocinética (FC) de PRV-015 cuando se administra a pacientes adultos con CNR. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Adult male or females, 18-70 years of age
• A diagnosis of celiac disease by intestinal biopsy
• Following a GFD for at least 12 consecutive months
• Must have detectable (above the lower limit of detection) serum celiac-related antibodies
• Must have human leukocyte antigen DQ (HLA-DQ) typing consistent with celiac disease (DQ2 and/or DQ8)
• Subjects must have had at least one of the following symptoms at least once per week during the month before screening: diarrhea, loose stools, abdominal pain, abdominal cramping, bloating, or gas.
• Body weight between 35 and 120 kg |
• Adultos hombres o mujeres, 18-70 años de edad
• Un diagnóstico de enfermedad celiaca por biopsia intestinal
• Después de una DSG durante al menos 12 meses consecutivos
• Debe tener anticuerpos séricos relacionados con la enfermedad celíaca detectables (por encima del límite inferior de detección)
• Debe tener una tipificación del antígeno leucocitario humano DQ (HLA-DQ) compatible con la enfermedad celíaca (DQ2 y / o DQ8)
• Los pacientes deben haber tenido al menos uno de los siguientes síntomas al menos una vez por semana durante el mes anterior a la selección: diarrea, heces blandas, dolor abdominal, calambres abdominales, distensión abdominal o gases.
• Peso corporal entre 35 y 120 Kg |
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E.4 | Principal exclusion criteria |
• Current diagnosis of any severe complication of celiac disease, such as refractory celiac disease type 1 (RCD-I) or RCD-II, enteropathy-associated T-cell lymphoma (EATL), ulcerative jejunitis, or gastrointestinal (GI) perforation
• Diagnosis of any chronic, active GI disease other than celiac disease
• Presence of any active infection
• Selective immunoglobulin A (IgA) deficiency, defined as having undetectable levels of IgA
• Known or suspected exposure to coronavirus disease 2019 (COVID-19) infection in the 4 weeks before screening
• Administration of a live vaccine within 14 days prior to randomization and the first administration of study drug
• History or presence of any clinically significant disease that, in the opinion of the Investigator, may confound the subject's participation and follow-up in the clinical trial or put the subject at unnecessary risk
• Females who are pregnant or planning to become pregnant during the study period, or who are currently breastfeeding |
• Diagnóstico actual de cualquier complicación grave de la enfermedad celíaca, como enfermedad celíaca refractaria tipo 1 (ECR-I o ECR-II, linfoma de células T asociado a enteropatía (LCTAE), yeyunitis ulcerosa o perforación gastrointestinal (GI)
• Diagnóstico de cualquier enfermedad gastrointestinal activa crónica distinta de la enfermedad celíaca
• Presencia de infección activa
• Deficiencia selectiva de inmunoglobulina A (IgA), definida por tener niveles indetectables de IgA
• Exposición o sospecha conocida a la infección por coronavirus 2019 (COVID-19) en las 4 semanas previas al screening.
• Administración de una vacuna viva dentro de los 14 días anteriores a la aleatorización y la primera administración del fármaco del studio
• Antecedentes o presencia de cualquier enfermedad clínicamente significativa que, en opinión del investigador, pueda confundir la participación y el seguimiento del paciente en el ensayo clínico o poner al paciente en un riesgo innecesario.
• Antecedentes o presencia de cualquier enfermedad clínicamente significativa que, en opinión del investigador, pueda confundir la participación y el seguimiento del paciente en el ensayo clínico o poner al paciente en un riesgo innecesario.
• |
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy of PRV-015 in attenuating the symptoms of celiac disease in adult patients with NRCD as measured by the Celiac Disease Patient-Reported Outcome (CeD PRO) questionnaire |
Eficacia de PRV-015 en atenuar los síntomas de la enfermedad celiaca en pacientes adultos con CNR medidos por el cuestionario sobre resultados percibidos por el paciente en la enfermedad celíaca (RPP - EC) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1. Effect of treatment with PRV-015 on other measures of disease activity - Intraepithelial lymphocyte (IEL) density
2. Incidence of treatment-emergent adverse events (TEAEs) - Safety endpoint
3. Serum trough concentrations of PRV-015 at scheduled visits - Characterize the pharmacokinetics (PK) of PRV-015
4. Incidence of anti-PRV-015 antibodies - Immunogenicity endpoint |
1. Efecto del tratamiento con PRV-01 en otras medidas de actividad de las enfermedad - Densidad de linfocitos intraepiteliales (LIE)
2. Incidencia de acontecimientos adversos aparecidos tras el tratamiento (AAAT) - Criterio de Seguridad
3. Concentraciones mínimas séricas de PRV-015 en visitas programadas - Caracterizar la farmacocinética (FC) de PRV-015
4. Incidencia de anticuerpos anti-PRV. Criterio de Inmunogenicidad |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Week 24 to Week 28 |
Semana 24 a Semana 28 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
United States |
Netherlands |
Spain |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 11 |