Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia

Официальное название

Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia: a Double-blind, Randomized, Placebo-controlled Trial

Обзор

Celiac disease in children is frequently associated with iron deficiency and/or iron deficiency anemia due to intestinal malabsorption and chronic inflammation. Although a gluten-free diet is the standard treatment and can restore iron balance over time, there is currently no clear evidence or consensus on the role and timing of iron supplementation in pediatric patients at diagnosis. Given the potential impact of anemia on growth and neurodevelopment, strategies that enable a faster correction of iron deficiency are clinically relevant. Sucrosomial® iron has shown improved absorption and gastrointestinal tolerability compared to conventional oral iron in adult celiac patients. This study aims to evaluate whether Sucrosomial® iron supplementation, in addition to a gluten-free diet, is more effective and safe than diet alone in achieving a faster normalization of hemoglobin and iron stores in children with newly diagnosed celiac disease. The primary objective of this randomized, double-blind, placebo-controlled, parallel-group study is to assess whether oral supplementation with Sucrosomial® iron, when added to a gluten-free diet (GFD), accelerates the normalization of iron stores and hemoglobin levels compared with GFD alone in school-age children and adolescents newly diagnosed with celiac disease presenting with hypoferritinemia and/or iron deficiency anemia. Target Study Population: Children and adolescents with celiac disease and iron deficiency or anemia due to iron deficiency. Study Duration Total study duration (per patient) will be about 6 months; total treatment duration (per patient) will be 6 months. Number of Patients: 60 planned Two typologies of patients will be included: with hypoferritinemia and with anemia due to iron deficiency. The randomization process will be stratified, so that: * 15 patients with hypoferritinemia receive active treatment and 15 patients receive placebo; * 15 patients with anemia due to iron deficiency receive active treatment and 15 patients receive placebo. The age of patients will also be considered for the randomization (to assign the correct number of product bottles).

Вмешательства

• Пищевая добавка Sideral forte® VERUM drops
  Patients with hypoferritinemia (no anemia): * From 8 years until development (Tanner stage \<=3): 1 ml of Sideral forte® VERUM drops, equal to 14 mg of iron element; * From development (Tanner stage \>3) up to 18 years: 2 ml of Sideral forte® VERUM drops, equal to 28 mg of iron element.
• Пищевая добавка Sideral forte® VERUM drops
  Patients with anemia due to iron deficiency: * From 8 years until development (Tanner stage \<=3): 2 ml of Sideral forte® VERUM drops, equal to 28 mg of iron element; * From development (Tanner stage \>3) up to 18 years: 3 ml of SiderAL FORTE oral drops, equal to 42 mg of iron element.
• Пищевая добавка Sideral forte® matching PLACEBO drops
  Patients with hypoferritinemia (no anemia): * From 8 years until development (Tanner stage \<=3): 1 ml/day of PLACEBO drops; * From development (Tanner stage \>3) up to 18 years: 2 ml/day of of PLACEBO drops.
• Пищевая добавка Sideral forte® matching PLACEBO drops
  Patients with anemia due to iron deficiency: * From 8 years until development (Tanner stage \<=3): 2 ml of PLACEBO drops; * From development (Tanner stage \>3) up to 18 years: 3 ml of PLACEBO drops.

Первичные конечные точки

• Time to normalization of iron status [Срок оценки: From enrollment to the end of the treatment at 6 months]

Вторичные конечные точки (12)

• Change in hemoglobin [Срок оценки: Baseline to 6 months]
• Change in serum ferritin [Срок оценки: Baseline to 6 months]
• Change in mean corpuscular volume (MCV) [Срок оценки: Baseline to 6 months]
• Change in mean corpuscular hemoglobin (MCH) [Срок оценки: Baseline to 6 months]
• Change in mean corpuscular hemoglobin concentration (MCHC) [Срок оценки: Baseline to 6 months]
• Change in reticulocyte count [Срок оценки: Baseline to 6 months]
• Change in serum iron [Срок оценки: Baseline to 6 months]
• Change in transferrin saturation [Срок оценки: Baseline to 6 months]
• Change in vitamin B12 [Срок оценки: Baseline to 6 months]
• Change in folate [Срок оценки: Baseline to 6 months]
• Change in fatigue score assessed by PedsQL™ Multidimensional Fatigue Scale [Срок оценки: From enrollment to the end of the treatment at 6 months]
• Changes from baseline in disease-specific quality of life measured by Coeliac Disease Dutch Questionnaire (CDDUX) [Срок оценки: From enrollment to the end of the treatment at 6 months]

Критерии участия

Критерии включения

• Diagnosis of CD according to the current European ESPGHAN guidelines (clinical or histological) with confirmed hypoferritinemia or iron deficiency anemia.
• Age at diagnosis of CD between 8 and 18 years (inclusive).
• Absence of oral martial supplementation in the 30 days before the diagnosis and intravenous martial supplementation in the 90 days prior to the diagnosis of CD.
• Patients who have not already started GFD before diagnosis.
• Exclusion of other causes of anemia.
• Patients (and parents/legal guardian) able to understand and willing to participate in the study, with collaborative attitude.
• Informed consent release by both parents/legal guardian.

Критерии исключения

• Potential celiac disease.
• Hb < 8 g/dL at screening
• Other causes of anemia, hemoglobinopathies or coagulopathies.
• Active bleeding or surgery or major trauma in the last 6 months.
• Other inflammatory diseases, neoplasms or IgE mediated food allergies
• Syndromes or presence of vascular malformations
• Pregnant or lactating patients (based on self-certification by the parents and by the patient, where applicable)\*
• Patients with known or suspected allergy or hypersensitivity to the study products or any of their excipients.
• Taking oral iron-based medications in the 30 days prior to diagnosis and intravenous iron-based medications in the 90 days prior to diagnosis.
• Use of other investigational drug(s) within 30 days before study entry or during the study.
• Any other condition, illness or treatment that in the Investigator's opinion does not make the patient suitable for the study.

• Self-certification of non-pregnancy status is considered sufficient given that the product under study is a safe and well-tolerated dietary supplement that has already been tested in pregnant women.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Италия · 1 центр

• IRCCS Istituto Giannina Gaslini, pad 16 — Genova

Публикации

• Ministero della Salute "Linee di indirizzo sugli studi condotti per valutare la sicurezza e le proprietà di prodotti alimentari" - Revisione novembre 2018.
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• Varni JW, Burwinkle TM, Katz ER, Meeske K, Dickinson P. The PedsQL in pediatric cancer: reliability and validity of the Pediatric Quality of Life Inventory Generic Core Scales, Multidimensional Fatigue Scale, and Cancer Module. Cancer. 2002 Apr 1;94(7):2090-106. doi: 10.1002/cncr.10428. PMID 11932914
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Идентификаторы

NCT: NCT07563582 · CD-GAS-FESUCR

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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