Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia: a Double-blind, Randomized, Placebo-controlled Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Time to normalization of iron status
研究概览
简要总结
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Patients will be randomly assigned to one of the following treatment groups:
- Sideral forte verum drops for oral intake in addition to GFD.
- Sideral forte matching placebo drops for oral intake in addition to GFD. Randomization will be stratified in order to reach a balanced enrolment between patients with hypoferritinemia and patients with anemia due to iron deficiency. Patients will start the study treatment simultaneously with GFD start, after their eligibility is confirmed at Visit 1 (screening) and will continue for 6 months.
All patients will be treated with GFD that, so far, is considered the Standard of Care for CD.
The Principal Investigator will receive a study treatment identification key in the form of a sealed envelope containing the kit number (i.e. Kit code A-004 or Kit code B-001, etc.) and the corresponding treatment.
The envelope may be opened only in case of an emergency where the identification of the study treatment assigned to the patient needs to be disclosed
入排标准
- 年龄范围
- 8 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Sideral forte® VERUM drops for oral intake in addition to GFD
干预措施: Sideral forte® VERUM drops (Dietary Supplement)
Sideral forte® matching PLACEBO drops for oral intake in addition to GFD
干预措施: Sideral forte® matching PLACEBO drops (Dietary Supplement)
结局指标
主要结局
Time to normalization of iron status
时间窗: From enrollment to the end of the treatment at 6 months
Time from baseline (defined as the time from diagnosis of celiac disease) to the first documented normalization of iron status. Normalization is defined as: * hemoglobin (Hb) within age- and sex-specific reference ranges in participants with iron deficiency anemia at baseline, or * serum ferritin within reference ranges in participants with isolated hypoferritinemia at baseline Comparisons will be performed between participants receiving oral Sucrosomial® iron supplementation plus a gluten-free diet (GFD) and those receiving GFD alone.
次要结局
- Change in hemoglobin(Baseline to 6 months)
- Change in serum ferritin(Baseline to 6 months)
- Change in mean corpuscular volume (MCV)(Baseline to 6 months)
- Change in mean corpuscular hemoglobin (MCH)(Baseline to 6 months)
- Change in mean corpuscular hemoglobin concentration (MCHC)(Baseline to 6 months)
- Change in reticulocyte count(Baseline to 6 months)
- Change in serum iron(Baseline to 6 months)
- Change in transferrin saturation(Baseline to 6 months)
- Change in vitamin B12(Baseline to 6 months)
- Change in folate(Baseline to 6 months)
- Change in fatigue score assessed by PedsQL™ Multidimensional Fatigue Scale(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)(From enrollment to the end of the treatment at 6 months)
- Changes from baseline in generic health-related quality of life measured by Pediatric Quality of Life Inventory (PedsQL™ 4.0)(From enrollment to the end of treatment (6 months))
- Adherence to GFD(From enrollment to the end of the treatment at 6 months)
- Changes from baseline in gastrointestinal symptoms assessed with PedsQL™ 3.0 Gastrointestinal Symptoms Module score(From enrollment to the end of the treatment at 6 months)
- Number and proportion of participants with treatment-related adverse events, graded according to CTCAE v5.0, during Sucrosomial® iron supplementation(From enrollment to the end of the treatment at 6 months)