Vitamin D Status and Bone Metabolism Status in Children With Congenital Epidermolysis Bullosa

Phase 4

• Conditions: Bullosa Epidermolysis; Vitamin D Deficiency; Osteoporosis; Osteopenia; Phosphorus and Calcium Disorders
• Interventions: Drug: Vitamin D; Dietary Supplement: Oral nutritional supplement

• Registration Number: NCT05141838
• Lead Sponsor: National Medical Research Center for Children's Health, Russian Federation

Brief Summary

This retrospective prospective study is aimed at studying the level of vitamin D supply and identifying markers of bone tissue remodeling in order to develop approaches to the prevention of osteopenia and osteoporosis in children with congenital epidermolysis bullosa.

Detailed Description

This retrospective prospective interventional study will examine vitamin D availability and its relationship with clinical disease, patient gender and age, and season. The mechanisms of impairment of phosphorus-calcium metabolism and bone tissue metabolism will be analyzed on the basis of biochemical parameters and instrumental research methods in children with dystrophic epidermolysis bullosa in order to develop personalized approaches for correcting vitamin D status and bone metabolism disorders in children with epidermolysis bullosa, followed by an assessment of their clinical efficacy.

Study Timeline

• Study Start: 2020-11-21
• Study First Submitted: 2021-11-19
• Study First Submitted QC: 2021-11-19
• Status Verified: 2021-12
• Study First Posted: 2021-12-02
• Last Update Submitted: 2021-12-02
• Last Update Posted: 2021-12-17
• Primary Completion: 2023-01-01
• Study Completion: 2023-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

• Verified diagnosis of epidermolysis bullosa;
• Signing by parents (legal representatives) of informed consent to participate in the study and fulfill the requirements of the study

Exclusion Criteria

• not planned

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Group 1 - Vitamin D	Vitamin D	Patients receiving vitamin D supplements in therapy
Group 2 - Oral nutritional supplement	Oral nutritional supplement	Patients receiving oral nutritional supplement
Group 3 - Vitamin D+Oral nutritional supplement	Vitamin D	Patients receiving vitamin D supplements and oral nutritional supplement in therapy
Group 3 - Vitamin D+Oral nutritional supplement	Oral nutritional supplement	Patients receiving vitamin D supplements and oral nutritional supplement in therapy

Primary Outcome Measures

Name	Time	Method
Phosphorus-calcium metabolism assessment	24 week	Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood
Vitamin D level	24 week	Study of the provision of vitamin D in children with dystrophic and simple forms of epidermolysis bullosa based on the level of 25 (OH) vitamin D (25 hydroxycholecalciferol) in the blood
Assessment of the state of bone tissue	24 week	Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood
Assessment of indicators of physical development	24 wee	Z-score calculation (weight-for-height z-score)

Secondary Outcome Measures

Name	Time	Method
Assessment of bone mineral density	24 week	dual-energy X-ray densitometry
Bone age assessment	24 week	X-ray of the bones of the hand with an assessment of bone age

Trial Locations

Locations (1)

National Medical Research Center for Children's Health; 🇷🇺 Moscow, Russian Federation