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

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.

Study Overview

• Status: Recruiting
• Conditions: Vitamin D Deficiency; Osteoporosis; Osteopenia; Phosphorus and Calcium Disorders; Bullosa Epidermolysis
• Intervention / Treatment: Drug: Vitamin D; Dietary supplement: Oral nutritional supplement

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 Type: Interventional
• Enrollment (Anticipated): 110
• Phase: Phase 4

Contacts and Locations

Study Locations

• Russian Federation
  • Moscow, Russian Federation, 119296
    • Recruiting
    • National Medical Research Center for Children's Health
    • Contact: Irina Yu. Pronina; Phone Number: +7(916)934-40-81; Email: krapchatovaiv@yandex.ru

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Group 1 - Vitamin D; Patients receiving vitamin D supplements in therapy	Drug: Vitamin D; Adding vitamin D preparations to the therapy in the individual required dosage to correct the deficient state
EXPERIMENTAL: Group 2 - Oral nutritional supplement; Patients receiving oral nutritional supplement	Dietary supplement: Oral nutritional supplement; Adding оral nutritional supplement to the therapy depending on the degree of protein-energy malnutrition
EXPERIMENTAL: Group 3 - Vitamin D+Oral nutritional supplement; Patients receiving vitamin D supplements and oral nutritional supplement in therapy	Drug: Vitamin D; Adding vitamin D preparations to the therapy in the individual required dosage to correct the deficient state; Dietary supplement: Oral nutritional supplement; Adding оral nutritional supplement to the therapy depending on the degree of protein-energy malnutrition
NO_INTERVENTION: Group 4 - No intervention; Patients with dystrophic form of congenital epidermolysis bullosa who have not taken vitamin D supplements and/or oral nutritional supplement

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Vitamin D level	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	Baseline
Vitamin D level	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	24 week
Phosphorus-calcium metabolism assessment	Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood	Baseline
Phosphorus-calcium metabolism assessment	Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood	24 week
Assessment of the state of bone tissue	Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood	Baseline
Assessment of the state of bone tissue	Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood	24 week
Assessment of indicators of physical development	Z-score calculation (weight-for-height z-score)	Baseline
Assessment of indicators of physical development	Z-score calculation (weight-for-height z-score)	24 wee

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assessment of bone mineral density	dual-energy X-ray densitometry	Baseline
Assessment of bone mineral density	dual-energy X-ray densitometry	24 week
Bone age assessment	X-ray of the bones of the hand with an assessment of bone age	Baseline
Bone age assessment	X-ray of the bones of the hand with an assessment of bone age	24 week

Collaborators and Investigators

• Sponsor: National Medical Research Center for Children's Health, Russian Federation

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 21, 2020
• Primary Completion (ANTICIPATED): January 1, 2023
• Study Completion (ANTICIPATED): January 1, 2023

Study Registration Dates

• First Submitted: November 19, 2021
• First Submitted That Met QC Criteria: November 19, 2021
• First Posted (ACTUAL): December 2, 2021

Study Record Updates

• Last Update Posted (ACTUAL): December 17, 2021
• Last Update Submitted That Met QC Criteria: December 2, 2021
• Last Verified: December 1, 2021

More Information

Terms related to this study

• Keywords: Children, Only
• Additional Relevant MeSH Terms: Metabolic Diseases; Skin Diseases; Congenital Abnormalities; Nutrition Disorders; Genetic Diseases, Inborn; Musculoskeletal Diseases; Skin Diseases, Genetic; Avitaminosis; Deficiency Diseases; Malnutrition; Bone Diseases; Skin Diseases, Vesiculobullous; Skin Abnormalities; Vitamin D Deficiency; Osteoporosis; Bone Diseases, Metabolic; Epidermolysis Bullosa; Physiological Effects of Drugs; Micronutrients; Vitamins; Bone Density Conservation Agents; Vitamin D
• Other Study ID Numbers: 307308294

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No