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

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

Status and phase: Unknown; Phase 4

Conditions: Osteopenia; Osteoporosis; Bullosa Epidermolysis; Phosphorus and Calcium Disorders; Vitamin D Deficiency

Treatments: Dietary Supplement: Oral nutritional supplement; Drug: Vitamin D

Study type: Interventional

Funder types: Other

Identifiers

NCT05141838

307308294

Details and patient eligibility

About

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.

Full 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.

Enrollment: 110 estimated patients

Sex: All

Ages: Under 17 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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110 participants in 4 patient groups

Group 1 - Vitamin D

Experimental group

Description:

Patients receiving vitamin D supplements in therapy

Treatment:

Drug: Vitamin D

Group 2 - Oral nutritional supplement

Experimental group

Description:

Patients receiving oral nutritional supplement

Treatment:

Dietary Supplement: Oral nutritional supplement

Group 3 - Vitamin D+Oral nutritional supplement

Experimental group

Description:

Patients receiving vitamin D supplements and oral nutritional supplement in therapy

Treatment:

Drug: Vitamin D

Dietary Supplement: Oral nutritional supplement

Group 4 - No intervention

No Intervention group

Description:

Patients with dystrophic form of congenital epidermolysis bullosa who have not taken vitamin D supplements and/or oral nutritional supplement