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

• Sponsor: National Medical Research Center for Children's Health, Russian Federation (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT05141838
• Collaborator: (none)
• Enrollment: 110
• Location: 1
• Arms: 4
• Anticipated Duration (Months): 25.3
• Patients Per Site Per Month: 4.3

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Bullosa Epidermolysis; Vitamin D Deficiency; Osteoporosis; Osteopenia; Phosphorus and Calcium Disorders	Drug: Vitamin D; Dietary Supplement: Oral nutritional supplement	Phase 4

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 110 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Vitamin D Status and Bone Metabolism Status in Children With Congenital Epidermolysis Bullosa
• Actual Study Start Date: Nov 21, 2020
• Anticipated Primary Completion Date: Jan 1, 2023
• Anticipated Study Completion Date: Jan 1, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Vitamin D level [Baseline]

   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

2. 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

3. Phosphorus-calcium metabolism assessment [Baseline]

   Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood

4. Phosphorus-calcium metabolism assessment [24 week]

   Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood

5. Assessment of the state of bone tissue [Baseline]

   Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood

6. Assessment of the state of bone tissue [24 week]

   Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood

7. Assessment of indicators of physical development [Baseline]

   Z-score calculation (weight-for-height z-score)

8. Assessment of indicators of physical development [24 wee]

   Z-score calculation (weight-for-height z-score)

Secondary Outcome Measures

1. Assessment of bone mineral density [Baseline]

   dual-energy X-ray densitometry

2. Assessment of bone mineral density [24 week]

   dual-energy X-ray densitometry

3. Bone age assessment [Baseline]

   X-ray of the bones of the hand with an assessment of bone age

4. Bone age assessment [24 week]

   X-ray of the bones of the hand with an assessment of bone age

Eligibility Criteria

Criteria

• Ages Eligible for Study: 0 Years to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	National Medical Research Center for Children's Health	Moscow		Russian Federation	119296

Sponsors and Collaborators

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

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: National Medical Research Center for Children's Health, Russian Federation
• ClinicalTrials.gov Identifier: NCT05141838
• Other Study ID Numbers: 307308294
• First Posted: Dec 2, 2021
• Last Update Posted: Dec 17, 2021
• Last Verified: Dec 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by National Medical Research Center for Children's Health, Russian Federation: Children, Only
• Additional relevant MeSH terms: Osteoporosis; Bone Diseases, Metabolic; Epidermolysis Bullosa; Vitamin D Deficiency; Vitamin D

Study Results

No Results Posted as of Dec 17, 2021