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

Primary Purpose

Bullosa Epidermolysis, Vitamin D Deficiency, Osteoporosis

Status
Recruiting
Phase
Phase 4
Locations
Russian Federation
Study Type
Interventional
Intervention
Vitamin D
Oral nutritional supplement
Sponsored by
National Medical Research Center for Children's Health, Russian Federation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Bullosa Epidermolysis focused on measuring Children, Only

Eligibility Criteria

0 Years - 17 Years (Child)All SexesDoes not accept 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

Sites / Locations

  • National Medical Research Center for Children's HealthRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

No Intervention

Arm Label

Group 1 - Vitamin D

Group 2 - Oral nutritional supplement

Group 3 - Vitamin D+Oral nutritional supplement

Group 4 - No intervention

Arm Description

Patients receiving vitamin D supplements in therapy

Patients receiving oral nutritional supplement

Patients receiving vitamin D supplements and oral nutritional supplement in therapy

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

Outcomes

Primary Outcome Measures

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
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
Phosphorus-calcium metabolism assessment
Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood
Phosphorus-calcium metabolism assessment
Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood
Assessment of the state of bone tissue
Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood
Assessment of the state of bone tissue
Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood
Assessment of indicators of physical development
Z-score calculation (weight-for-height z-score)
Assessment of indicators of physical development
Z-score calculation (weight-for-height z-score)

Secondary Outcome Measures

Assessment of bone mineral density
dual-energy X-ray densitometry
Assessment of bone mineral density
dual-energy X-ray densitometry
Bone age assessment
X-ray of the bones of the hand with an assessment of bone age
Bone age assessment
X-ray of the bones of the hand with an assessment of bone age

Full Information

First Posted
November 19, 2021
Last Updated
December 2, 2021
Sponsor
National Medical Research Center for Children's Health, Russian Federation
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1. Study Identification

Unique Protocol Identification Number
NCT05141838
Brief Title
Vitamin D Status and Bone Metabolism Status in Children With Congenital Epidermolysis Bullosa
Official Title
Vitamin D Status and Bone Metabolism Status in Children With Congenital Epidermolysis Bullosa
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Recruiting
Study Start Date
November 21, 2020 (Actual)
Primary Completion Date
January 1, 2023 (Anticipated)
Study Completion Date
January 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Medical Research Center for Children's Health, Russian Federation

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Bullosa Epidermolysis, Vitamin D Deficiency, Osteoporosis, Osteopenia, Phosphorus and Calcium Disorders
Keywords
Children, Only

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
110 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Group 1 - Vitamin D
Arm Type
Experimental
Arm Description
Patients receiving vitamin D supplements in therapy
Arm Title
Group 2 - Oral nutritional supplement
Arm Type
Experimental
Arm Description
Patients receiving oral nutritional supplement
Arm Title
Group 3 - Vitamin D+Oral nutritional supplement
Arm Type
Experimental
Arm Description
Patients receiving vitamin D supplements and oral nutritional supplement in therapy
Arm Title
Group 4 - No intervention
Arm Type
No Intervention
Arm Description
Patients with dystrophic form of congenital epidermolysis bullosa who have not taken vitamin D supplements and/or oral nutritional supplement
Intervention Type
Drug
Intervention Name(s)
Vitamin D
Intervention Description
Adding vitamin D preparations to the therapy in the individual required dosage to correct the deficient state
Intervention Type
Dietary Supplement
Intervention Name(s)
Oral nutritional supplement
Intervention Description
Adding оral nutritional supplement to the therapy depending on the degree of protein-energy malnutrition
Primary Outcome Measure Information:
Title
Vitamin D level
Description
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
Time Frame
Baseline
Title
Vitamin D level
Description
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
Time Frame
24 week
Title
Phosphorus-calcium metabolism assessment
Description
Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood
Time Frame
Baseline
Title
Phosphorus-calcium metabolism assessment
Description
Study of levels of calcium, phosphorus, parathyroid hormone, creatinine, magnesium, albumin in the blood
Time Frame
24 week
Title
Assessment of the state of bone tissue
Description
Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood
Time Frame
Baseline
Title
Assessment of the state of bone tissue
Description
Study of levels of alkaline phosphatase, osteocalcin, b-CrossLaps, P1NP in the blood
Time Frame
24 week
Title
Assessment of indicators of physical development
Description
Z-score calculation (weight-for-height z-score)
Time Frame
Baseline
Title
Assessment of indicators of physical development
Description
Z-score calculation (weight-for-height z-score)
Time Frame
24 wee
Secondary Outcome Measure Information:
Title
Assessment of bone mineral density
Description
dual-energy X-ray densitometry
Time Frame
Baseline
Title
Assessment of bone mineral density
Description
dual-energy X-ray densitometry
Time Frame
24 week
Title
Bone age assessment
Description
X-ray of the bones of the hand with an assessment of bone age
Time Frame
Baseline
Title
Bone age assessment
Description
X-ray of the bones of the hand with an assessment of bone age
Time Frame
24 week

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
National Medical Research Center for Children's Health
City
Moscow
ZIP/Postal Code
119296
Country
Russian Federation
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Irina Yu. Pronina
Phone
+7(916)934-40-81
Email
krapchatovaiv@yandex.ru

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

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

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