NCT05141838

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
110

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Nov 2020

Typical duration for phase_4

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 21, 2020

Completed
12 months until next milestone

First Submitted

Initial submission to the registry

November 19, 2021

Completed
13 days until next milestone

First Posted

Study publicly available on registry

December 2, 2021

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2023

Completed
Last Updated

December 17, 2021

Status Verified

December 1, 2021

Enrollment Period

2.1 years

First QC Date

November 19, 2021

Last Update Submit

December 2, 2021

Conditions

Bullosa EpidermolysisVitamin D DeficiencyOsteoporosisOsteopeniaPhosphorus and Calcium Disorders

Keywords

Children, Only

Outcome Measures

Primary Outcomes (8)

  • 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 Outcomes (4)

  • Assessment of bone mineral density

    Baseline

  • Assessment of bone mineral density

    24 week

  • Bone age assessment

    Baseline

  • Bone age assessment

    24 week

Study Arms (4)

Group 1 - Vitamin D

EXPERIMENTAL

Patients receiving vitamin D supplements in therapy

Drug: Vitamin D

Group 2 - Oral nutritional supplement

EXPERIMENTAL

Patients receiving oral nutritional supplement

Dietary Supplement: Oral nutritional supplement

Group 3 - Vitamin D+Oral nutritional supplement

EXPERIMENTAL

Patients receiving vitamin D supplements and oral nutritional supplement in therapy

Drug: Vitamin DDietary Supplement: Oral nutritional supplement

Group 4 - No intervention

NO INTERVENTION

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

Interventions

Vitamin DDRUG

Adding vitamin D preparations to the therapy in the individual required dosage to correct the deficient state

Group 1 - Vitamin DGroup 3 - Vitamin D+Oral nutritional supplement
Oral nutritional supplementDIETARY_SUPPLEMENT

Adding оral nutritional supplement to the therapy depending on the degree of protein-energy malnutrition

Group 2 - Oral nutritional supplementGroup 3 - Vitamin D+Oral nutritional supplement

Eligibility Criteria

Age0 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • not planned

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Medical Research Center for Children's Health

Moscow, 119296, Russia

RECRUITING

MeSH Terms

Conditions

Epidermolysis BullosaVitamin D DeficiencyOsteoporosisBone Diseases, Metabolic

Interventions

Vitamin DDietary Supplements

Condition Hierarchy (Ancestors)

Skin AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornSkin DiseasesSkin and Connective Tissue DiseasesSkin Diseases, VesiculobullousAvitaminosisDeficiency DiseasesMalnutritionNutrition DisordersNutritional and Metabolic DiseasesBone DiseasesMusculoskeletal DiseasesMetabolic Diseases

Intervention Hierarchy (Ancestors)

SecosteroidsSteroidsFused-Ring CompoundsPolycyclic CompoundsFoodDiet, Food, and NutritionPhysiological PhenomenaFood and Beverages

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 19, 2021

First Posted

December 2, 2021

Study Start

November 21, 2020

Primary Completion

January 1, 2023

Study Completion

January 1, 2023

Last Updated

December 17, 2021

Record last verified: 2021-12

Locations

RU(1)