Nutritional Assessment in Patient of Mucopolysaccharide "

NCT07136896 | Not Yet Recruiting

• 1 other identifier: Mucopolysacharidosis
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

" Nutritional assessment in patients of Mucopolysaccharide "

Conditions

Mucopolysaccharidosis (MPS); Malnutrition (Calorie); Undernutrition

Keywords

Caloric deficiency; Pediatric nutrition

Outcome Measures

Primary Outcomes (3)

• Prevalence of malnutrition among patients with mucopolysaccharidosis

  The percentage of patients diagnosed with malnutrition, as determined using the World Health Organization (WHO) growth standards. Malnutrition will be defined based on weight-for-age, height-for-age, and weight-for-height Z-scores below -2 standard deviations. Data will be collected at baseline and expressed as a percentage of the study population.

  1 year

• severity of malnutrition among patients with mucopolysaccharidosisby WHO growth standards Z-scores (weight-for-age, height-for-age, weight-for-height)

  Severity of malnutrition will be assessed using the Gomez classification based on weight-for-age percentage of the median for the reference population. Categories include: mild (75-89%), moderate (60-74%), and severe (\<60%). Data will be expressed as the proportion of patients in each severity category at baseline

  At baseline (upon enrollment)

• Prevalence and severity of malnutrition among patients with mucopolysaccharidosis

  The proportion of patients diagnosed with mucopolysaccharidosis (MPS) who present with any degree of malnutrition, categorized as mild, moderate, or severe according to WHO growth standards. Severity will be determined using anthropometric indices (weight-for-age, height-for-age, BMI-for-age z-scores, and mid-upper arm circumference) and compared across disease subtypes and age groups.

  At baseline (upon enrollment)

Study Arms (1)

Nutritional Assessment in Children with Mucopolysaccharidosis

OTHER

Participants diagnosed with Mucopolysaccharidosis will undergo detailed nutritional assessment, including anthropometric measurements, skinfold thickness evaluation, and bioelectrical impedance analysis. Dietary intake will be recorded, and relevant biochemical parameters will be measured.

Other: Nutritional Assessment Protocol

Interventions

Nutritional Assessment Protocol OTHER

Participants will undergo a comprehensive nutritional assessment, including anthropometric measurements (weight, height, BMI, skinfold thickness), dietary intake evaluation through 24-hour recall, and bioelectrical impedance analysis to assess body composition. Relevant biochemical parameters will also be measured to evaluate nutritional status

Nutritional Assessment in Children with Mucopolysaccharidosis

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age between 1 and 18 years
• Stable clinical condition at time of enrollment
• Informed consent obtained from parent/guardian, and assent from child when applicable -

You may not qualify if:

• Acute illness or hospitalization within the last 2 weeks prior to assessment
• Patients who received nutritional supplementation or dietary intervention within 3 months prior to enrollment that could affect baseline nutritional assessment
• Refusal or inability to comply with study assessments

Sponsors & Collaborators

• Assiut University: lead

MeSH Terms

Conditions

Mucopolysaccharidoses; Malnutrition

Condition Hierarchy (Ancestors)

Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Nutrition Disorders

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator,

Model Details: Participants will be assigned to one of two parallel groups: children with mucopolysaccharidosis (MPS) and age-matched healthy controls. Both groups will undergo the same nutritional assessment protocol for comparison.

Study Record Dates

• First Submitted: August 9, 2025
• First Posted: August 22, 2025
• Study Start: August 16, 2025
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): August 30, 2026
• Last Updated: August 22, 2025

Record last verified: 2025-08