The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome

Study Overview
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Brief Summary
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Randomized, placebo-controlled trial of oral calcium supplementation for osteopenia in girls and women with Rett syndrome

Detailed Description
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Objective: The investigators conducted a randomized, double-blind, placebo-controlled trial to determine the safety and efficacy of oral calcium supplementation for the treatment of bone mineral deficits in individuals with Rett syndrome (RTT). Methods: The investigators measured total body bone mineral content (BMC) and bone mineral density (BMD) by dual-energy x-ray absorptiometry and biomarkers of bone turnover by clinical laboratory techniques before and one year after oral calcium or placebo supplementation in 32 pre- and post-pubertal girls and young women with RTT. The calcium supplement was calculated to provide a twofold increase in the recommended dietary allowance of calcium for age.

Official Title
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The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome

Conditions
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Rett Syndrome

Intervention / Treatment
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* Dietary Supplement: calcium
* Dietary Supplement: placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria clinical diagnosis of Rett syndrome Exclusion Criteria parathyroid disease renal disease

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 30 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: randomized, double blind
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Rett Active Supplement<br>Females with clinical diagnosis of Rett syndrome treated orally daily with calcium carbonate, dose based on Dietary Reference Intake for age, for one year | Dietary Supplement: calcium<br>* calcium carbonate<br>|
| Placebo Comparator: Rett Placebo Supplement<br>Females with clinical diagnosis of Rett syndrome treated orally daily with sodium bicarbonate, dose based on equivalent weight for active supplement for age, for one year | Dietary Supplement: placebo<br>* sodium bicarbonate<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Bone mineral content (BMC) | Bone mineral content (z-score) measured by dual-energy x-ray absorptiometry | change from baseline to 1 year thereafter | 
| Bone mineral density (BMD) | Bone mineral density (z-score) measured by dual-energy x-ray absorptiometry | change from baseline to 1 year thereafter | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Osteocalcin | Serum Osteocalcin (ng/ml) | change from baseline to 1 year thereafter | 
| Bone alkaline phosphatase | Serum Bone alkaline phosphatase (mcg/ml) | change from baseline to 1 year thereafter | 
| C-telopeptide | Serum C-telopeptide (pg/ml) | change from baseline to 1 year thereafter | 
| Calcium/creatinine ratio | Urinary calcium/creatinine ratio (mg/g) | change from baseline to 1 year thereafter | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Low bone mass