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

• ClinicalTrials.gov Identifier: NCT05352373
• Recruitment Status: Completed
• First Posted: April 28, 2022
• Last Update Posted: April 28, 2022
• Sponsor: Baylor College of Medicine
• Information provided by (Responsible Party): Kathleen J Motil, Baylor College of Medicine

Tracking Information

• First Submitted Date: April 15, 2022
• First Posted Date: April 28, 2022
• Last Update Posted Date: April 28, 2022
• Actual Study Start Date: March 1, 2008
• Actual Primary Completion Date: March 1, 2012 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 27, 2022)

• Bone mineral content (BMC) [ Time Frame: change from baseline to 1 year thereafter ]
  Bone mineral content (z-score) measured by dual-energy x-ray absorptiometry
• Bone mineral density (BMD) [ Time Frame: change from baseline to 1 year thereafter ]
  Bone mineral density (z-score) measured by dual-energy x-ray absorptiometry

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted

Current Secondary Outcome Measures (submitted: April 27, 2022)

• Osteocalcin [ Time Frame: change from baseline to 1 year thereafter ]
  Serum Osteocalcin (ng/ml)
• Bone alkaline phosphatase [ Time Frame: change from baseline to 1 year thereafter ]
  Serum Bone alkaline phosphatase (mcg/ml)
• C-telopeptide [ Time Frame: change from baseline to 1 year thereafter ]
  Serum C-telopeptide (pg/ml)
• Calcium/creatinine ratio [ Time Frame: change from baseline to 1 year thereafter ]
  Urinary calcium/creatinine ratio (mg/g)

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome
• Official Title: The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome
• Brief Summary: Randomized, placebo-controlled trial of oral calcium supplementation for osteopenia in girls and women with Rett syndrome

Detailed Description

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.

• Study Type: Interventional
• Study Phase: Not Applicable

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

randomized, double blind

Masking: Double (Participant, Investigator)
Primary Purpose: Treatment

• Condition: Rett Syndrome

Intervention

• Dietary Supplement: calcium
  calcium carbonate
• Dietary Supplement: placebo
  sodium bicarbonate

Study Arms

• Active Comparator: Rett Active Supplement
  Females with clinical diagnosis of Rett syndrome treated orally daily with calcium carbonate, dose based on Dietary Reference Intake for age, for one year
  Intervention: Dietary Supplement: calcium
• Placebo Comparator: Rett Placebo Supplement
  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
  Intervention: Dietary Supplement: placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 27, 2022): 32
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: March 1, 2012
• Actual Primary Completion Date: March 1, 2012 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria

• clinical diagnosis of Rett syndrome

Exclusion Criteria

• parathyroid disease
• renal disease

Sex/Gender

• Sexes Eligible for Study: Female

• Ages: 5 Years to 30 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT05352373
• Other Study ID Numbers: H-19205
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Kathleen J Motil, Baylor College of Medicine
• Original Responsible Party: Same as current
• Current Study Sponsor: Baylor College of Medicine
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Kathleen J Motil, MD, PHD; Baylor College of Medicine

• PRS Account: Baylor College of Medicine
• Verification Date: April 2022