Efficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis Imperfecta

Efficacy and Safety of Alendronate in Chinese Children or Adolescents With Osteogenesis Imperfecta: an Age Stratified Prospective Study

Alendronate should be considered as an alternative therapy of osteogenesis imperfecta (OI) because it significantly increased areal bone mineral density (BMD) and its Z score, decreased fracture incidence, inhibited bone resorption biomarkers. Alendronate exerted beneficial roles in different age brackets, especially in young patients with OI.

Study Overview

• Status: Completed
• Conditions: Osteogenesis Imperfecta
• Intervention / Treatment: Drug: Alendronate

• Study Type: Interventional
• Enrollment (Actual): 99
• Phase: Phase 4

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. children or adolescents aged 0-18 years,
2. had either a history of at least once minor-impact fracture or age and sex adjusted areal BMD Z score of -1.0 or less at lumbar spine or total hip;
3. with or without blue sclera, impaired hearing, joint hypermobility or dentinogenesis imperfecta;
4. with or without slim long bone; with or without cranial epactal bones, signs of multiple fractures, bony deformity in skeletal X-ray films.

Exclusion Criteria:

1. previous history of rickets, hyperparathyroidism, other metabolic or inherited bone diseases; malignant disease; coeliac disease; hyperthyroidism;
2. therapy history of BPs within recent two years; severe renal failure (creatinine clearance <40 ml/min), chronic liver disease; severe diseases of gastrointestinal tract;
3. unable to keep upright for at least 30 minutes daily .

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: an open label, prospective, self-controlled study; Children or adolescents under the age of 18 years old with minor trauma fracture were recruited from 30 provinces of China. The diagnosis of OI was made by endocrinology department of Peking Union Medical College Hospital(PUMCH).	Drug: Alendronate; Alendronate was administrated as 70 mg/week orally (Fosamax, Merck Sharp & Dohme．LTD.).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
changes from baseline of areal BMD at lumbar spine and total hip	baseline and 12,24,36 months
annual clinical fracture incidence	baseline and 12,24,36 months

Secondary Outcome Measures

Outcome Measure	Time Frame
changes of bone turnover biomarkers	baseline and 6,12,24,36 months
changes of height	baseline and 12,24,36 months

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital
• Collaborators: National Natural Science Foundation of China

Study record dates

Study Major Dates

• Study Start: March 1, 2007
• Primary Completion (Actual): July 1, 2011
• Study Completion (Actual): August 1, 2014

Study Registration Dates

• First Submitted: November 19, 2014
• First Submitted That Met QC Criteria: November 25, 2014
• First Posted (Estimate): December 1, 2014

Study Record Updates

• Last Update Posted (Estimate): December 10, 2014
• Last Update Submitted That Met QC Criteria: December 9, 2014
• Last Verified: November 1, 2014

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Musculoskeletal Diseases; Connective Tissue Diseases; Bone Diseases; Bone Diseases, Developmental; Osteochondrodysplasias; Collagen Diseases; Osteogenesis Imperfecta; Physiological Effects of Drugs; Bone Density Conservation Agents; Alendronate
• Other Study ID Numbers: No. 2007-03-12