Official Title: “A Non-Randomized, Open-Label, Prospective, Non-Controlled, 12-Month Clinical Trial to Determine the Effects of Alendronate 35 or 70 mg/Week Depending Upon Body Weight, in Children and Adolescent With IJO”

OBJECTIVES:

I. Determine the effects of alendronate sodium on skeletal remodeling and bone mineral density of the hip and spine in children with high-turnover idiopathic juvenile osteoporosis.

Study Type: Interventional

Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Study Primary Completion Date: October 2003

PROTOCOL OUTLINE:

Patients receive oral alendronate sodium daily for 1 year. Treatment continues in the absence of disease progression or unacceptable toxicity.

Intervention(s) in this Clinical Trial

• Drug: alendronate
  • Pill, 35mg or 70mg weekly, depending upon the body weight for 12 months.

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: 1
  • Ten children will take alendronate 35mg or 70mg weekly depending upon the body weight for 12 months. Patients will also take calcium suplement daily.

Primary Measures

• Bone Mineral Density (BMD) of the lumbar spine and hip at 12 months.
  • Time Frame: at 12 months
    Safety Issue?: Yes

Secondary Measures

• Fracture rate before and during therapy; bone biopsies and biochemical markers to determine whether or not the primary effect of therapy is on bone formation or resorption; bone biopsies will also be used to assess the safety.
  • Time Frame: at 12 months
    Safety Issue?: Yes

Eligibility Criteria:

• 5-14 years of age
• Weight 20 kg or greater
• History of one or more atraumatic fracture
• Sexual development no greater than Tanner II
• Osteoporosis by DXA (Diagnosis of high-turnover osteoporosis with no underlying cause (e.g., malignancy, hyperthyroidism, hyperparathyroidism, or vitamin D intoxication)

Inclusion Criteria:

• Male and female children with a history of one or more atraumatic fractures, or evidence of one or more compression fractures on radiographs of the spine (reduction of >20%).
• Bone mineral density by DXA at 2 standard deviations (SD) below normal mean for age (Z-score at least 2 SD below normal mean at the lumbar spine or hip)
• Parental consent (and patient assent after age 12 years) to participate in the study.
• Sexual development at Tanner stage II or less (Prepubertal stage)
• Weight 20kg and more

Exclusion Criteria:

• History of severe gastritis or reflux
• Marked kyphoscoliosis or inability to sit or stand for at least 30 minutes.
• Abnormalities of the esophagus that delay emptying (e.g., strictures or achalasia)
• Hypersensitivity to bisphosphonates
• Uncorrected hypocalcemia
• History of gastric or duodenal ulcers
• Renal dysfunction as indicated by serum Creatinine greater than 1.5 mg/dL
• Liver dysfunction as indicated by serum SGPT greater than 2 times upper limit of normal for age or serum total bilirubin greater than 2.0 mg/dL
• Diagnosis of osteogenesis imperfecta (including family history) or blue sclerae or deafness
• Diagnosis of active rickets, osteomalacia, or bone alkaline phosphatase > 2 times normal for age
• Severe gastritis or reflux
• Pregnancy
• Anorexia Nervosa
• Prior/Concurrent Therapy—
• Prior course of prednisone allowed
• No concurrent prednisone except inhaled steroids
• No concurrent high-dose glucocorticoids
• No concurrent salmon calcitonin
• No other concurrent bisphosphonates
• No concurrent long-term anti-seizure medication

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 5 Years

Maximum Age for this Clinical Trial: 14 Years

Are Healthy Volunteers Accepted for this Clinical Trial?: No

Lead Sponsor: FDA Office of Orphan Products Development

Overall Clinical Trial Officials and Contacts

L. Lyndon Key, Jr. Study Chair Medical University of South Carolina  

Information obtained from ClinicalTrials.gov on August 28, 2008

Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00010439

Study ID Number: 199/15705

ClinicalTrials.gov Identifier: NCT00010439

Health Authority: United States: Food and Drug Administration

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