Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis
Information source: FDA Office of Orphan Products Development
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Osteoporosis
Intervention: Alendronate (Drug)
Phase: Phase 2
Status: Active, not recruiting
Sponsored by: FDA Office of Orphan Products Development Official(s) and/or principal investigator(s):
Lyndon L Key, M.D., Principal Investigator, Affiliation: Medical University of South Carolina
Summary
We have previously evaluated the safety and efficacy of Fosamax in 10 patients with juvenile
osteoporosis during a 12-month clinical trial. We have documented that Fosamax improved BMD
of the spine and hip without any major side effects. There were no additional fractures
during therapy. The present study is designed to further evaluate the safety and efficacy of
Fosamax in 20 children with juvenile osteoporosis using a double-blind, randomized,
placebo-controlled, cross-over protocol.
Clinical Details
Official title: A Randomized, Double-Blind, Placebo-Controlled, Prospective, Cross-Over Phase II Clinical Trial to Determine the Safety and Efficacy of Alendronate (Fosamax) in Juvenile Osteoporosis (IND#60,017)
Study design: Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Crossover Assignment, Safety/Efficacy Study
Primary outcome: Bone mineral density (BMD) of the lumbar spine at 12 and 24 months at 12 months
Secondary outcome: Bone Mineral Density of Hip at 12 and 24 months. Fracture rate before and during therapy. Biochemical markers to determine whether the primary effect of therapy is on bone formation or resorption.
Detailed description:
Osteoporosis is an uncommon disease in children and early adolescents. Patients have a low
bone mineral density, develop fractures with minimal or no trauma, and frequently have a
negative family history. The disease results from either diminished bone formation or
increased bone removal (resorption). No specific drug therapy has been recommended for
juvenile osteoporosis. Alendronate (Fosamax) is effective in inhibiting bone resorption,
increasing BMD and reducing fractures in adults with postmenopausal osteoporosis, but have
not become established therapies in children. In the present study, we plan to evaluate the
safety and efficacy of Fosamax in 20 patients with juvenile osteoporosis in a two-year
period. This is a randomized, double-blind, placebo-controlled protocol. In the year-1, 10
patients will be assigned to receive Fosamax and 10 patients placebo. In the year-2, patients
will be crossed over to the second arm of the study. Those who received Fosamax in the
year-1, will receive placebo in the second year and vice versa. The patients will have 5
visits, the initial screening visit followed by 4 post therapy visits in a six-month
interval. Measurements include DEXA of spine and hip, urinalysis and blood work.
Eligibility
Minimum age: 5 Years.
Maximum age: 15 Years.
Gender(s): Both.
Criteria:
Eligibility Criteria:
- 5-15 yrs of age
- Weighing 20 kg and more
- History of multiple fractures
- Tanner stage II or less
- Osteoporosis by DEXA (normative data available for these age group will be used to
calculate Z scores.
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 percent).
- Bone Mineral Density (BMD) determined by DEXA (QDR4500) to confirm osteoporosis at a Z
score greater than 2 SD (standard deviations) below the normal mean for age (Z score <
- 2 SD).
- 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 = 20 kg and more.
Exclusion Criteria:
- History of severe gastritis or reflux.
- Abnormalities of the esophagus that delay emptying, such as strictures or achalasia
- Marked kyphoscoliosis or the inability to sit or stand for at least 30 minutes
- Hypersensitivity to bisphosphonates
- Uncorrected hypocalcemia
- History of gastric or duodenal ulcers
- Renal dysfunction as indicated by serum Cr >1. 5 mg/dl.
- Liver dysfunction as indicated by serum SGPT > 2 times the upper limit for age or
serum total bilirubin > 2. 0 mg/dl.
- Diagnosis of osteogenesis imperfecta, a family history of osteogenesis imperfecta,
blue sclerae or deafness.
- Diagnosis of active rickets or osteomalacia or serum bone alkaline phosphatase 2 times
greater than normal for age.
- Pregnancy
- Anorexia Nervosa
Locations and Contacts
Medical University of South Carolina, Charleston, South Carolina 29425, United States
Additional Information
Medical University of South Carolina, Children's Hospital
Starting date: October 2003
Ending date: September 2008
Last updated: April 15, 2008
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