Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis - Full Text View

Sponsors and Collaborators:	FDA Office of Orphan Products Development Medical University of South Carolina
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00259857

Purpose

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.

Condition	Intervention	Phase
Osteoporosis	Drug: Alendronate	Phase II

MedlinePlus related topics: Fractures Minerals Osteoporosis

Drug Information available for: Alendronate Alendronate sodium

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Investigator), Placebo Control, Crossover Assignment, Safety/Efficacy Study
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)

Further study details as provided by FDA Office of Orphan Products Development:

Primary Outcome Measures:

Bone mineral density (BMD) of the lumbar spine at 12 and 24 months at 12 months [ Time Frame: 20-30 min ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

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. [ Time Frame: 20-30 min (BMD); 4 hours (biochemical assays) ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	20
Study Start Date:	October 2003
Estimated Study Completion Date:	September 2008
Estimated Primary Completion Date:	April 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
2: Placebo Comparator Crossover study. Year-1, 10 participants will take study medication and other 10 participants will take placebo. Year-2, they will crossover to the second arm of the study. Those who took study medication in year-1, will take placebo in the year-2, and those 10 participants who took placebo in the year-1, will take study medications in the year-2.	Drug: Alendronate dosage form:tablet dosage: 35mg (<40kg body weight) or 70mg (>40kg body weight) at the time of start. frequency: weekly duration:12 months
1: Placebo Comparator year-1, 10 participants will take Alendronate (study medication). and another 10 participants will take placebo. In year-2 they will crossover.	Drug: Alendronate dosage form:tablet dosage: 35mg (<40kg body weight) or 70mg (>40kg body weight) at the time of start. frequency: weekly duration:12 months

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

Ages Eligible for Study:	5 Years to 15 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00259857

Locations

United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425

Sponsors and Collaborators

FDA Office of Orphan Products Development

Medical University of South Carolina

Investigators

Principal Investigator:

Lyndon L Key, M.D.

Medical University of South Carolina

More Information

Medical University of South Carolina, Children's Hospital This link exits the ClinicalTrials.gov site

Publications of Results:

Key LL Jr, Ries W, Madyastha P, Reed F. Juvenile osteoporosis: recognizing the risk. J Pediatr Endocrinol Metab. 2003 May;16 Suppl 3:683-6.

Responsible Party:	Medical University of South Carolina ( L Lyndon Key, MD, Prof and Chairman )
Study ID Numbers:	5 R01 FD001847-05, FD-R-001847-03, FD-R01-2-0, FD-188-03
Study First Received:	November 29, 2005
Last Updated:	April 15, 2008
ClinicalTrials.gov Identifier:	NCT00259857
Health Authority:	United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:

Fracture
Bone Mineral Density
DEXA

Study placed in the following topic categories:

Musculoskeletal Diseases
Alendronate
Fractures, Bone
Osteoporosis

Bone Diseases, Metabolic
Juvenile osteoporosis
Bone Diseases

Additional relevant MeSH terms:

Physiological Effects of Drugs
Bone Density Conservation Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009