Clinical trial NCT00010439: Phase II Study of Alendronate Sodium in Children With High-Turnover Idiopathic Juvenile Osteoporosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Osteoporosis
Keywords: arthritis & connective tissue diseases
idiopathic juvenile osteoporosis
rare disease
Lead sponsor: Medical University of South Carolina
Sponsor class: OTHER
Collaborators: name: Merck Sharp & Dohme LLC
First posted: 2001-02-02
Enrollment: 10
Sex: ALL
Minimum age (years): 5
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: PROTOCOL OUTLINE:

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

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Increased Bone Mineral Density
timeFrame: at 12 months
Secondary outcomes:
measure: Participants (1) With Fractures Before and After Therapy,(2)Analysed for Average Changes From High to Near Normal Mineral Apposition Rate (MAR) After Therapy,(3)Analysed for Average Insignificant Changes in Biochemical Markers After Therapy.
timeFrame: Before and 12 months after treatment with alendronate
Other outcomes:
Locations:
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
affiliation: Medical University of South Carolina
name: Deborah A Bowlby, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.