Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis

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Primary Purpose

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Alendronate

About this trial

This is an interventional treatment trial for Osteoporosis focused on measuring Fracture, Bone Mineral Density, DXA

Eligibility Criteria

5 Years - 15 Years (Child)All SexesDoes not accept healthy volunteers

Eligibility Criteria: 5-15 yrs of age Weighing 20 kg and more History of multiple fractures Tanner stage II or less Osteoporosis by DXA. 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 DXA sacn 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

Sites / Locations

Medical University of South Carolina

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1 Alendronate, Calcium, Vitamin D

2 Placebo, Calcium and Vitamin D

Arm Description

Crossover study. Year-1, 10 participants will take study medication, calcium and vitamin D supplements and other 10 participants will take placebo, calcium and vitamin D supplements. Year-2, they will crossover to the second arm of the study. Those who took study medication and supplements in year-1, will take placebo and supplements in the year-2, and those 10 participants who took placebo and supplements in the year-1, will take study medications and supplements in the year-2.

Year-1, 10 participants will take Alendronate (study medication)and calcium and vitamin D supplement). Another 10 participants will take placebo, calcium and vitamin D. In year-2 they will crossover. Those who took alendronate in the first year, will take Placebo, calcium and vitamin D for 12 months and those who took Placebo in the first year, will take Alendronate, calcium and vitamin D in the second year (12 months).

Outcomes

Primary Outcome Measures

Number of Participants With Improvement in Bone Mineral Density (BMD) of Spine After Therapy

Participants were screened for BMD of lumbar spine using DXA scan at visit 12 months after alendronate or placebo treatment.

Number of Participants With Improvement in Bone Mineral Density (BMD) of Spine After Therapy

BMD of lumbar spine was measured using DXA scan at visit 24 months (Year-2)after alendronate or placebo treatment.

Secondary Outcome Measures

Number of Participants With Improvement in Bone Mineral Density (BMD) of Hip After Therapy

Number of Participants With Improvement in BMD of Hip

Analysis was done per protocol and intention to treat.

Participants With Atraumatic Fractures

Number of Participants with Atraumatic fractures before therapy.

Participants With Atraumatic Fractures

Number of participants with fractures at the completion of therapy.

Full Information

NCT ID

NCT00259857

First Posted

November 29, 2005

Last Updated

December 29, 2010

Sponsor

Medical University of South Carolina

Collaborators

FDA Office of Orphan Products Development, Merck Sharp & Dohme LLC

1. Study Identification

Unique Protocol Identification Number

NCT00259857

Brief Title

Safety and Efficacy of Alendronate (Fosamax) in Children With Osteoporosis

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 Type

Interventional

2. Study Status

Record Verification Date

December 2010

Overall Recruitment Status

Completed

Study Start Date

October 2003 (undefined)

Primary Completion Date

March 2008 (Actual)

Study Completion Date

August 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Medical University of South Carolina

Collaborators

FDA Office of Orphan Products Development, Merck Sharp & Dohme LLC

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

We have previously evaluated the safety and efficacy of Alendronate in 10 patients with juvenile osteoporosis during a 12-month clinical trial. We have documented that Alendronate 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 Alendronate in 20 children with juvenile osteoporosis using a double-blind, randomized, placebo-controlled, cross-over protocol.

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 Alendronate 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 Alendronate and 10 patients placebo. In the year-2, patients will be crossed over to the second arm of the study. Those who received Alendronate in the year-1, will receive placebo in the second year and vice verse. The patients will have 5 visits, the initial screening visit followed by 4 post therapy visits in a six-month interval. Measurements include DXA bone density scan of spine and hip, urinalysis and blood work.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Osteoporosis

Keywords

Fracture, Bone Mineral Density, DXA

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Crossover Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

22 (Actual)

8. Arms, Groups, and Interventions

Arm Title

1 Alendronate, Calcium, Vitamin D

Arm Type

Experimental

Arm Description

Crossover study. Year-1, 10 participants will take study medication, calcium and vitamin D supplements and other 10 participants will take placebo, calcium and vitamin D supplements. Year-2, they will crossover to the second arm of the study. Those who took study medication and supplements in year-1, will take placebo and supplements in the year-2, and those 10 participants who took placebo and supplements in the year-1, will take study medications and supplements in the year-2.

Arm Title

2 Placebo, Calcium and Vitamin D

Arm Type

Placebo Comparator

Arm Description

Year-1, 10 participants will take Alendronate (study medication)and calcium and vitamin D supplement). Another 10 participants will take placebo, calcium and vitamin D. In year-2 they will crossover. Those who took alendronate in the first year, will take Placebo, calcium and vitamin D for 12 months and those who took Placebo in the first year, will take Alendronate, calcium and vitamin D in the second year (12 months).

Intervention Type

Drug

Intervention Name(s)

Alendronate

Other Intervention Name(s)

Fosamax, TUMS or Viactive, Drisdol or Calciferol

Intervention Description

Group-1/Year-1:Alendronate, pill, 35mg or 70mg, weekly; calcium, pill, 500mg or 1000mg daily; vitamin D, liquid, 800IU, daily, depending upon the body weight, for 12 months. Group-1/Year-2:Placebo, pill, 35mg or 70mg, weekly; calcium, pill, 500mg or 1000mg daily; vitamin D, liquid, 800IU, daily, depending upon the body weight for 12 months. Group-2/Year-1:Placebo, pill, 35mg or 70mg, weekly; calcium, pill, 500mg or 1000mg daily; vitamin D, liquid, 800IU, daily, depending upon the body weight for 12 months. Group-2/Year-2: Alendronate, pill, 35mg or 70mg, weekly; calcium, pill, 500mg or 1000mg daily; vitamin D, liquid, 800IU, daily, depending upon the body weight for 12 months.

Primary Outcome Measure Information:

Title

Number of Participants With Improvement in Bone Mineral Density (BMD) of Spine After Therapy

Description

Participants were screened for BMD of lumbar spine using DXA scan at visit 12 months after alendronate or placebo treatment.

Time Frame

12 months therapy

Title

Number of Participants With Improvement in Bone Mineral Density (BMD) of Spine After Therapy

Description

BMD of lumbar spine was measured using DXA scan at visit 24 months (Year-2)after alendronate or placebo treatment.

Time Frame

24 months therapy

Secondary Outcome Measure Information:

Title

Number of Participants With Improvement in Bone Mineral Density (BMD) of Hip After Therapy

Time Frame

12 months of therapy

Title

Number of Participants With Improvement in BMD of Hip

Description

Analysis was done per protocol and intention to treat.

Time Frame

24 months of therapy

Title

Participants With Atraumatic Fractures

Description

Number of Participants with Atraumatic fractures before therapy.

Time Frame

0 months

Title

Participants With Atraumatic Fractures

Description

Number of participants with fractures at the completion of therapy.

Time Frame

24 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

15 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

Eligibility Criteria: 5-15 yrs of age Weighing 20 kg and more History of multiple fractures Tanner stage II or less Osteoporosis by DXA. 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 DXA sacn 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Deborah A Bowlby, M.D.

Organizational Affiliation

Medical University of South Carolina

Official's Role

Principal Investigator

Facility Information:

Facility Name

Medical University of South Carolina

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29425

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

12795371

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.

Results Reference

result

Osteoporosis

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial