Alendronate in Juvenile Osteoporosis
Study Details
Study Description
Brief Summary
The investigators earlier have shown that treatment of patients with juvenile osteoporosis with alendronate (Fosamax) for 12 months increased the bone density without side effects. In an open label study (10 patients) and double blind, crossover study (11 patients alendronate and 11 patients placebo), the investigators have further observed that alendronate increased the bone density significantly where as placebo (calcium and vitamin D) increased only minimally. These trials were completed. Thus, a post study is designed to evaluate the current status of the bone density and fractures after the patients discontinued the alendronate treatment. No treatment is involved.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Detailed Description
With the availability of Dual Energy X-ray Absorptiometry (DXA), juvenile osteoporosis has been recognized and diagnosed in recent years. The disease results from either diminished bone formation or increased bone removal (resorption) resulting in low bone density and fractures. No specific drug therapy has been recommended for juvenile osteoporosis. In an open label study, we earlier have shown that alendronate treatment (10 patients) for 12 months increased bone density without side effects. Subsequently, in a double blind, crossover study, we have further confirmed that alendronate treatment (11 patients) increased bone density significantly whereas, placebo (11 patients with calcium and vitamin D), increased the bone density only minimally. There were no side effects. These patients were treated with alendronate only for 12 months and the clinical trials have been completed. We therefore, have designed a post study to evaluate the current status of the bone density and fracture history in the above participants after the discontinuation (1-6 years) of alendronate treatment. Available participants, who have completed the earlier study, will be scheduled for a one time clinic visit. Measurements include DXA bone density measurement of spine and hip, urinalysis and blood work. No treatment is involved.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 1 Alendronate for 12 months, post study Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
Drug: Alendronate (Fosamax)
Pill, 35mg or 70mg depending upon the body weight for 12 months. This was given for participants during the open label or double blind study. Current study is a post study evaluation of the current status of bone density after the participants completed the study. In this post study, no treatment is involved.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Bone Mineral Density (BMD) of the Lumbar Spine (Participants With Percentage Increase). [Post study (1-6 yrs), one clinical visit]
Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Bone density of spine was measured by DXA scan.
Secondary Outcome Measures
- Bone Mineral Density (BMD) of the Hip (Participants With Percentage Increase). [Post study (1-6 years), one clincial visit]
Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Bone density of hip was measured by DXA scan.
- Number of Participants With Fracture [Post study (1-6 years), one clinical visit]
Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Their bone densities of spine and hip were measured by DXA scan. During this visit, their fracture history was obtained.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Participated in our earlier clinical study;
-
Completed the earlier open label or double blind study;
-
Availability to participate in the post study;
-
Male and female children who have earlier participated in our clinical trial; AND
-
Parental consent (and patient assent after age 12 years) to participate in the study. Participant's consent for those who have completed 18 years of age and above at the time of clinic visit.
Exclusion Criteria:
-
Not participated in our earlier clinical study;
-
Not completed our earlier trials; OR
-
Pregnancy.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Medical University of South Carolina | Charleston | South Carolina | United States | 29425 |
Sponsors and Collaborators
- Medical University of South Carolina
- FDA Office of Orphan Products Development
- Merck Sharp & Dohme LLC
Investigators
- Principal Investigator: Deborah A Bowlby, M.D., Medical University of South Carolina
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 5R01FD001847-05 REVISED
- FD-R-001847-03
- 5R01FD001847-05
Study Results
Participant Flow
| Recruitment Details | Participants who have completed our earlier phase-1 and II study on alendronate in juvenile osteoporosis will be invited in this post study to evaluate the bone density/ fractures after discontinuation of therapy. Interested participants will be scheduled for a one time clinic visit. No treatment is involved. |
|---|---|
| Pre-assignment Detail | Participants should have completed our earlier phase I or II study on alendronate in juvenile osteoporosis. Participants will be recruited by invitation only. |
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
| Period Title: Last Study, 25 Pts Invited,11 Responded | |
| STARTED | 11 |
| COMPLETED | 11 |
| NOT COMPLETED | 0 |
| Period Title: Last Study, 25 Pts Invited,11 Responded | |
| STARTED | 11 |
| COMPLETED | 11 |
| NOT COMPLETED | 0 |
Baseline Characteristics
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
| Overall Participants | 11 |
| Age (Count of Participants) | |
| <=18 years |
11
100%
|
| Between 18 and 65 years |
0
0%
|
| >=65 years |
0
0%
|
| Age (years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [years] |
12.9
(2.7)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
6
54.5%
|
| Male |
5
45.5%
|
| Region of Enrollment (participants) [Number] | |
| United States |
11
100%
|
Outcome Measures
| Title | Bone Mineral Density (BMD) of the Lumbar Spine (Participants With Percentage Increase). |
|---|---|
| Description | Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Bone density of spine was measured by DXA scan. |
| Time Frame | Post study (1-6 yrs), one clinical visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants who earlier completed our phase I or phase II study on alendronate in juvenile osteoporosis, were invited to participate in the current post study evaluation of bone density and fractures. |
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
| Measure Participants | 11 |
| Number [participants] |
11
100%
|
| Title | Bone Mineral Density (BMD) of the Hip (Participants With Percentage Increase). |
|---|---|
| Description | Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Bone density of hip was measured by DXA scan. |
| Time Frame | Post study (1-6 years), one clincial visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| 11 participants responded to participate in the post study. Their bone density of Hip was measured by DXA scan. Increase in percentage density of Hip was obtained from that of previous values. One participant showed a slight decrease in bone density. |
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
| Measure Participants | 11 |
| Number [participants] |
10
90.9%
|
| Title | Number of Participants With Fracture |
|---|---|
| Description | Participants who earlier completed in our open labeled or double blind study of alendronate treatment for juvenile osteoporosis, were invited for one clinical visit. Their bone densities of spine and hip were measured by DXA scan. During this visit, their fracture history was obtained. |
| Time Frame | Post study (1-6 years), one clinical visit |
Outcome Measure Data
| Analysis Population Description |
|---|
| 11 participants responded to participate in the post study. During their one clinic visit, their fracture history during the period before coming to the post study was obtained. |
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. |
| Measure Participants | 11 |
| Number [participants] |
0
0%
|
Adverse Events
| Time Frame | 1-6 years post study | |
|---|---|---|
| Adverse Event Reporting Description | Participants who completed our earlier phase I/II study on alendronate in juvenile osteoporosis were invited to participate in the current post study evaluation of their current bone density/fracure history. Durine their one time clinic visit, they were asked regarding any adverse events they had during the period before coming to the post study. | |
| Arm/Group Title | 1 Alendronate for 12 Months, Post Study | |
| Arm/Group Description | Participants earlier were treated with alendronate for 12 months either in an open label study (without control) or double blind study with placebo control. These studies were completed. In this post study evaluation, available participants will be scheduled for one clinic visit to assess their current status of the bone density and no treatment is involved. | |
All Cause Mortality |
||
| 1 Alendronate for 12 Months, Post Study | ||
| Affected / at Risk (%) | # Events | |
| Total | / (NaN) | |
Serious Adverse Events |
||
| 1 Alendronate for 12 Months, Post Study | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/11 (0%) | |
Other (Not Including Serious) Adverse Events |
||
| 1 Alendronate for 12 Months, Post Study | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/11 (0%) | |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Agreement with Merck: to submit a copy of any proposed abstract, manuscript and/or press release to Merck for review and comment at least 30 days prior to submission for publication or presentation.
Results Point of Contact
| Name/Title | Deborah A Bowlby, Asst.Professor, Pediatric Endocrinology |
|---|---|
| Organization | Medical University of South Carolina |
| Phone | 843-792-6807 |
| bowlbyd@musc.edu |
- 5R01FD001847-05 REVISED
- FD-R-001847-03
- 5R01FD001847-05