Fractures, Juvenile Osteoporosis, Low Bone Density
Conditions
Keywords
Juvenile Osteoporosis, Bone Mineral Density, Fosamax, Dual Energy X-Ray Absorptiometry, Fracture
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.
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.
Interventions
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.
Sponsors
FDA Office of Orphan Products Development
Merck Sharp & Dohme LLC
Medical University of South Carolina
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
8 Years to 21 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
United States
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 details
Participants should have completed our earlier phase I or II study on alendronate in juvenile osteoporosis. Participants will be recruited by invitation only.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 11 |
| Total | 11 |
Baseline characteristics
| Characteristic | 1 Alendronate for 12 Months, Post Study |
|---|---|
| Age, Categorical <=18 years | 11 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 12.9 years STANDARD_DEVIATION 2.7 |
| Region of Enrollment United States | 11 participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 11 |
| serious Total, serious adverse events | 0 / 11 |
Outcome results
Primary
Bone Mineral Density (BMD) of the Lumbar Spine (Participants With Percentage Increase).
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
Population: 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 | Measure | Value (NUMBER) |
|---|---|---|
| 1 Alendronate for 12 Months, Post Study | Bone Mineral Density (BMD) of the Lumbar Spine (Participants With Percentage Increase). | 11 participants |
Secondary
Bone Mineral Density (BMD) of the Hip (Participants With Percentage Increase).
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
Population: 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 | Measure | Value (NUMBER) |
|---|---|---|
| 1 Alendronate for 12 Months, Post Study | Bone Mineral Density (BMD) of the Hip (Participants With Percentage Increase). | 10 participants |
Secondary
Number of Participants With Fracture
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
Population: 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 | Measure | Value (NUMBER) |
|---|---|---|
| 1 Alendronate for 12 Months, Post Study | Number of Participants With Fracture | 0 participants |