Adult Idiopathic Generalized Osteoporosis
Conditions
Keywords
osteoporosis, premenopausal, denosumab, idiopathic, Idiopathic Osteoporosis in Premenopausal Women
Brief summary
The purpose of this research study is to evaluate antiresorptive therapy with denosumab (Prolia) for prevention of bone loss after stopping teriparatide (TPTD) in premenopausal women with idiopathic osteoporosis. Premenopausal women who have received TPTD in the FDA Orphan Diseases Program-funded trial, A Phase 2 Study of Teriparatide for the Treatment of Idiopathic Osteoporosis in Premenopausal Women (NCT01440803) may be eligible to participate in the current study, a 36-month open-label pilot study of denosumab (Prolia®, 60mg subcutaneous (SC) every 6 months). The goals of the study are to estimate the effects of denosumab on central and peripheral, as well as trabecular and cortical, bone mass and microstructure and to obtain preliminary data to inform the design of a future randomized study. This study presents the first opportunity to study the effects of denosumab after TPTD in this unique and severely affected group of young women. Funding Source: FDA Office of Orphan Products Development (OOPD).
Detailed description
Osteoporosis in premenopausal women with normal menstrual function and no specific cause is termed idiopathic osteoporosis (IOP). IOP is a rare disease with an estimated prevalence of \<200,000 affected premenopausal women in the United States. Denosumab, a potent inhibitor of osteoclast-mediated bone resorption, leads to continuous gains in both trabecular and cortical bone mineral density (BMD). Moreover, denosumab is not retained in the skeleton, and may thus be preferable for use in young women who may be contemplating future pregnancies. The investigators hypothesize that denosumab, initiated after completion of two years of TPTD, will maintain or improve central and peripheral areal and volumetric BMD, microstructure and stiffness in premenopausal women with IOP.
Interventions
DRUGDenosumab
Denosumab 60mg, administered every 6 months by subcutaneous injection for 36 months
Sponsors
Creighton University
Elizabeth Shane
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
20 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* All women completing NCT01440803 who remain without a disease or medication that causes osteoporosis will be offered enrollment into this study. * (Premenopausal status is no longer be required for entry.)
Exclusion criteria
* Renal insufficiency or liver disease: Creatinine, transaminase (AST)/alanine transaminase (ALT) above upper limit of normal * Vitamin D deficiency: 25-hydroxyvitamin D (25-OHD) \<30 ng/mL * Pregnancy: urine pregnancy test must be negative
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Lumbar Spine Areal BMD by DXA | Baseline and 12 months | Bone mineral density (BMD) will be measured with dual-energy X-ray absorptiometry (DXA). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in Lumbar Spine Areal BMD by DXA at 24 Months | Baseline and 24 months | Bone mineral density (BMD) will be measured with dual-energy X-ray absorptiometry (DXA). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Denosumab Denosumab 60mg, administered every 6 months by subcutaneous injection for 36 months.
Denosumab: Denosumab 60mg, administered every 6 months by subcutaneous injection for 36 months | 33 |
| Total | 33 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Denosumab |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 33 Participants |
| Age, Continuous | 39 years STANDARD_DEVIATION 8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 32 Participants |
| Sex: Female, Male Female | 33 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 33 |
| other Total, other adverse events | 26 / 33 |
| serious Total, serious adverse events | 0 / 33 |
Outcome results
Primary
Percent Change in Lumbar Spine Areal BMD by DXA
Bone mineral density (BMD) will be measured with dual-energy X-ray absorptiometry (DXA).
Time frame: Baseline and 12 months
Population: 33 participants completed baseline but 1 participant withdrew from the study before the 12 month visit.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Denosumab | Percent Change in Lumbar Spine Areal BMD by DXA | 5.2 percentage of change | Standard Deviation 2.6 |
Secondary
Percent Change in Lumbar Spine Areal BMD by DXA at 24 Months
Bone mineral density (BMD) will be measured with dual-energy X-ray absorptiometry (DXA).
Time frame: Baseline and 24 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Denosumab | Percent Change in Lumbar Spine Areal BMD by DXA at 24 Months | 6.9 percentage of change | Standard Deviation 2.6 |