Approval Probability
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Adjusted LOA
ML Risk
BPS804 · 3 trials · 4 indications
The assessment of safety will be based on primarily on the frequency of adverse events, laboratory abnormalities, and serious adverse events suspected by the investigators to be related to study treatments. The AE collection period extends from the time of first study drug administration drug until study completion. The intensity of each AE will be characterized and classified into 1 of the 3 generic categories (mild, moderate, or severe). The number and percentage of patients with adverse events will be tabulated by treatment group, body system and preferred term. The periods for adverse event tabulation will be from dose administration up to next dose administration if a further dose is given, and from dose administration to EOS for the last dose administration of a patient.
Biomarker data from serum bone formation biomarkers: procollagen type I N-terminal propeptide, procollagen type I C-terminal propeptide, osteocalcin, and bone-specific alkaline phosphatase, and from serum bone resporption mbiomarkers: C-telopeptides of type I collagen cross-links, and N-telopeptides of type I collagen cross-links will be reported as concentration results, measured using a specific assay with a working range defined by the Lower limit of quantification and Upper limit of quantification. It will be considered a sign for efficacy, if any of the above show significant (2-sided, alpha=0.05) increase versus baseline in the BPS804 group on day 43.
Bone mineral density will be assessed by dual-energy X-ray absorptiometry of the lumbar spine. Analysis will include four vertebral levels from L1 to L4. Individual vertebral levels may be excluded due to artifact. Bone mineral density Z-scores will be used as these are a comparison of a patient's BMD to that of a patient of the same age, sex, and ethnicity. The comparison of change from baseline with the matching change in the reference group will be done by 2-sample t-tests (1-sided). It will be considered a sign for efficacy, if any of the above show significant (2-sided, alpha=0.05) increase versus baseline in the BPS804 group on day 141.
| Arm | Type | Description |
|---|---|---|
| BPS804 dosing frequency 1 | EXPERIMENTAL | Subjects dosed 20mg/Kg BPS804 monthly |
| placebo dosing frequency 1 | PLACEBO_COMPARATOR | Subjects dosed with matching placebo to 20mg/Kg BPS804 monthly |
| BPS804 dosing frequency 2 | EXPERIMENTAL | Subjects dosed with 20mg/Kg BPS804 quarterly |
| placebo dosing frequency 2 | PLACEBO_COMPARATOR | Subjects dosed with matching placebo to 20mg/Kg BPS804 every 3 months |
| BPS804 dosing frequency 3 | EXPERIMENTAL | Subjects dosed with 20mg/Kg BPS804 weekly |
| Placebo dosing frequency 3 | PLACEBO_COMPARATOR | Subjects dosed with matching placebo to 20mg/Kg BPS804 weekly |
| BPS804 dose escalation | EXPERIMENTAL | BPS804 IV Setrusumab given in escalating doses from 5mg/Kg to 20mg/Kg |
| Treatment group | EXPERIMENTAL | - |
| Untreated reference group | NO_INTERVENTION | - |
| Name | Type | Description |
|---|---|---|
| BPS804 20mg/Kg | DRUG | - |
| Placebo to 20mg/Kg BPS804 | DRUG | - |
| BPS804 | DRUG | - |
Inclusion Criteria: * Postmenopausal women (natural or surgically induced menopause) * Low bone mineral density (BMD), as defined by a T score or equivalent BMD absolute value (g/cm2) for lumbar spine of between -2.0 and -3.5, inclusive * Body mass index (BMI) must be within the range of 18 to 35kg...
BPS804 is an investigational small molecule being developed by Ultragenyx Pharmaceutical Inc. for rare bone disorders. It has been studied in Phase 2 clinical trials for hypophosphatasia, osteogenesis imperfecta, and osteopenia. BPS804 is not approved for any indication and remains in clinical development.
BPS804 has been evaluated in a Phase 2 clinical trial for adults with hypophosphatasia (HPP), a rare genetic bone disorder. The trial, NCT01406977, was a dose escalation study assessing safety and tolerability of multiple infusions. BPS804 is investigational and has not been approved for hypophosphatasia or any other condition.
BPS804 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare diseases. Ultragenyx trades on the Nasdaq under the ticker symbol RARE. The company is responsible for the clinical development of BPS804.
BPS804 is in Phase 2 clinical development. It has completed Phase 2 trials for hypophosphatasia, osteogenesis imperfecta, and osteopenia. There are currently no active trials listed for BPS804, and it has not received FDA approval for any indication.
BPS804 has been studied in three completed Phase 2 trials: NCT01417091 in osteogenesis imperfecta, NCT01406977 in hypophosphatasia, and NCT01406548 in postmenopausal women with low bone mineral density. All trials are completed, and no active trials are ongoing.