Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density

Ultragenyx

Status and phase: Completed; Phase 2

Conditions: Osteoporosis; Osteopenia

Treatments: Drug: BPS804 20mg/Kg; Drug: Placebo to 20mg/Kg BPS804

Study type: Interventional

Funder types: Industry

Identifiers

NCT01406548

CBPS804A2203

Details and patient eligibility

About

This study is designed to provide information on the safety, tolerability, pharmacokinetics (PK) and bone biomarker response following multiple BPS804 administration in multiple dosing regimens. This information will permit a comparison of the possible risks and benefits of different dosing regimens of the study drug to enable optimal doses and dose intervals to be tested in subsequent studies.

Full description

This study was conducted and previously posted by Novartis. The record was transferred to Ultragenyx in February 2021.

Enrollment: 44 patients

Sex: Female

Ages: 45 to 85 years old

Volunteers: No Healthy Volunteers

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/m2. Subjects must weigh between 45 and 120kg inclusive to participate.
• 25-(OH) vitamin D serum level of ≥ 15ng/ml
• Serum calcium within normal limits

Exclusion criteria

• Subjects with suspected neural foraminal stenosis (e.g., cervical, spinal, lumbar), or history of Bell's palsy, cranial nerve disorders, temporomandibular joint and muscle disorders.

• Subjects who have an increased baseline risk of osteosarcoma: Paget's disease of the bone or unexplained and clinically significant elevations of alkaline phosphatase and/or subjects who have received radiation therapy involving the skeleton.

• Subjects with any known bone diseases other than postmenopausal osteoporosis.

• Subjects with a history of an osteoporotic fracture (e.g., vertebral fracture, fragility fracture of the wrist, radius, humerus, hip, or pelvis).

• Subjects who are regularly using or have regularly used agents affecting bone metabolism:

  • Calcitonin, estrogen, SERMs (raloxifene, Tamoxifen, etc.), Tibolone progestin, or androgens within the last three (3) months prior to screening.
  • Any oral bisphosphonate, lithium chloride, fluoride or systemic glucocorticosteroids (p.o. or i.v.) where the total dose exceeds 750 mg of prednisone or equivalent within the last year prior to screening.
  • Any previous use of denusomab (ProliaTM), parathyroid hormone (ForteoTM), and/or PTH analogs, strontium ranelate, or parenteral formulations of bisphosphonates.

• Current disease(s) known to influence calcium metabolism including hyperparathyroidism, hypoparathyroidism, hypocalcemia or hypercalcemia.

• Any disease, abnormality or deformation of the spine (e.g., scoliosis, ankylosing spondylitis, osteophytes) or hip (e.g., joint prosthesis) which would preclude the proper acquisition of a lumbar spine DXA (L1-L4) or femur DXA, respectively.

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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44 participants in 6 patient groups, including a placebo group

BPS804 dosing frequency 1

Experimental group

Description:

Subjects dosed 20mg/Kg BPS804 monthly

Treatment:

Drug: BPS804 20mg/Kg

placebo dosing frequency 1

Placebo Comparator group

Description:

Subjects dosed with matching placebo to 20mg/Kg BPS804 monthly

Treatment:

Drug: Placebo to 20mg/Kg BPS804

BPS804 dosing frequency 2

Experimental group

Description:

Subjects dosed with 20mg/Kg BPS804 quarterly

Treatment:

Drug: BPS804 20mg/Kg

placebo dosing frequency 2

Placebo Comparator group

Description:

Subjects dosed with matching placebo to 20mg/Kg BPS804 every 3 months

Treatment:

Drug: Placebo to 20mg/Kg BPS804

BPS804 dosing frequency 3

Experimental group

Description:

Subjects dosed with 20mg/Kg BPS804 weekly

Treatment:

Drug: BPS804 20mg/Kg

Placebo dosing frequency 3

Placebo Comparator group

Description:

Subjects dosed with matching placebo to 20mg/Kg BPS804 weekly

Treatment:

Drug: Placebo to 20mg/Kg BPS804