An open-label, intra-patient dose-escalation study to evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of multiple infusions of BPS804 in adults with hypophosphatasia

ovartis Pharma Services AG0 个研究点目标入组 9 人开始时间: 2011年2月7日最近更新: 2013年1月7日

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•Male and female patients 18 to 75 years of age in good health (other than pre-established clinical diagnosis of HPP) as determined by past medical history, physical examination, vital signs, electrocardiogram, and laboratory tests at screening.
•Previously established clinical diagnosis of HPP with confirmed ALPL mutation by genetic test and as manifested by:
•Serum alkaline phosphatase levels below the age-adjusted normal
•Radiologic evidence of osteopenia or osteomalacia or
•History of plasma PLP at least twice the upper limit of normal range or
•History of rickets, or history of premature loss of decidious teeth, or bone deformity consistent with osteomalacia or past rickets, or past non-traumatic fracture, pseudofracture, or non-healing fracture.
•25-(OH) vitamin D3 serum level of =10 ng/mL.
•Normocalcemia with serum calcium =8.5 mg/dL and =10.2 mg/dL (or
•according to local laboratory ranges).
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 7
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•A history of clinically significant ECG abnormalities.
•History of malignancy of any organ system (other than localized basal cell carcinoma of the skin and for skeletal malignancies see below), within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
•History of skeletal malignancies or bone metastases at any time.
•History of external beam radiation to the skeleton.
•Open epiphyses as judged by the Investigator based on previous clinical assessments.
•Patients with suspected neural foraminal stenosis (e.g., at cervical, spinal, or lumbar site) as judged by the Investigator which could be caused by disc herniation and are described as sciatic pain, tingling,
•burning sensation with numbness and/or weakness.
•History of or concomitant diseases such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, Pagets disease, previous neck surgery involving partial or complete thyroidectomy and abnormal thyroid function or thyroid disease or other endocrine disorders or conditions.
•Treatment with any anti-resorptive medication (e.g., oral and/or injectable), bisphosphonates and/or teriparatide (e.g., ForteoTM) within the last 6 months.
•Exposure to blood products or monoclonal antibodies within previous 12 months.
•Any deformation of the spine (e.g., severe scoliosis, ankylosing
•spondylitis) or the hip which would preclude proper acquisition of one of either the lumbar spine or the hip BMD by DXA.

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