A clinical study to evaluate the safety and preliminary efficacy of BPS804 in adults with hypophosphatasia

• Conditions: Hypophosphatasia (HPP) is a rare genetic metabolic disorder which results in impaired skeletal mineralization, and which is caused by the absence of or by deficient enzymatic activity of the tissue-nonspecific alkaline phosphatase (TNSALP); MedDRA version: 14.1Level: PTClassification code 10049933Term: HypophosphatasiaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2010-024013-31-DE
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-02-07
• Last Update Posted: 7 January 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 9

Inclusion Criteria

•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
range and
•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

Exclusion Criteria

•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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified