Adult patients with Thalassemia major and osteoporosis
Conditions
Interventions
Trade Name: Prolia
Product Name: Prolia
Pharmaceutical Form: Injection
Pharmaceutical form of the placebo: Injection
Route of administration of the placebo: Subcutaneous use
Sponsors
Ersi Voskaridou-Dimoula
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Adults (>30 years of age) described as skeletally mature subjects • Thalassemia Major • Low BMD (T-score =65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: •BMD T-score 2 fold the upper limit of normal laboratory range; or elevated direct bilirubin > 1.5 x the upper limit of normal laboratory range. •Heart failure (NYHA above 2). •Patients with life expectancy of less than one year. •Subject refuses to use a reliable contraceptive method (oral contraceptives, progesterone implants, intrauterine device, condoms) throughout the study by women of childbearing potential. Women of childbearing potential agree to use 2 highly effective forms of contraception and to continue this practice for 7 months after last injection of study medication. •Pregnancy, planning a pregnancy or currently lactating •Severe concurrent illness which in the investigator’s opinion may confound patient evaluation, e.g. malignancy (except basal cell carcinoma, cervical or breast ductal carcinoma in situ) within the last 5 years. Known alcohol or drug abuse or any other condition associated with poor compliance. •Patients that have received oral bisphosphonates within 6 months of study enrollment or intravenous bisphosphonates, fluoride and strontium ranelate within 1 year of study enrollment. •PTH, PTH derivatives, teriparatide, odanacatib, anabolic steroids, testosterone, glucocorticosteroids (> 5 mg/day of prednisone equivalent for > 10 days), systemic hormone-replacement therapy, selective estrogen receptor modulators (SERMs), raloxifene, tibolone, calcitonin or calcitriol use within the last 6 weeks. •Evidence of hyper- or hypothyroidism; patients with an abnormal TSH level on thyroid treatment (patients on stable thyroid treatment with a normal TSH allowed); current hyper-or hypoparathyroidism; current hyper or hypocalcemia (hypocalcemia based on albumin adjusted serum calcium < 8.5 mg/dL); vitamin D deficiency (25-hydroxy Vitamin D level < 12 ng/mL; if repeat 12-20 ng/mL after repletion, subject will be allowed); rheumatoid arthritis; Paget’s disease; bone disease that would interfere with interpretation of findings. •Known sensitivity to mammalian cell-derived drug products. •History of any Solid Organ or Bone Marrow Transplant •History of osteonecrosis of the jaw, and/or recent tooth extraction or other dental surgery; or planned invasive dental work during the study. •Intolerance to calcium supplements. •Malabsorption syndrome; severe malabsorptin including Celiac disease, Short Bowel Syndrome, Crohn's disease, Previous Gastric Bypass.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the effect of Denosumab (plus vitamin D & calcium) on lumbar spine BMD in patients with Thalassemia Major and Osteoporosis as compared with control (placebo plus vitamin D & calcium) at 12 months.;Secondary Objective: 1) To evaluate the effect of Denosumab (plus vitamin D & calcium) on femoral neck and wrist bone BMD in patients with Thalassemia Major and Osteoporosis as compared with control (placebo plus vitamin D & calcium) at 12 months. 2) To evaluate the effect of Denosumab on markers of bone remodeling of patients with ThalassemiaMajor and Osteoporosis. 3) To evaluate the safety profile of Denosumab in patients with Thalassemia ajor and Osteoporosis. ;Primary end point(s): The primary efficacy endpoint is the percent change from baseline in the lumbar spine BMD.;Timepoint(s) of evaluation of this end point: at month 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Percent change from baseline in BMD of the total hip and femoral neck . 2. Percent change from baseline in BMD at the distal third radius. 3 Percent change from baseline in sCTX.;Timepoint(s) of evaluation of this end point: 1. At month 12. 2. At month 12. 3. At month 3 post injection. | — |
Countries
Greece
Contacts
Public ContactErsi Voskaridou-Dimoula
Ersi Voskaridou-Dimoula
Outcome results
None listed