This is a Phase 2, open-label, randomized, controlled clinical study of pediatric subjects treated with pamidronate with calcium and vitamin D versus calcium and vitamin D alone following hematopoietic cell transplantation (HCT). The purpose of this study is to test the hypothesis that subjects receiving pamidronate with calcium and vitamin D will have higher lumbar spine bone mineral content (LBMC) measured by dual-energy X-ray tomography (DXA) at 1 year post-HCT than subjects receiving calcium and vitamin D alone (Control Group).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Enrollment
63
Subjects randomized to pamidronate treatment will receive infusions, 1 mg/kg (to a max dose of 60mg) over 4 hours, every 3 months at approximately 100 days, 180 days, and 270 days after HCT.
All subjects will receive a standard recommended dose of 600 IU/day of vitamin D. Subjects who do not meet the RDA will receive additional calcium supplementation.
University of Minnesota Amplatz Children's Hospital
Minneapolis, Minnesota, United States
Seattle Children's Hospital
Seattle, Washington, United States
Lumbar Spine Bone Mineral Content
Time frame: 1 year after HCT
Total Body Bone Mineral Content (TBMC; Excluding Head; Adjusted for Height, Age, Sex, Tanner Stage, and Race)
Time frame: 1 year after HCT
Total Bone Mineral Density (BMD), Cortical BMD, Trabecular BMD, and Estimated Bone Strength Measured by pQCT
Measured in g/cm2.
Time frame: 1 year after HCT
Cytokine Levels (Interleukin IL-6, IL-7, and TNF-α)
Measured in pg/ml.
Time frame: 7 days, 14 days, 21 days, 90 days after HCT
Receptor Activator of the Nuclear Factor-κB Ligand [RANKL], Osteoprotegerin [OPG]
Measured in pg/ml.
Time frame: 7 days, 14 days, 21 days, and 90 days after HCT
Marker of Bone Resorption (Carboxy-terminal Collagen Crosslinks [CTX]
CTX measured in ng/ml.
Time frame: 7, 14, 21, 90, 180, 360 days after HCT
Markers of Bone Formation (Procollagen Type 1 N-terminal Propeptide [P1NP])
P1NP measured in ng/ml.
Time frame: 7, 14, 21, 90, 180, 360 days after HCT
Ratio of Receptor Activator of the Nuclear Factor-κB Ligand [RANKL] and Osteoprotegerin [OPG]
Time frame: 7 days, 14 days, 21 days, and 90 days after HCT
Marker of Bone Resorption Deoxypyridinoline [DPD])
DPD measured in mmol/L.
Time frame: 7, 14, 21, 90, 180, 360 days after HCT
Marker of Bone Formation Osteocalcin [OCN])
OCN measured in pg/ml.
Time frame: 7, 14, 21, 90, 180, 360 days after HCT
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