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Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation

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ClinicalTrials.gov Identifier: NCT02074631
Recruitment Status : Recruiting
First Posted : February 28, 2014
Last Update Posted : September 25, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Osteopenia Osteoporosis Drug: Pamidronate Drug: Calcium and vitamin D Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
Actual Study Start Date : February 2015
Estimated Primary Completion Date : February 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Calcium Vitamin D
Drug Information available for: Vitamin D

Arm Intervention/treatment
Active Comparator: Control group
Subjects will receive a standard recommended dose of calcium and vitamin D.
Drug: Calcium and vitamin D
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.
Other Names:
  • Cholecalciferol
  • Ergocalciferol

Experimental: Pamidronate Group
Subjects randomized to pamidronate treatment will receive infusions approximately 100, 180, and 270 days after HCT along with calcium and vitamin D.
Drug: Pamidronate
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.
Other Names:
  • Aredia
  • Bonapam

Drug: Calcium and vitamin D
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.
Other Names:
  • Cholecalciferol
  • Ergocalciferol




Primary Outcome Measures :
  1. Lumbar spine bone mineral content [ Time Frame: 1 year after HCT ]

Secondary Outcome Measures :
  1. Total body bone mineral content (TBMC; excluding head; adjusted for height, age, sex, Tanner stage, and race) [ Time Frame: 1 year after HCT ]
  2. Total bone mineral density (BMD), cortical BMD, trabecular BMD, and estimated bone strength measured by pQCT [ Time Frame: 1 year after HCT ]
  3. Cytokine levels (interleukin IL-6, IL-7, and TNF-α) [ Time Frame: 7 days, 14 days, 21 days, 100 days after HCT ]
  4. Receptor activator of the nuclear factor-κB ligand [RANKL], osteoprotegerin [OPG], RANKL/OPG ratio [ Time Frame: 7 days, 14 days, 21 days, and 100 days after HCT ]
  5. Markers of bone resorption (carboxy-terminal collagen crosslinks [CTX] and deoxypyridinoline [DPD]) [ Time Frame: 7, 14, 21, 100, 180, 360 days after HCT ]
  6. Markers of bone formation (procollagen type 1 N-terminal propeptide [P1NP] and osteocalcin [OCN]) [ Time Frame: 7, 14, 21, 100, 180, 360 days after HCT ]


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Ages Eligible for Study:   1 Year to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Allogeneic hematopoietic cell transplant for hematologic malignancy (i.e. leukemia, lymphoma including ALL, AML, CML, NHL, HL) in complete remission; myelodysplastic syndrome (active dysplasia and/or blasts are permitted, but must not have active leukemia) or idiopathic severe aplastic anemia (SAA)
  • Non-malignant diseases including idiopathic severe aplastic anemia (SAA) and other bone marrow failure disorders, hemoglobinopathies, adrenoleukodystrophy, immune deficiencies/dysregulation disorders who will be receiving myeloablative or reduced toxicity preparative regimens that meet the following criteria:

    • Regimens include those that are TBI based if the TBI dose is > 500cGy single dose or > 800cGy fractionated, or doses <500 cGy if combined with busulfan or treosulfan. These also include chemotherapy only based regimens that contain myeloablative doses of busulfan (>8mg/kg) or treosulfan without TBI.
    • Patients with severe aplastic anemia are eligible regardless of conditioning regimen
  • Myeloablative preparative regimen (for SAA any conditioning therapy allowed)
  • Male or female ≥1 but ≤ 20 years of age at time of study enrollment
  • Patient or parent(s)/legal guardian(s) is able and willing to provide informed consent. Assent will be obtained per local institutional policy. Subjects who turn 18 during the course of the study will be consented at that time of their next visit by a member of the research staff.

Exclusion Criteria:

  • History of a primary bone malignancy involving the lumbar spine
  • Prior and/or planned concomitant medical therapy during the study period (through Day 360 post-HCT) with other bisphosphonates, Denosumab, or Teriparatide
  • Pregnancy or breastfeeding - menstruating females must have a negative pregnancy test prior to study enrollment and agree to repeat pregnancy testing and contraception use per protocol as pamidronate is Pregnancy Category D - positive evidence of human fetal risk based on adverse reaction data
  • Renal insufficiency, defined as creatinine level greater than the upper limit of normal for age
  • Hereditary metabolic bone disease or skeletal dysplasia (e.g., osteopetrosis or OI) or primary hyperparathyroidism
  • Other indications for HCT, including Fanconi anemia, other form of inherited bone marrow failure diseases, metabolic disorder, hemoglobinopathy, or immune deficiency
  • Clinically significant fractures as defined by ISCD (a long bone fracture of the lower extremities, vertebral compression fracture, or two or more long bone fractures of the upper extremities) (88,89) indicated by a cast or a spine x-ray within the last 2 weeks
  • Known or suspected allergy to pamidronate or related products
  • Planned administration of an investigational study drug or agent that either can interact with pamidronate or have an independent effect on bone mineral density within the 4 weeks prior to randomization (Day 90) or planned use during study participation (Day 90 through Day 360)
  • Impending invasive dental procedure that would be expected to occur during study participation (through Day 360)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02074631


Contacts
Contact: Kim Nelson, RN 612-273-2925 knelso62@fairview.org
Contact: Kyriakie Sarafoglou, MD (612) 624-0123 saraf010@umn.edu

Locations
United States, Minnesota
University of Minnesota Amplatz Children's Hospital Recruiting
Minneapolis, Minnesota, United States, 55454
Contact: Kim Nelson, RN    612-273-2925    knelso62@fairview.org   
Principal Investigator: Kyriakie Sarafoglou, M.D.         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Scott Baker, M.D.    206-667-5594    ksbaker@fhcrc.org   
Principal Investigator: Scott Baker, M.D.         
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Kyriakie Sarafoglou, MD University of Minnesota - Clinical and Translational Science Institute

Publications of Results:
Other Publications:

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT02074631     History of Changes
Other Study ID Numbers: 2013LS023
First Posted: February 28, 2014    Key Record Dates
Last Update Posted: September 25, 2018
Last Verified: September 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Masonic Cancer Center, University of Minnesota:
bone mineral content
bone mineral density
bone formation
bone resorption
pamidronate
children
DXA
bone marrow transplant

Additional relevant MeSH terms:
Osteoporosis
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metabolic Diseases
Vitamins
Vitamin D
Ergocalciferols
Cholecalciferol
Calcium, Dietary
Micronutrients
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents