Clinical trials: Metabolic Bone Disease
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Anorexia Nervosa
Adrenal and Gonadal Hormone Replacement in Anorexia Nervosa
This study seeks to gain new information on why young women with anorexia nervosa are predisposed to early bone loss and osteoporosis.
- Trial name
- NCT00310791
- Testing
- Hormone replacement therapy (estrogen/progestin), Dehydroepiandrosterone (DHEA)
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Boston Children's Hospital
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Chronic Iron Overload
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
- Trial name
- NCT02720536
- Testing
- Deferasirox
- Where
- 14 sites in Austria, Greece and 1 more
- Volunteers
- 53 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia, Lymphoblastic, Acute, Osteoporosis
A Severity-Adapted Clinical Trial of Diminished Bone Mineral Density in Acute Lymphoblastic Leukemia Survivors
Research studies have shown that children who are long-term survivors of childhood leukemia may be at greater risk for early bone loss called osteoporosis.
- Trial name
- NCT00186901
- Testing
- Calcium carbonate (Tums), vitamin D
- Where
- 3 sites in United States
- Volunteers
- 429 people
- Sponsor
- St. Jude Children's Research Hospital
Turner Syndrome
Somatropin Treatment to Final Height in Turner Syndrome
A randomized, controlled trial in girls with Turner syndrome at least 7 years old and younger than 13 at study entry, to determine the efficacy and safety of Humatrope (somatropin) treatment in promoting linear growth to final…
- Trial name
- GDCT (NCT00191113)
- Testing
- Somatropin, Ethinyl estradiol, Medroxyprogesterone acetate
- Where
- 14 sites in Canada
- Volunteers
- 154 people
- Sponsor
- Eli Lilly and Company
Osteopenia, Osteoporosis
Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
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).
- Trial name
- NCT02074631
- Testing
- Pamidronate, Calcium and vitamin D
- Where
- 2 sites in United States
- Volunteers
- 63 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Leukemia, Lymphoma
Cyproheptadine Hydrochloride and Nutritional Supplementation in Treating Young Patients With Weight Loss With Cancer
RATIONALE: Cyproheptadine hydrochloride may help improve appetite and lessen weight loss caused by cancer or cancer treatment. It is not yet known whether cyproheptadine hydrochloride is more effective with or without nutritional…
- Conditions
- Leukemia, Lymphoma, Malnutrition, Myelodysplastic Syndromes
- Trial name
- NCT00949117
- Testing
- Ensure, PediaSure, cyproheptadine hydrochloride
- Volunteers
- 9 people
- Sponsor
- University of South Florida
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Stem Cell Transplantation for Hurler
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for Hurler syndrome…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
- Trial name
- NCT00176917
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Osteoporosis
Intervenous (IV) Zoledronic Acid After Forteo in Postmenopausal Women
The purpose of this study is to evaluate the adequacy of zoledronic acid in maintaining bone mass after two years of treatment with Forteo, in postmenopausal women.
- Trial name
- NCT00361595
- Testing
- zoledronic acid
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- The Cleveland Clinic
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Osteoporosis
A Randomized, Double-blind, Placebo-controlled, Dose Response Study of AMG 162 (Denosumab) in Japanese Postmenopausal Osteoporotic Subjects
The primary objective of this study is to assess the effect of AMG 162 (denosumab) treatment on the lumbar spine BMD at month 12 and safety profile across the dose range tested (14, 60 and 100 mg SC once every 6 months) in…
- Trial name
- NCT00306189
- Testing
- 100 mg AMG 162, 60 mg AMG 162, 14 mg AMG 162
- Volunteers
- 212 people
- Sponsor
- Amgen
Hypophosphatasia (HPP)
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
- Trial name
- NCT00744042
- Testing
- asfotase alfa
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Vitamin D Deficiency
Evaluation of Vitamin D Requirements During Pregnancy
The purpose of this study is to determine the effectiveness of vitamin D supplementation during pregnancy starting at the beginning of the second trimester.
- Trial name
- NCT00292591
- Testing
- cholecalciferol (vitamin D3), cholecalciferol
- Where
- United States
- Volunteers
- 501 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Post-Menopausal Osteoporosis
BONCURE Study: A Study of Monthly Bonviva (Ibandronate) in Women With Post-Menopausal Osteoporosis on Bisphosphonate Therapy.
This single arm study will assess participant preference for monthly Bonviva, versus daily or weekly alendronate or risedronate, in the treatment of postmenopausal osteoporosis.
- Trial name
- NCT00545779
- Testing
- Ibandronate
- Where
- 32 sites in Albania, Bosnia and Herzegovina and 4 more
- Volunteers
- 677 people
- Sponsor
- Hoffmann-La Roche
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota