Clinical trials: Bone Disease, Metabolic
- Condition Bone Disease, Metabolic
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hypophosphatasia
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT02496689
- Testing
- asfotase alfa
- Where
- 5 sites in United States, France
- Sponsor
- Alexion Pharmaceuticals, Inc.
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
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
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
Hypophosphatasia
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT00894075
- Testing
- ENB-0040
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
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, Fracture
Develop and Validate Ultrasonic Device for Osteoporotic Fracture Risk Assessment
Study to demonstrate that the Bone UltraSonic Scanner (BUSS) can aid in detection of osteoporosis and predict prevalent osteoporotic fractures.
- Conditions
- Osteoporosis, Fracture, Aging
- Trial name
- BUSS03 (NCT01123421)
- Where
- United States
- Volunteers
- 113 people
- Sponsor
- Artann Laboratories
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
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
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.
Osteoporosis
An Observational Study Assessing Effects in Patients With Combination Therapy With Raloxifene/Cholecalciferol
This study examines quality of life and patient satisfaction in postmenopausal women receiving raloxifene to prevent/treat osteoporosis, and vitamin D to reduce risk of fracture using clinical practice data.
- Trial name
- NCT04788290
- Testing
- Observational
- Where
- South Korea
- Volunteers
- 3,907 people
- Sponsor
- Hanmi Pharmaceutical Company Limited
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
End Stage Renal Disease, Hyperparathyroidism, Secondary
Mineral and Bone Disorders Outcomes in Stage 5D of Chronic Kidney Disease
The MBD-5D is a prospective observational study with a case-cohort and a cohort design. Eligible patients are receiving hemodialysis and have secondary hyperparathyroidism.
- Trial name
- MBD-5D (NCT00995163)
- Where
- Japan
- Volunteers
- 8,020 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Osteoporosis
Post Marketing Surveillance For General Drug Use To Assess the Safety And Efficacy Profile Of Viviant In Usual Practice
This survey is conducted for preparing application material for re examination under the Pharmaceutical Affairs Laws and its Enforcement Regulation, and assessing the safety and efficacy profiles of VIVIANT in usual practice…
- Trial name
- NCT01793142
- Testing
- Viviant
- Where
- 60 sites in South Korea
- Volunteers
- 3,430 people
- Sponsor
- Pfizer
Osteoporosis
Study of the Experience of Patients With Osteoporosis Using the Forteo B Pen to Self Administer Once Daily Teriparatide Therapy
This study is being conducted to collect information from subjects with osteoporosis regarding their experience using the Forteo B Pen to self-administer teriparatide in the community setting.
- Trial name
- NCT00577863
- Testing
- teriparatide
- Where
- 18 sites in United States
- Volunteers
- 200 people
- Sponsor
- Eli Lilly and Company
Rhizomelic Chondrodysplasia Punctata
RCDP Natural History Study
A prospective, longitudinal observational trial in patients with RCDP. Study participants will be evaluated at baseline and approximately every 6 months by the study team.
- Trial name
- NCT04031287
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- MED-LIFE DISCOVERIES LP