Clinical trials: Deformity; Bone
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period…
- Trial name
- ApproaCH (NCT05598320)
- Testing
- Navepegritide (TransCon CNP)
- Where
- 10 sites in United States, Australia and 5 more
- Volunteers
- 84 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
Sickle Cell Disease, Hemoglobinopathies
Non-Myeloablative Conditioning and Bone Marrow Transplantation
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia.
- Trial name
- NCT01850108
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide (CTX)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Vanderbilt-Ingram Cancer Center
Growth Hormone Deficiency, Septo-Optic Dysplasia
Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
Hypotheses: 1. The prevalence of endocrinopathies, and growth hormone (GH) deficiency in particular, among young children diagnosed with optic nerve hypoplasia (ONH) is higher than is commonly thought. 2.
- Conditions
- Growth Hormone Deficiency, Septo-Optic Dysplasia, Hypopituitarism
- Trial name
- NCT00140413
- Testing
- Nutropin AQ
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
Fanconi Anemia, Severe Aplastic Anemia
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
The purpose of this study is to discover whether children and adults with Fanconi anemia (FA) can be safely and effectively transplanted with Human Leukocyte Antigen (HLA) mismatched (up to one haplotype), HLA-matched sibling, or…
- Trial name
- Mafia (NCT00590460)
- Testing
- CAMPATH-1H, Anti-CD45, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
Breast Cancer, Chronic Myeloproliferative Disorders
Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, granisetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients treated with chemotherapy.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00293384
- Testing
- Aprepitant, Cyclophosphamide, Dexamethasone
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Chronic Myeloproliferative Disorders, Leukemia
Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy, such as busulfan and fludarabine, before a donor stem cell transplant helps stop the growth of cancer and abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00448201
- Testing
- anti-thymocyte globulin, sargramostim, therapeutic allogeneic lymphocytes
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Thalassemia
Zinc & Bone Health in Thalassemia: The Think Zinc Study
The purpose of this study is to test whether zinc can improve bone health in young patients with thalassemia.
- Trial name
- ThinkZn (NCT00459732)
- Testing
- Zinc
- Where
- 3 sites in United States
- Volunteers
- 45 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Adult Rhabdomyosarcoma, Adult Synovial Sarcoma
Cixutumumab in Treating Patients With Relapsed or Refractory Solid Tumors
This phase II trial is studying the side effects and how well cixutumumab works in treating patients with relapsed or refractory solid tumors. Monoclonal antibodies, such as cixutumumab, can block tumor growth in different ways.
- Conditions
- Adult Rhabdomyosarcoma, Adult Synovial Sarcoma, Childhood Hepatoblastoma, Childhood Synovial Sarcoma
- Trial name
- NCT00831844
- Testing
- cixutumumab, laboratory biomarker analysis
- Where
- 105 sites in United States, Australia and 1 more
- Volunteers
- 116 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Osteoarthritis, Avascular Necrosis
Trilogy AB Acetabular Hip System Post Approval Study
This is a post-approval study with prospective enrollment in the United States and a retrospective enrollment outside the United States to obtain functional, quality of life and radiographic data, which will facilitate assessment…
- Conditions
- Osteoarthritis, Avascular Necrosis, Congenital Hip Dysplasia, Traumatic Arthritis
- Trial name
- NCT00715026
- Testing
- Trilogy AB Acetabular Hip Implant System
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Zimmer Biomet
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
X-linked Hypophosphatemia
Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
- Trial name
- NCT02537431
- Testing
- burosumab
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 14 people
- Sponsor
- Kyowa Kirin, Inc.
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Short Stature
Individualizing Dose of Growth Hormone to Maintain Normal Growth Velocity After Fulfilled Catch up Growth in Children
The objective was to study whether normal growth velocity can be maintained with adapted GH dosage in GH treated prepubertal children who have responded to GH treatment with fulfilled catch up growth (=difference to target height…
- Trial name
- MAINTENANCE (NCT02879747)
- Testing
- Genotropin
- Volunteers
- 99 people
- Sponsor
- Göteborg University
Hypovolemia, Craniosynostoses
Bedside Resources to Gauge Intravascular Volume Status
The goal if this study is to employ the CardioQ-Esophageal Aortic Doppler probe to define fluid responders from non-responders among infants undergoing cranial vault reconstruction for craniosynostosis.
- Trial name
- NCT03915587
- Testing
- CardioQ-EDM and CipherOx-CRI
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- University of Colorado, Denver
Severe Combined Immunodeficiency
SCID Bu/Flu/ATG Study With T Cell Depletion
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with a diagnosis of Severe Combined Immune Deficiency (SCID) who do not have an HLA-matched sibling donor.
- Trial name
- NCT02127892
- Testing
- unrelated BM with T cell depletion, unrelated cord blood, haplo BM with T cell depletion
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Neena Kapoor, M.D.
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Myofacial Pain, Pain, Neck
Trigger Point Injections in Anterior Cervical Surgery
To achieve appropriate exposure for an anterior neck surgery (for example an Anterior Cervical Discectomy and Fusion or ACDF), patients are positioned supine with their neck extended.
- Conditions
- Myofacial Pain, Pain, Neck, Pain, Back, Cervical Fusion
- Trial name
- NCT04640896
- Testing
- Trigger point injection with bupivacaine, Trigger point injection with normal saline, Lidocaine skin wheal
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- George Washington University
Osteogenesis Imperfecta
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss.
- Trial name
- NCT03064074
- Testing
- Fresolimumab
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baylor College of Medicine