Clinical trials: Deformity; Bone
Adult Spinal Deformity, Scoliosis
Prospective, Multicenter, Case-Control Analysis of the VersaTie Posterior Fixation System to Prevent Proximal Junctional Failure in Long Posterior Spinal Fusion Constructs for Adult Patients
Prospective, Multicenter, Case-Control Analysis of the VersaTie Posterior Fixation System to Prevent Proximal Junctional Failure in Long Posterior Spinal Fusion Constructs for Adult Patients
- Conditions
- Adult Spinal Deformity, Scoliosis, Kyphosis
- Trial name
- NCT04888104
- Testing
- Index or revision spine surgery for complex adult spinal deformity
- Where
- 9 sites in United States
- Volunteers
- 225 people
- Sponsor
- International Spine Study Group Foundation
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.
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Acute Myeloid Leukemia (AML)
Safety & Efficacy Study of Oral Panobinostat (LBH589) With Chemotherapy in Patients < 65 Years Old With Acute Myeloid Leukemia (AML)
This study will be conducted to assess the maximum tolerated dose (MTD) of panobinostat given 3 times a week (administered on weeks 2 and 3 of a 4 week cycle) in combination with induction chemotherapy (idarubicin and cytarabine)…
- Trial name
- NCT01242774
- Testing
- Panobinostat
- Where
- 10 sites in United States, Germany and 1 more
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals
Biliary Tract Cancer
Futibatinib (TAS-120) in Patients With Advanced Biliary Tract Cancer
To compare overall survival (OS) of patients in Futibatinib and Zimberelimab in Combination with Gemcitabine plus Cisplatin versus Durvalumab or Pembrolizumab in Combination with Gemcitabine plus Cisplatin for patients with…
- Trial name
- FOENIX-BTC (NCT07710885)
- Testing
- Futibatinib, Zimberelimab, Durvalumab
- Where
- 21 sites in Australia, Japan and 2 more
- Volunteers
- 784 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies
The objective of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of S241656 and to determine the recommended dose for expansion (RDE) of S241656 in participants with relapsed/refractory (R/R) acute…
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT07719348
- Testing
- S241656, Posaconazole
- Volunteers
- 64 people
- Sponsor
- Servier
Achondroplasia
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.
- Trial name
- NCT06842355
- Testing
- TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg, TYRA-300 0.375 mg/kg
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 92 people
- Sponsor
- Tyra Biosciences, Inc
Congenital Heart Disease
Phase I Safety and Feasibility Study of Intracoronary Delivery of Autologous Bone Marrow Derived Mononuclear Cells
The objective of this study is to determine the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) and intracoronary delivery for individuals with declining performance of their single…
- Trial name
- NCT02549625
- Testing
- Autologous Bone Marrow-derived Mononuclear Cells
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Timothy J Nelson, MD, PhD
Chronic Myeloproliferative Disorders, Leukemia
Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor bone marrow transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00134004
- Testing
- cyclophosphamide, fludarabine phosphate, mycophenolate mofetil
- Where
- 3 sites in United States
- Volunteers
- 210 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Telomere Biology Disorders, Dyskeratosis Congenita
Nucleoside Therapy in Patients With Telomere Biology Disorders
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology disorders.
- Conditions
- Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome
- Trial name
- NCT06817590
- Testing
- deoxycytidine, deoxythymidine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Suneet Agarwal
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
FORTIFI-HN01: A Study of Ficerafusp Alfa (BCA101) or Placebo in Combination With Pembrolizumab in First-Line PD-L1-pos, R or M HNSCC
Ficerafusp alfa is directed against two targets, Epidermal Growth Factor Receptor (EGFR) and Transforming Growth Factor beta (TGF-β).
- Trial name
- FORTIFI-HN01 (NCT06788990)
- Testing
- Ficerafusp alfa, Pembrolizumab (KEYTRUDA®)
- Where
- 231 sites in United States, Argentina and 26 more
- Volunteers
- 650 people
- Sponsor
- Bicara Therapeutics
RETT Syndrome With Proven MECP2 Mutation
Osteoporosis in RETT Syndrome
Based on our clinical observations, many girls with RETT syndrome, a severe neuro-developmental encephalopathy, suffer from osteoporosis which can appear at a very early age (before age 10) and can lead to fractures, pain and a…
- Trial name
- OSRETT (NCT02110797)
- Testing
- biological markers and evaluation of the mineral density at the lumber spine using DEXA
- Where
- France
- Volunteers
- 98 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cockayne Syndrome
Observational Study to Assess Natural History in Cockayne Syndrome Patients
This is an Observational Study of children under the age of 11 diagnosed with Cockayne Syndrome to assess the natural progression of Cockayne Syndrome disease, with special attention to hearing and physical changes in length or…
- Trial name
- NCT00985413
- Where
- 4 sites in United States, France and 1 more
- Volunteers
- 40 people
- Sponsor
- DNage B.V.
Bone Marrow Transplant, Sickle Cell Disease
Screening for Subjects to Participate in Studies of Blood Disorders
This study will determine eligibility for participation in research studies on blood disorders conducted by the National Heart, Lung and Blood Institute and the National Institute of Diabetes, Digestive, and Kidney Diseases.
- Conditions
- Bone Marrow Transplant, Sickle Cell Disease, G-CSF
- Trial name
- NCT00695123
- Where
- United States
- Volunteers
- 816 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Pectus Excavatum, Cardiac Anomaly
Capturing Physiologic Autonomic Data from Clinically Indicated Magnetic Resonance Imaging Scans in Children
The Fontan Procedure is a palliative surgical procedure used in pediatric patients with one functional ventricle. The procedure, a series of stepwise operations that alter cardiorespiratory physiology, separate the systemic and…
- Trial name
- CMRI (NCT06110689)
- Testing
- VU-AMS device
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pectus Excavatum
Postoperative Recovery Quality According to Preoperative Fasting Time in Pediatric Patients Undergoing Nuss Operation
Preoperative fasting is intended to lower the amount of gastric contents in order to decrease the incidence of aspiration associated with endotracheal intubation.
- Trial name
- NCT04068324
- Testing
- New Care No Nil Per Os
- Where
- South Korea
- Volunteers
- 90 people
- Sponsor
- Jung Min Koo
Maxillary Retrusion
Radiological Evaluation of Anterior Maxilla For Immediate Implant Placement
According to the literature age and gender may be associated with differences in skeletal size, anatomy and architecture in humans.
- Trial name
- NCT07133503
- Testing
- radiological evaluation
- Where
- 2 sites in Turkey (Türkiye)
- Volunteers
- 278 people, healthy volunteers welcome
- Sponsor
- TC Erciyes University
Anemia, Aplastic, Fanconi Anemia
Cord Blood Stem Cell Transplantation Study (COBLT)
To evaluate if HLA-mismatched, unrelated-donor umbilical cord blood stem and progenitor cell units (UCBU) offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation with…
- Conditions
- Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia
- Trial name
- NCT00000603
- Testing
- stem cell transplantation
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Basal Cell Carcinoma in Basal Cell Nevus Syndrome
Study of ASN-002 to Treat Basal Cell Carcinomas (BCCs) in Individuals With Basal Cell Nevus Syndrome (BCNS)
The primary objective is to confirm the safety of treating multiple BCCs once weekly x 3 weeks in individuals with Basal Cell Nevus Syndrome (BCNS).
- Trial name
- NCT03208296
- Testing
- ASN-002
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Ascend Biopharmaceuticals Ltd
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