Clinical trials: Deformity; Bone
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Myelodysplastic Syndrome, Thalassemia
This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
- Trial name
- NCT01250951
- Testing
- Deferasirox
- Where
- 2 sites in Russia
- Volunteers
- 111 people
- Sponsor
- Novartis Pharmaceuticals
Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma
Tipifarnib for the Treatment of Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With HRAS Gene Alterations, a Pediatric MATCH Treatment Trial
This phase II pediatric MATCH trial studies how well tipifarnib works in treating patients with solid tumors that have recurred or spread to other places in the body (advanced), lymphoma, or histiocytic disorders, that have a…
- Conditions
- Malignant Solid Neoplasm, Recurrent Adrenal Gland Pheochromocytoma, Recurrent Ectomesenchymoma, Recurrent Ependymoma
- Trial name
- NCT04284774
- Testing
- Tipifarnib
- Where
- 172 sites in United States, Puerto Rico
- Volunteers
- 5 people
- Sponsor
- National Cancer Institute (NCI)
Breast Cancer, Chronic Myeloproliferative Disorders
Motexafin Gadolinium and Doxorubicin in Treating Patients With Advanced Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Motexafin gadolinium may increase the effectiveness of doxorubicin by making tumor cells more sensitive to…
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Colorectal Cancer, Head and Neck Cancer
- Trial name
- NCT00036790
- Testing
- doxorubicin hydrochloride, motexafin gadolinium
- Where
- 2 sites in United States
- Sponsor
- University of Wisconsin, Madison
Quality of Life, Spinal Deformity
Quality of Life in Parents of Adolescents With Spinal Deformities: Development of a New Questionnaire.
This study aims to develop a new instrument capable of providing an efficient measure of the quality of life of parents of conservatively treated patients with spinal deformity.
- Conditions
- Quality of Life, Spinal Deformity, Scoliosis; Adolescence
- Trial name
- NCT04899297
- Testing
- Questionnaire
- Where
- Italy
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Istituto Scientifico Italiano Colonna Vertebrale
Aplastic Anemias, Hemoglobinopathies
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood…
- Conditions
- Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
- Trial name
- HOPE Resource (NCT07227155)
- Where
- 14 sites in United States
- Volunteers
- 375 people, healthy volunteers welcome
- Sponsor
- Medical College of Wisconsin
Early-Onset Scoliosis Deformity of Spine
Long-Term Follow-Up Of Surgical Management Of Early Onset Scoliosis Using Growing Rods
The aim of this work is to evaluate the long-term outcome of the graduates (those who completed the gradual lengthening procedures), to better understand the full scope and natural course of Growing Rods treatment for EOS and to…
- Trial name
- NCT04945343
- Testing
- Growing Rods
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Assiut University
Leukemia, Myelodysplastic Syndromes
3-AP and Cytarabine in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy such as cytarabine use different ways to stop cancer cells from dividing so they stop growing or die.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00064090
- Testing
- cytarabine, triapine
- Where
- United States
- Sponsor
- Vion Pharmaceuticals
Cystic Fibrosis, Contraception
Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
The long-term goal is to study the safety and effectiveness of hormonal contraception for women with cystic fibrosis (CF) and contribute to national guidelines that the Cystic Fibrosis Foundation and the Centers for Disease…
- Trial name
- CASE4CF (NCT04568980)
- Where
- 10 sites in United States
- Volunteers
- 562 people, healthy volunteers welcome
- Sponsor
- University of Washington
Turner Syndrome, Bicuspid Aortic Valve
Aortopathy in Persons With Bicuspid Aortic Valve, Turner and Marfan Syndrome
The study aim is: 1. To examine aortic tissue by light microscopy 2. To examine aortic tissue by electron microscopy 3. To study changes in the epigenome and transcriptome of the X chromosome specific to aortic tissue. 4.
- Conditions
- Turner Syndrome, Bicuspid Aortic Valve, Marfan Syndrome
- Trial name
- NCT01760668
- Where
- Denmark
- Volunteers
- 5 people
- Sponsor
- University of Aarhus
Cancer
Vaccine Therapy in Treating Patients Who Have Undergone a Donor Stem Cell Transplant and Have Cytomegalovirus Infection That Has Not Responded to Therapy
RATIONALE: Vaccines may help the body build an effective immune response to kill cytomegalovirus infections. PURPOSE: This phase I trial is studying the side effects and best dose of vaccine therapy in treating patients who have…
- Trial name
- NCT00509691
- Testing
- cytomegalovirus pp65-specific cytotoxic T lymphocytes, polymerase chain reaction, diagnostic laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Louisville
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
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
Marfan Syndrome
Biological Collection for Marfan and Related Syndromes
The present study will establish a collection of biological samples from Marfan patients or with associated diseases to be used for research purposes only, with due respect for confidentiality.
- Trial name
- MARFANS (NCT04970459)
- Testing
- collection of samples of blood and urine
- Where
- France
- Volunteers
- 300 people
- Sponsor
- University Hospital, Toulouse
Pheochromocytoma, Paraganglioma
Genetic Analysis of Pheochromocytomas, Paragangliomas and Associated Conditions
Pheochromocytomas and paragangliomas are neural crest-derived tumors of the nervous system that are often inherited and genetically heterogeneous.
- Conditions
- Pheochromocytoma, Paraganglioma, Inherited Cancer Syndrome, Associated Conditions
- Trial name
- NCT03160274
- Testing
- Genetic screening
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
Cholangiocarcinoma
Efficacy and Safety of Pemigatinib in Subjects With Advanced/Metastatic or Surgically Unresectable Cholangiocarcinoma Who Failed Previous Therapy - (FIGHT-202)
The purpose of this study is evaluate the efficacy of pemigatinib in subjects with advanced/metastatic or surgically unresectable cholangiocarcinoma with FGFR2 translocation who have failed at least 1 previous treatment.
- Trial name
- NCT02924376
- Testing
- Pemigatinib
- Where
- 120 sites in United States, Belgium and 10 more
- Volunteers
- 147 people
- Sponsor
- Incyte Corporation
Short Stature, Growth Disorders
Bone Age Assessment in Children Using Ultrasound Compared to Wrist X-ray
Bone age assessment in children is based on the interpretation of hand x-ray scans according to Greulich and Pyle (GP) standard atlas and frequently used for evaluating growth and puberty in children and adolescents.
- Trial name
- NCT03123003
- Testing
- SonicBone Ultrasound, Wrist X-ray
- Where
- Israel
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Meir Medical Center
Congenital Hypothyroidism, Neurodevelopmental Disorders
Understanding the Subnormal Rescue ofNeurological Development in Congenital Hypothyroidism (2022E5SZEM)
Congenital hypothyroidism (CH) is a condition present at birth in which the thyroid gland does not produce enough thyroid hormones. These hormones are essential for normal brain development.
- Trial name
- 2022E5SZEM (NCT07893522)
- Where
- 3 sites in Italy
- Volunteers
- 178 people
- Sponsor
- Istituto Giannina Gaslini
Osteogenesis Imperfecta
Effects of Bisphosphonates on OI-Related Hearing Loss
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in…
- Trial name
- NCT04152551
- Testing
- Risedronate Oral Tablet
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Hospital for Special Surgery, New York
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Tacrolimus and Mycophenolate Mofetil in Preventing Acute Graft-Versus-Host Disease in Patients With Advanced Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplantation
RATIONALE: Tacrolimus and mycophenolate mofetil may be an effective treatment for graft-versus-host disease caused by donor stem cell transplantation.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00096096
- Testing
- mycophenolate mofetil, tacrolimus
- Where
- United States
- Sponsor
- Fred Hutchinson Cancer Center
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