Clinical trials: Bone Deformity
Funnel Chest
Cardiorespiratory Assessment and Quality of Life After Surgical Correction of Congenital Wall Malformations
The main objective of this study is to characterize the cardiorespiratory functional gain in patients with pectus excavatum following corrective surgery.
- Trial name
- HeartSoar (NCT03770390)
- Testing
- Surgical correction of pectus excavatum
- Where
- France
- Volunteers
- 43 people
- Sponsor
- University Hospital, Montpellier
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
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
Based on success in other diseases, the Fred Hutchinson Cancer Research Center (FHCRC) has developed a transplant procedure for Fanconi anemia (FA), which does not completely destroy the patient's remaining bone marrow.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT00093743
- Testing
- fludarabine phosphate, cyclosporine, total-body irradiation
- Where
- 5 sites in United States
- Volunteers
- 2 people
- Sponsor
- Fred Hutchinson Cancer Center
Thoracic Aorta Dilatation, Marfan Syndrome
Thoracic Aortic Dilatation Syndromes
Aortic dilatation syndromes are comprised by a group of different syndromes, of which Marfan syndrome is the best described. Many of the aorta dilatation associated syndromes are heritable connective tissue disorders but some…
- Trial name
- NCT02111668
- Where
- Denmark
- Volunteers
- 120 people
- Sponsor
- University of Aarhus
Marfan Syndrome
The Oxford Marfan Trial
The primary objective of the trial is to estimate the effects of allocation to irbesartan, or doxycycline, or a combination of both irbesartan and doxycycline, compared with placebo, on measures of elastic function of the aorta…
- Trial name
- NCT01949233
- Testing
- Irbesartan 150-300mg capsules daily for 6 months, Doxycycline 100-200mg capsules daily for 6 months
- Where
- 2 sites in United Kingdom
- Volunteers
- 56 people
- Sponsor
- University of Oxford
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
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 an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Sarcoidosis, Tuberculosis
The Serum Angiotensin Converting Enzyme and Lysozyme Levels in Patients Non-infectious and Infectious Uveitis
This study evaluates the significance of differences in serum angiotensin converting enzyme and lysozyme levels of patients with ocular involvement of other autoimmune inflammatory and infectious diseases.
- Trial name
- uveitis (NCT02627209)
- Testing
- spectrophotometric assay, radial immunodiffusion
- Where
- Turkey (Türkiye)
- Volunteers
- 280 people
- Sponsor
- Ozlem Sahin
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma
Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma
- Trial name
- NCT03213665
- Testing
- Tazemetostat
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Exostoses, Multiple Hereditary
Height, Ulnar Length and Forearm Function in Multiple Hereditary Exostoses
the purpose of the present registry is to describe the epidemiology of forearm deformities in patients with Hereditary Multiple Exostoses and to identify, independent predictors of severity of the disease and potential…
- Trial name
- NCT05914298
- Testing
- blood and buccal swab genetic test, PUL, Range of motion
- Where
- Italy
- Volunteers
- 408 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Leukemia, Myelodysplastic Syndromes
Bone Marrow Transplant Plus Cyclophosphamide and Total-Body Irradiation in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00002809
- Testing
- anti-thymocyte globulin, filgrastim, sargramostim
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Temple University
Gaucher's Disease
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages.
- Trial name
- NCT00001234
- Testing
- human glucocerebrosidase cDNA
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Osteogenesis Imperfecta (OI)
Open-label Extension of Study 20130173 of Denosumab in Children and Young Adults With Osteogenesis Imperfecta
To evaluate long-term safety of denosumab in children/young adults with pediatric osteogenesis imperfecta (OI) who completed the prior study 20130173 (NCT02352753).
- Trial name
- NCT03638128
- Testing
- Denosumab, Alternative osteoporosis medications
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 75 people
- Sponsor
- Amgen
Multiple Osteochondroma
Descriptive Analysis of Surgeries in Patients With Multiple Osteochondromas
Multiple Osteochondromas (MO), also known as Multiple Cartilaginous Exostoses, is an autosomal dominant skeletal disorder with an incidence of approximately 1 in 50,000 in Western populations.
- Trial name
- CHIROS (NCT07556874)
- Testing
- Surgery for osteochondromas
- Where
- Italy
- Volunteers
- 300 people
- Sponsor
- Istituto Ortopedico Rizzoli
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
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
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
Leukemia, Lymphoma
AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders
RATIONALE: Giving chemotherapy with or without total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00547196
- Testing
- filgrastim, Busulfan, Cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- City of Hope Medical Center
Muscular Dystrophy, Duchenne Muscular Dystrophy
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
This Study is single arm, single center trial to check the safety and efficacy of BMMNC (100 million per dose) for the patient with Duchenne Muscular Dystrophy,
- Trial name
- BMMNC (NCT01834040)
- Testing
- Intralesional/ Intravenous of Autologous Stem cells.
- Where
- India
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Chaitanya Hospital, Pune
Fanconi Anemia
Gene Therapy for Fanconi Anemia
This clinical trial will access the toxicity and efficacy of infusion of gene modified cells for patients with Fanconi anemia (FA).
- Trial name
- NCT01331018
- Testing
- Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Fred Hutchinson Cancer Center
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.