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Conditions: Twin to Twin Transfusion Syndrome
Interventions: S-LPC:Selective Laser Photocoagulation; Device
Lead sponsor: University of Tennessee; Other
Eligibility: 16 Weeks to 27 Weeks · Female only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2020
U.S. locations: 2
States / cities: Memphis, Tennessee

Conditions: Cystinuria, Nephrolithiasis, Calcium Oxalate
Interventions: Cystone, Sugar Pill (Placebo); Drug
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 100 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Hemophilia B
Interventions: HEMGENIX, Factor IX (FIX); Genetic · Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2043
U.S. locations: 1
States / cities: Rochester, New York

Conditions: Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
Interventions: Daunorubicin, Cytarabine, Clofarabine, Decitabine, Observation, Allogeneic hematopoietic stem cell transplantation; Drug · Other · Procedure
Lead sponsor: ECOG-ACRIN Cancer Research Group; Network
Eligibility: 60 Years and older
Enrollment: 727 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2022
U.S. locations: 266
States / cities: Birmingham, Alabama • Scottsdale, Arizona • Tucson, Arizona + 181 more

Conditions: Enterocolitis, Necrotizing, Bacteremia, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage, Periventricular Leukomalacia, Chronic Lung Disease, Ileal Perforation
Interventions: Antibiotic, Gastric fluid, Breast milk, Stool samples, Antibiotics; Drug · Other
Lead sponsor: University of Florida; Other
Eligibility: 23 Weeks to 33 Weeks
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Cystic Fibrosis, Ventilatory Defect
Interventions: 19F MRI; Combination Product
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: 18 Years to 99 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Thyroid Eye Disease, TED
Interventions: MHB018A injection, MHB018A injection Placebo; Drug
Lead sponsor: Minghui Pharmaceutical (Hangzhou) Ltd; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 135 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: Coral Gables, Florida

Conditions: Autosomal Dominant Polycystic Kidney Disease
Interventions: ADPKD Diet; Other
Lead sponsor: University of Kansas; Other
Eligibility: 18 Years to 65 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Kansas City, Kansas

Conditions: Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Interventions: Burosumab; Drug
Lead sponsor: Laura Tosi; Other
Eligibility: Not listed
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Cytomegalovirus Infections
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 9 Years to 14 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2005
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Sickle Cell Disease
Interventions: Conditioning Regimen with Bone Marrow Transplant; Drug
Lead sponsor: Emory University; Other
Eligibility: 16 Years to 40 Years
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 8
States / cities: Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 4 more

Conditions: Cellulite
Interventions: Sofwave; Device
Lead sponsor: Sofwave Medical LTD; Industry
Eligibility: 18 Years to 60 Years · Female only
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023
U.S. locations: 5
States / cities: Sacramento, California • Hackensack, New Jersey • New York, New York + 2 more

Conditions: Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
Interventions: filgrastim, clofarabine, melphalan, mycophenolate mofetil, tacrolimus, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 0 Years to 54 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2021
U.S. locations: 1
States / cities: New York, New York

Conditions: Wilms' Tumor, Sarcoma, Adenaocortical Carcinoma, Refractory Cancer, Coldrhood Cancer
Interventions: Tariquidar; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 2 Years to 18 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2016
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Congenital Ichthyosis
Interventions: Isotretinoin, Vehicle; Drug · Other
Lead sponsor: LEO Pharma; Industry
Eligibility: 9 Years and older
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 8
States / cities: Phoenix, Arizona • Palo Alto, California • New Haven, Connecticut + 5 more

Conditions: Sickle Cell Disease
Interventions: mHealth app + booklets; Other
Lead sponsor: Ohio State University; Other
Eligibility: 18 Years and older
Enrollment: 287 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 4
States / cities: Chicago, Illinois • St Louis, Missouri • Columbus, Ohio + 1 more

Conditions: Sickle Cell Disease
Interventions: Crovalimab, Placebo; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 12 Years to 55 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Atlanta, Georgia • New York, New York

Conditions: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Childhood Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Ringed Sideroblasts, Refractory Cytopenia With Multilineage Dysplasia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions: iodine I 131 monoclonal antibody BC8, fludarabine phosphate, total-body irradiation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, cyclosporine, mycophenolate mofetil, laboratory biomarker analysis; Radiation · Drug · Procedure + 1 more
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 16 Years to 50 Years
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2019
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Vascular Ehlers-Danlos Syndrome
Interventions: ACER-002 (celiprolol) 200 mg BID, Placebo BID; Drug
Lead sponsor: Acer Therapeutics Inc.; Industry
Eligibility: 15 Years to 64 Years
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 1
States / cities: Culver City, California

Conditions: Sickle Cell Disease
Interventions: Crizanlizumab; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 6 Months to 100 Years
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2031
U.S. locations: 7
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Augusta, Georgia + 3 more

Conditions: Ventricular Septal Defect
Interventions: saline, ketamine; Drug
Lead sponsor: Arkansas Children's Hospital Research Institute; Other
Eligibility: Up to 1 Year
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2007
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Interventions: Del-desiran (AOC 1001); Drug
Lead sponsor: Avidity Biosciences, Inc.; Industry
Eligibility: 16 Years and older
Enrollment: 199 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 19
States / cities: Los Angeles, California • Stanford, California • Denver, Colorado + 16 more

Conditions: Hypophosphatasia (HPP)
Interventions: Asfotase Alfa; Biological
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 2
States / cities: St Louis, Missouri • Durham, North Carolina

Conditions: Hermansky-Pudlak Syndrome (HPS)
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 115 Years
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1995
U.S. locations: 1
States / cities: Bethesda, Maryland

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Laser Photocoagulation in Twin to Twin Transfusion Syndrome

Use of an Herbal Preparation to Prevent and Dissolve Kidney Stones

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

Clofarabine or Daunorubicin Hydrochloride and Cytarabine Followed By Decitabine or Observation in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia

Antibiotic "Dysbiosis" in Preterm Infants

Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis

Treatment of Chronic Thyroid Eye Disease With MHB018A

A New Diet for Patients With Autosomal Dominant Polycystic Disease (ADPKD)

Burosumab for CSHS

Neonatal CMV-Ganciclovir Follow-up Study

Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease

Treatment for Improvement of Cellulite Appearance Using Form Applicator

Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease

Phase I Trial of Tariquidar (XR9576) in Combination With Doxorubicin, Vinorelbine, or Docetaxel in Pediatric Patients With Solid Tumors

A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis

Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).

Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Total Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome

Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome

Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

Use of Ketamine Prior to Cardiopulmonary Bypass in Children

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome