Search | Clinical Trials Finder

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition, Small Intestine Cancer
Interventions: anti-thymocyte globulin, graft-versus-tumor induction therapy, cyclophosphamide, fludarabine phosphate, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Case Comprehensive Cancer Center; Other
Eligibility: Up to 70 Years
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2011
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions: cyclophosphamide, TBI; Drug · Radiation
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute; Other
Eligibility: 15 Years to 50 Years
Enrollment: 72 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1996 – 2009
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Immune Thrombocytopenia
Interventions: Avatrombopag, Placebo; Drug
Lead sponsor: Sobi, Inc.; Industry
Eligibility: 1 Year to 17 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 21
States / cities: Phoenix, Arizona • Long Beach, California • Orange, California + 18 more

Conditions: Hemophilia A
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 2 Years to 18 Years · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Immune Thrombocytopenic Purpura
Interventions: Sym001; Drug
Lead sponsor: Symphogen A/S; Industry
Eligibility: 18 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 3
States / cities: Omaha, Nebraska • Philadelphia, Pennsylvania • Charleston, South Carolina

Conditions: Bleeding Disorder, Ischemic Stroke, Hemorrhagic Stroke, Hematological Diseases, Thrombocytopenia, Coagulation Disorder, Anemia, Renal Insufficiency, Coronary Artery Disease
Interventions: XIENCE, DAPT (aspirin and/or P2Y12 receptor inhibitor); Device · Drug
Lead sponsor: Abbott Medical Devices; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 1,605 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 54
States / cities: Huntsville, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 47 more

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions: rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan, fludarabine phosphate, tacrolimus, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, methotrexate; Biological · Drug · Procedure
Lead sponsor: UNC Lineberger Comprehensive Cancer Center; Other
Eligibility: 18 Years to 55 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2015
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Acute Myeloid Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Eosinophilic Leukemia, Not Otherwise Specified, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Essential Thrombocythemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Myeloid Neoplasm, Myeloproliferative Neoplasm, Myeloproliferative Neoplasm, Unclassifiable, Overt Primary Myelofibrosis, Polycythemia Vera, Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase, Prefibrotic/Early Primary Myelofibrosis
Interventions: Azacitidine, Pevonedistat, Venetoclax; Drug
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions: busulfan, cyclophosphamide, cyclosporine, methotrexate, in vitro-treated peripheral blood stem cell transplantation; Drug · Procedure
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 18 Years to 65 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 2
States / cities: Seattle, Washington

Conditions: Heparin Induced Thrombocytopenia
Interventions: Not listed
Lead sponsor: Carelon Research; Other
Eligibility: Not listed
Enrollment: 668 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010
U.S. locations: 21
States / cities: Iowa City, Iowa • New Orleans, Louisiana • Baltimore, Maryland + 12 more

Conditions: Myeloproliferative Disorder, Myeloproliferative Syndrome, Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia
Interventions: USDA Diet, Mediterranean Diet; Other
Lead sponsor: University of California, Irvine; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Irvine, California

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions: anti-thymocyte globulin, filgrastim, cyclophosphamide, fludarabine phosphate, umbilical cord blood transplantation, methylprednisolone; Biological · Drug · Procedure
Lead sponsor: Case Comprehensive Cancer Center; Other
Eligibility: Up to 120 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2011
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: CLL, Leukemia
Interventions: Eltrombopag; Drug
Lead sponsor: M.D. Anderson Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2022
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions: alemtuzumab, therapeutic allogeneic lymphocytes, fludarabine phosphate, melphalan, tacrolimus, thiotepa, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Yale University; Other
Eligibility: 18 Years to 55 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Autoimmune Disease, Autoimmune Hemolytic Anemia, Thrombocytopenia
Interventions: Isolex 300i; Device
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 65 Years
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Chronic Myeloproliferative Disorders
Interventions: filgrastim, busulfan, cytarabine, idarubicin, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: Up to 75 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2004
U.S. locations: 7
States / cities: Anchorage, Alaska • Tampa, Florida • Chicago, Illinois + 4 more

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Nonmalignant Neoplasm, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: anti-thymocyte globulin, busulfan, carboplatin, cyclophosphamide, etoposide, fludarabine phosphate, melphalan, thiotepa, total-body irradiation; Biological · Drug · Radiation
Lead sponsor: Roswell Park Cancer Institute; Other
Eligibility: 4 Years to 70 Years
Enrollment: 361 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2019
U.S. locations: 1
States / cities: Buffalo, New York

Conditions: Myelodysplastic Syndrome
Interventions: eltrombopag olamine, Placebo; Drug · Other
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years and older
Enrollment: 98 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 24
States / cities: Birmingham, Alabama • Santa Monica, California • Stanford, California + 20 more

Conditions: Primary Immune Thrombocytopenia
Interventions: Rozanolixizumab; Drug
Lead sponsor: UCB Biopharma SRL; Industry
Eligibility: 18 Years and older
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Polycythemia Vera, Essential Thrombocythemia
Interventions: Blood specimen; Procedure
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: Not listed
Enrollment: 131 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: New York, New York

Conditions: Wounds and Injuries, Shock, Hemorrhagic
Interventions: LTOWB, Components; Biological
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 15 Years and older
Enrollment: 1,100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 13
States / cities: Birmingham, Alabama • Los Angeles, California • New Orleans, Louisiana + 10 more

Conditions: Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Interventions: Placebo, SHP655, Standard of Care; Other · Drug
Lead sponsor: Shire; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 7
States / cities: Birmingham, Alabama • Boston, Massachusetts • Minneapolis, Minnesota + 4 more

Conditions: Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome, Prenatal Benign Hypophosphatasia, Perinatal Lethal Hypophosphatasia, Odontohypophosphatasia, Adult Hypophosphatasia, Childhood-onset Hypophosphatasia, Infantile Hypophosphatasia, Hypophosphatasia, Kabuki Syndrome, Bohring-Opitz Syndrome, Narcolepsy Without Cataplexy, Narcolepsy-cataplexy, Hypersomnolence Disorder, Idiopathic Hypersomnia Without Long Sleep Time, Idiopathic Hypersomnia With Long Sleep Time, Idiopathic Hypersomnia, Kleine-Levin Syndrome, Kawasaki Disease, Leiomyosarcoma, Leiomyosarcoma of the Corpus Uteri, Leiomyosarcoma of the Cervix Uteri, Leiomyosarcoma of Small Intestine, Acquired Myasthenia Gravis, Addison Disease, Hyperacusis (Hyperacousis), Juvenile Myasthenia Gravis, Transient Neonatal Myasthenia Gravis, Williams Syndrome, Lyme Disease, Myasthenia Gravis, Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome), Isolated Klippel-Feil Syndrome, Frasier Syndrome, Denys-Drash Syndrome, Beckwith-Wiedemann Syndrome, Emanuel Syndrome, Isolated Aniridia, Axenfeld-Rieger Syndrome, Aniridia-intellectual Disability Syndrome, Aniridia - Renal Agenesis - Psychomotor Retardation, Aniridia - Ptosis - Intellectual Disability - Familial Obesity, Aniridia - Cerebellar Ataxia - Intellectual Disability, Aniridia - Absent Patella, Aniridia, Peters Anomaly - Cataract, Peters Anomaly, Potocki-Shaffer Syndrome, Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11, Silver-Russell Syndrome Due to Imprinting Defect of 11p15, Silver-Russell Syndrome Due to 11p15 Microduplication, Syndromic Aniridia, WAGR Syndrome, Wolf-Hirschhorn Syndrome, 4p16.3 Microduplication Syndrome, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, Autosomal Recessive Stickler Syndrome, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Stickler Syndrome, Mucolipidosis Type 4, X-linked Spinocerebellar Ataxia Type 4, X-linked Spinocerebellar Ataxia Type 3, X-linked Intellectual Disability - Ataxia - Apraxia, X-linked Progressive Cerebellar Ataxia, X-linked Non Progressive Cerebellar Ataxia, X-linked Cerebellar Ataxia, Vitamin B12 Deficiency Ataxia, Toxic Exposure Ataxia, Unclassified Autosomal Dominant Spinocerebellar Ataxia, Thyroid Antibody Ataxia, Sporadic Adult-onset Ataxia of Unknown Etiology, Spinocerebellar Ataxia With Oculomotor Anomaly, Spinocerebellar Ataxia With Epilepsy, Spinocerebellar Ataxia With Axonal Neuropathy Type 2, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 5, Spinocerebellar Ataxia Type 4, Spinocerebellar Ataxia Type 37, Spinocerebellar Ataxia Type 36, Spinocerebellar Ataxia Type 35, Spinocerebellar Ataxia Type 34, Spinocerebellar Ataxia Type 32, Spinocerebellar Ataxia Type 31, Spinocerebellar Ataxia Type 30, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 29, Spinocerebellar Ataxia Type 28, Spinocerebellar Ataxia Type 27, Spinocerebellar Ataxia Type 26, Spinocerebellar Ataxia Type 25, Spinocerebellar Ataxia Type 23, Spinocerebellar Ataxia Type 22, Spinocerebellar Ataxia Type 21, Spinocerebellar Ataxia Type 20, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 19/22, Spinocerebellar Ataxia Type 18, Spinocerebellar Ataxia Type 17, Spinocerebellar Ataxia Type 16, Spinocerebellar Ataxia Type 15/16, Spinocerebellar Ataxia Type 14, Spinocerebellar Ataxia Type 13, Spinocerebellar Ataxia Type 12, Spinocerebellar Ataxia Type 11, Spinocerebellar Ataxia Type 10, Spinocerebellar Ataxia Type 1 With Axonal Neuropathy, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia - Unknown, Spinocerebellar Ataxia - Dysmorphism, Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Spasticity-ataxia-gait Anomalies Syndrome, Spastic Ataxia With Congenital Miosis, Spastic Ataxia - Corneal Dystrophy, Spastic Ataxia, Rare Hereditary Ataxia, Rare Ataxia, Recessive Mitochondrial Ataxia Syndrome, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Posterior Column Ataxia - Retinitis Pigmentosa, Post-Stroke Ataxia, Post-Head Injury Ataxia, Post Vaccination Ataxia, Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract, Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus, Non-hereditary Degenerative Ataxia, Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity, Olivopontocerebellar Atrophy - Deafness, NARP Syndrome, Myoclonus - Cerebellar Ataxia - Deafness, Multiple System Atrophy, Parkinsonian Type, Multiple System Atrophy, Cerebellar Type, Multiple System Atrophy, Maternally-inherited Leigh Syndrome, Machado-Joseph Disease Type 3, Machado-Joseph Disease Type 2, Machado-Joseph Disease Type 1, Leigh Syndrome, Late-onset Ataxia With Dementia, Infection or Post Infection Ataxia, GAD Ataxia, Hereditary Episodic Ataxia, Gliadin/Gluten Ataxia, Friedreich Ataxia, Fragile X-associated Tremor/Ataxia Syndrome, Familial Paroxysmal Ataxia, Exposure to Medications Ataxia, Episodic Ataxia With Slurred Speech, Episodic Ataxia Unknown Type, Episodic Ataxia Type 7, Episodic Ataxia Type 6, Episodic Ataxia Type 5, Episodic Ataxia Type 4, Episodic Ataxia Type 3, Episodic Ataxia Type 1, Epilepsy and/or Ataxia With Myoclonus as Major Feature, Early-onset Spastic Ataxia-neuropathy Syndrome, Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity, Early-onset Cerebellar Ataxia With Retained Tendon Reflexes, Early-onset Ataxia With Dementia, Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia, Dilated Cardiomyopathy With Ataxia, Cataract - Ataxia - Deafness, Cerebellar Ataxia, Cayman Type, Cerebellar Ataxia With Peripheral Neuropathy, Cerebellar Ataxia - Hypogonadism, Cerebellar Ataxia - Ectodermal Dysplasia, Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss, Brain Tumor Ataxia, Brachydactyly - Nystagmus - Cerebellar Ataxia, Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia, Autosomal Recessive Syndromic Cerebellar Ataxia, Autosomal Recessive Spastic Ataxia With Leukoencephalopathy, Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria, Autosomal Recessive Spastic Ataxia, Autosomal Recessive Metabolic Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine, Autosomal Recessive Ataxia, Beauce Type, Autosomal Recessive Ataxia Due to Ubiquinone Deficiency, Autosomal Recessive Ataxia Due to PEX10 Deficiency, Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia, Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia, Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome, Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity, Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency, Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect, Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion, Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation, Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness, Autosomal Recessive Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly, Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation, Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy, Autosomal Dominant Spastic Ataxia Type 1, Autosomal Dominant Spastic Ataxia, Autosomal Dominant Optic Atrophy, Ataxia-telangiectasia Variant, Ataxia-telangiectasia, Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy, Autosomal Dominant Cerebellar Ataxia Type 4, Autosomal Dominant Cerebellar Ataxia Type 3, Autosomal Dominant Cerebellar Ataxia Type 2, Autosomal Dominant Cerebellar Ataxia Type 1, Autosomal Dominant Cerebellar Ataxia, Ataxia-telangiectasia-like Disorder, Ataxia With Vitamin E Deficiency, Ataxia With Dementia, Ataxia - Oculomotor Apraxia Type 1, Ataxia - Other, Ataxia - Genetic Diagnosis - Unknown, Acquired Ataxia, Adult-onset Autosomal Recessive Cerebellar Ataxia, Alcohol Related Ataxia, Multiple Endocrine Neoplasia, Multiple Endocrine Neoplasia Type II, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Multiple Endocrine Neoplasia, Type IV, Multiple Endocrine Neoplasia, Type 3, Multiple Endocrine Neoplasia (MEN) Syndrome, Multiple Endocrine Neoplasia Type 2B, Multiple Endocrine Neoplasia Type 2A, Atypical Hemolytic Uremic Syndrome, Atypical HUS, Wiedemann-Steiner Syndrome, Breast Implant-Associated Anaplastic Large Cell Lymphoma, Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA), Hemophagocytic Lymphohistiocytosis, Behcet's Disease, Alagille Syndrome, Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD), Lowe Syndrome, Pitt Hopkins Syndrome, 1p36 Deletion Syndrome, Jansen Type Metaphyseal Chondrodysplasia, Cockayne Syndrome, Chronic Recurrent Multifocal Osteomyelitis, CRMO, Malan Syndrome, Hereditary Sensory and Autonomic Neuropathy Type Ie, VCP Disease, Hypnic Jerking, Sleep Myoclonus, Mollaret Meningitis, Recurrent Viral Meningitis, CRB1, Leber Congenital Amaurosis, Retinitis Pigmentosa, Rare Retinal Disorder, KCNMA1-Channelopathy, Primary Biliary Cirrhosis, ZMYND11, Transient Global Amnesia, Glycogen Storage Disease, Alstrom Syndrome, White Sutton Syndrome, DNM1, EIEE31, Myhre Syndrome, Recurrent Respiratory Papillomatosis, Laryngeal Papillomatosis, Tracheal Papillomatosis, Refsum Disease, Nicolaides Baraitser Syndrome, Leukodystrophy, Tango2, Cauda Equina Syndrome, Rare Gastrointestinal Disorders, Achalasia-Addisonian Syndrome, Achalasia Cardia, Achalasia Icrocephaly Syndrome, Anal Fistula, Congenital Sucrase-Isomaltase Deficiency, Eosinophilic Gastroenteritis, Idiopathic Gastroparesis, Hirschsprung Disease, Rare Inflammatory Bowel Disease, Intestinal Pseudo-Obstruction, Scleroderma, Short Bowel Syndrome, Sacral Agenesis, Sacral Agenesis Syndrome, Caudal Regression, Scheuermann Disease, SMC1A Truncated Mutations (Causing Loss of Gene Function), Cystinosis, Juvenile Nephropathic Cystinosis, Nephropathic Cystinosis, Kennedy Disease, Spinal Bulbar Muscular Atrophy, Warburg Micro Syndrome, Mucolipidoses, Mitochondrial Diseases, Mitochondrial Aminoacyl-tRNA Synthetases, Mt-aaRS Disorders, Hypertrophic Olivary Degeneration, Non-Ketotic Hyperglycinemia, Fish Odor Syndrome, Halitosis, Isolated Congenital Asplenia, Lambert Eaton (LEMS), Biliary Atresia, STAG1 Gene Mutation, Coffin Lowry Syndrome, Borjeson-Forssman-Lehman Syndrome, Blau Syndrome, Arginase 1 Deficiency, HSPB8 Myopathy, Beta-Mannosidosis, TBX4 Syndrome, DHDDS Gene Mutations, MAND-MBD5-Associated Neurodevelopmental Disorder, Constitutional Mismatch Repair Deficiency (CMMRD), SPATA5 Disorder, SPATA5L1 Related Disorder, Acrodysostosis, Multi-systematic Smooth Muscle Dysfunction Syndrome, CRELD1 (Cysteine Rich With EGF Like Domains 1), GNB1 Syndrome, Pyruvate Dehydrogenase Complex Deficiency Disease, Beta Mannosidosis, Kbg Syndrome, Labrune Syndrome, Metachromatic Leukodystrophy (MLD), Moyamoya Disease, OPHN1 Syndrome, Oculopharyngeal Muscular Dystrophy (OPMD), TUBB3 Mutation, WOREE (WWOX-related Epileptic Encephalopathy, SCAR12, Skraban-Deardorff Syndrome, Hereditary Myopathy With Early Respiratory Failure
Interventions: Not listed
Lead sponsor: Sanford Health; Other
Eligibility: Not listed
Enrollment: 20,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2100
U.S. locations: 1
States / cities: Sioux Falls, South Dakota

Conditions: Warm Autoimmune Hemolytic Anemia (WAIHA), ITP - Immune Thrombocytopenia, Warm Autoimmune Hemolytic Anemia, Immune Thrombocytopenic Purpura
Interventions: CTX112; Biological
Lead sponsor: CRISPR Therapeutics AG; Industry
Eligibility: 18 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2033
U.S. locations: 3
States / cities: Boston, Massachusetts • Rochester, Minnesota • Omaha, Nebraska

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Non-Ablative Allo HSCT For Hematologic Malignancies or SAA

Donor Bone Marrow Transplant in Treating Patients With Leukemia, Lymphoma, or Nonmalignant Hematologic Disorders

Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

Platelet Function in Patients With Hemophilia A

Open Label Trial on the Safety and Efficacy of Sym001 in the Treatment of Immune Thrombocytopenic Purpura (ITP)

XIENCE 28 USA Study

Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen

Azacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome, Acute Myeloid Leukemia, or Myeloproliferative Disorder

Heparin-Induced Thrombocytopenia - Retrospective Analysis of Data on Incidence and Outcomes Study

The NUTRIENT Trial (NUTRitional Intervention Among myEloproliferative Neoplasms): Feasibility Phase

Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

Phase II Eltrombopag in Chronic Lymphocytic Leukemia (CLL)

Alemtuzumab and Combination Chemotherapy Followed By Donor Lymphocytes in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer

Treatment of Autoimmune Thrombocytopenia (AITP)

Chemotherapy Followed by Peripheral Stem Cell Transplantation in Treating Patients With Myelofibrosis

Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer

Eltrombopag Treatment of Thrombocytopenia in Subjects With Advanced Myelodysplastic Syndrome (MDS) or Secondary Acute Myeloid Leukemia After MDS (sAML/MDS)

A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Tissue Banking Study - Polycythemia Vera or Essential Thrombocythemia (PV & ET) Patients

Trauma Resuscitation With Low-Titer Group O Whole Blood or Products

Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Safety and Preliminary Efficacy of CTX112 in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease