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Clinical Trials

NCT03655223 | ACTIVE_NOT_RECRUITING
30000 participants

Early Check: Expanded Screening in Newborns

Study Type:
OBSERVATIONAL
Last Updated:
2026-07-01
Start Date:
2018-10-15
Completion:
2026-11-30
Conditions
  • SRD
  • Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency
  • Barth Syndrome
  • Adrenocorticotropic Hormone Deficiency
  • Transcobalamin II Deficiency
  • Thyroid Dyshormonogenesis 3
  • Segawa Syndrome, Autosomal Recessive
  • Autosomal Recessive Nonsyndromic Hearing Loss
  • Thyroid Dyshormonogenesis 2A
  • Congenital Isolated Thyroid Stimulating Hormone Deficiency
  • Hypothyroidism Due to TSH Receptor Mutations
  • Usher Syndrome Type 1C
  • Usher Syndrome Type 1G (Diagnosis)
  • Von Willebrand Disease, Type 3
  • Spinal Muscular Atrophy
  • Fragile X Syndrome
  • Fragile X - Premutation
  • Duchenne Muscular Dystrophy
  • Hyperinsulinemic Hypoglycemia, Familial 1
  • Diabetes Mellitus
  • Adrenoleukodystrophy, Neonatal
  • Medium-chain Acyl-CoA Dehydrogenase Deficiency
  • Very Long Chain Acyl Coa Dehydrogenase Deficiency
  • Beta-ketothiolase Deficiency
  • Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency
  • Primary Hyperoxaluria Type 1
  • Congenital Bile Acid Synthesis Defect Type 2
  • Pyridoxine-Dependent Epilepsy
  • Hereditary Fructose Intolerance
  • Hypophosphatasia
  • Hyperargininemia
  • Mucopolysaccharidosis Type 6
  • Argininosuccinic Aciduria
  • Citrullinemia, Type I
  • Wilson Disease
  • Maple Syrup Urine Disease, Type 1A
  • Maple Syrup Urine Disease, Type 1B
  • Biotinidase Deficiency
  • Neonatal Severe Primary Hyperparathyroidism
  • Intrinsic Factor Deficiency
  • Usher Syndrome Type 1D/F Digenic (Diagnosis)
  • Cystic Fibrosis
  • Stickler Syndrome Type 2
  • Stickler Syndrome Type 1
  • Alport Syndrome, Autosomal Recessive
  • Alport Syndrome, X-Linked
  • Carbamoyl Phosphate Synthetase I Deficiency Disease
  • Carnitine Palmitoyl Transferase 1A Deficiency
  • Carnitine Palmitoyltransferase II Deficiency
  • Cystinosis
  • Chronic Granulomatous Disease
  • Cerebrotendinous Xanthomatoses
  • Maple Syrup Urine Disease, Type 2
  • Severe Combined Immunodeficiency Due to DCLRE1C Deficiency
  • Thyroid Dyshormonogenesis 6
  • Thyroid Dyshormonogenesis 5
  • Supravalvar Aortic Stenosis
  • Factor X Deficiency
  • Hemophilia A
  • Hemophilia B
  • Tyrosinemia, Type I
  • Fructose 1,6 Bisphosphatase Deficiency
  • Glycogen Storage Disease Type I
  • G6PD Deficiency
  • Glycogen Storage Disease II
  • Galactokinase Deficiency
  • Mucopolysaccharidosis Type IV A
  • Galactosemias
  • Guanidinoacetate Methyltransferase Deficiency
  • Agat Deficiency
  • Glutaryl-CoA Dehydrogenase Deficiency
  • Gtp Cyclohydrolase I Deficiency
  • Hyperinsulinism-Hyperammonemia Syndrome
  • Primary Hyperoxaluria Type 2
  • 3-Hydroxyacyl-CoA Dehydrogenase Deficiency
  • Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
  • Mitochondrial Trifunctional Protein Deficiency
  • Sickle Cell Disease
  • Beta-Thalassemia
  • Holocarboxylase Synthetase Deficiency
  • 3-Hydroxy-3-Methylglutaric Aciduria
  • Primary Hyperoxaluria Type 3
  • Hermansky-Pudlak Syndrome 1
  • Hermansky-Pudlak Syndrome 4
  • Apparent Mineralocorticoid Excess
  • HSDB
  • CBAS1
  • Mucopolysaccharidosis Type 2
  • Mucopolysaccharidosis Type 1
  • Severe Combined Immunodeficiency, X Linked
  • Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency
  • Diabetes Mellitus, Permanent Neonatal
  • Isovaleric Acidemia
  • Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder)
  • Jervell and Lange-Nielsen Syndrome 2
  • Hyperinsulinemic Hypoglycemia, Familial, 2
  • Diabetes Mellitus, Permanent Neonatal, With Neurologic Features
  • Jervell and Lange-Nielsen Syndrome 1
  • Lysosomal Acid Lipase Deficiency
  • CblF
  • 3-Methylcrotonyl CoA Carboxylase 1 Deficiency
  • 3-Methylcrotonyl CoA Carboxylase 2 Deficiency
  • Waardenburg Syndrome Type 2A
  • Methylmalonic Aciduria cblA Type
  • Methylmalonic Aciduria cblB Type
  • Methylmalonic Aciduria and Homocystinuria Type cblC
  • MAHCD
  • Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency
  • Congenital Disorder of Glycosylation Type 1B
  • Mthfr Deficiency
  • Methylcobalamin Deficiency Type Cbl G (Disorder)
  • Methylcobalamin Deficiency Type cblE
  • Usher Syndrome, Type 1B
  • N-acetylglutamate Synthase Deficiency
  • Ornithine Transcarbamylase Deficiency
  • Phenylketonurias
  • Waardenburg Syndrome Type 1
  • Congenital Hypothyroidism
  • Propionic Acidemia
  • Usher Syndrome, Type 1F
  • Pancreatic Agenesis 1
  • Hereditary Hypophosphatemic Rickets
  • Glycogen Storage Disease IXB
  • Glycogen Storage Disease IXC
  • MOWS
  • Epilepsy, Early-Onset, Vitamin B6-Dependent
  • Pyridoxal Phosphate-Responsive Seizures
  • Pituitary Hormone Deficiency, Combined, 1
  • Ptsd
  • Dihydropteridine Reductase Deficiency
  • Severe Combined Immunodeficiency Due to RAG1 Deficiency
  • Severe Combined Immunodeficiency Due to RAG2 Deficiency
  • Retinoblastoma
  • Multiple Endocrine Neoplasia Type 2B
  • Pseudohypoaldosteronism, Type I
  • Liddle Syndrome
  • Biotin-Responsive Basal Ganglia Disease
  • SCD
  • DIAR1
  • GSD1C
  • Acrodermatitis Enteropathica
  • Thyroid Dyshormonogenesis 1
  • Riboflavin Transporter Deficiency
  • Waardenburg Syndrome, Type 2E
  • Combined Immunodeficiency Due to ZAP70 Deficiency
  • Adenine Phosphoribosyltransferase Deficiency
  • Metachromatic Leukodystrophy
  • Canavan Disease
  • Menkes Disease
  • Carbonic Anhydrase VA Deficiency
  • Developmental and Epileptic Encephalopathy 2
  • 17 Alpha-Hydroxylase Deficiency
  • Smith-Lemli-Opitz Syndrome
  • Krabbe Disease
  • Glutathione Synthetase Deficiency
  • Mucopolysaccharidosis Type 7
  • Rett Syndrome
  • Molybdenum Cofactor Deficiency, Type A
  • Niemann-Pick Disease, Type C1
  • Niemann-Pick Disease Type C2
  • Ornithine Aminotransferase Deficiency
  • 3-Phosphoglycerate Dehydrogenase Deficiency
  • Leber Congenital Amaurosis 2
  • Dravet Syndrome
  • Mucopolysaccharidosis Type 3 A
  • Ornithine Translocase Deficiency
  • Carnitine-acylcarnitine Translocase Deficiency
  • Glucose Transporter Type 1 Deficiency Syndrome
  • Creatine Transporter Deficiency
  • Niemann-Pick Disease Type A
  • Pitt Hopkins Syndrome
  • Tuberous Sclerosis 1
  • Tuberous Sclerosis 2
  • Ataxia With Isolated Vitamin E Deficiency
  • Angelman Syndrome
  • Prader-Willi Syndrome
  • Homocystinuria
  • Permanent Neonatal Diabetes Mellitus
  • Transient Neonatal Diabetes Mellitus
  • Factor VII Deficiency
  • Glycogen Storage Disease Type IXA1
  • Glycogen Storage Disease, Type IXA2
  • Glycogen Storage Disease IC
  • Glycogen Storage Disease Type IB
  • Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions
  • DIAGNOSTIC_TEST
No Results Yet
NCT03494569 | SUSPENDED | PHASE1
36 participants

Total Marrow and Lymphoid Irradiation, Fludarabine, and Melphalan Before Donor Stem Cell Transplant in Treating Participants With High-Risk Acute Leukemia or Myelodysplastic Syndrome

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2018-07-06
Completion:
2026-09-08
Conditions
  • Acute Lymphoblastic Leukemia
  • Acute Lymphoblastic Leukemia in Remission
  • Acute Myeloid Leukemia
  • Acute Myeloid Leukemia in Remission
  • Hematopoietic Cell Transplantation Recipient
  • Minimal Residual Disease
  • Myelodysplastic Syndrome
  • Secondary Acute Myeloid Leukemia
Interventions
  • DRUG
  • OTHER
  • RADIATION
No Results Yet
NCT03179904 | COMPLETED | PHASE2
17 participants

TVB-2640 and Trastuzumab With Paclitaxel or Endocrine Therapy for Treatment of HER2 Positive Metastatic Breast Cancer

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2017-08-03
Completion:
2024-07-30
Conditions
  • Advanced Breast Carcinoma
  • HER2-Positive Breast Carcinoma
  • Anatomic Stage III Breast Cancer AJCC v8
  • Anatomic Stage IV Breast Cancer AJCC v8
  • HER2-positive Breast Cancer
Interventions
  • DRUG
  • PROCEDURE
  • BIOLOGICAL
Results Available
NCT03101891 | COMPLETED | PHASE1
55 participants

Renal Anhydramnios Fetal Therapy

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2018-12-21
Completion:
2025-03-10
Conditions
  • Anhydramnios
  • Potter Syndrome
  • Lung Hypoplasia
  • Multicystic Dysplastic Kidney
  • Multicystic Renal Dysplasia, Bilateral
  • Lower Urinary Tract Obstructive Syndrome
  • Fetal Renal Anomaly
Interventions
  • PROCEDURE
  • DEVICE
  • DRUG
Results Available
NCT02964884 | ACTIVE_NOT_RECRUITING | PHASE2
120 participants

Interventions for Reading Disabilities in NF1

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2016-11-30
Completion:
2027-01-31
Conditions
  • Neurofibromatosis Type 1
  • Learning Disability
  • Reading Disability
  • NF1
Interventions
  • DRUG
  • BEHAVIORAL
No Results Yet
NCT02957968 | ACTIVE_NOT_RECRUITING | PHASE2
46 participants

Neoadjuvant Pembrolizumab + Decitabine Followed by Std Neoadj Chemo for Locally Advanced HER2- Breast Ca

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2017-01-24
Completion:
2022-08-04
Conditions
  • Breast Adenocarcinoma
  • Estrogen Receptor- Negative Breast Cancer
  • Estrogen Receptor-positive Breast Cancer
  • HER2/Neu Negative
  • Invasive Breast Carcinoma
  • Progesterone Receptor Negative
  • Progesterone Receptor Positive Tumor
  • Stage II Breast Cancer
  • Stage IIA Breast Cancer
  • Stage IIB Breast Cancer
  • Stage IIIA Breast Cancer
  • Stage IIIB Breast Cancer
  • Triple-negative Breast Carcinoma
Interventions
  • DRUG
Results Available
NCT02814383 | COMPLETED
70 participants

Prediction of Brain Injury in Premature Infants

Study Type:
OBSERVATIONAL
Last Updated:
2026-07-01
Start Date:
2016-08-11
Completion:
2025-05-21
Conditions
  • Intraventricular Hemorrhage
  • Periventricular Leukomalacia
  • Brain Injury
  • Extreme Prematurity
Interventions
  • OTHER
No Results Yet
NCT02502266 | ACTIVE_NOT_RECRUITING | PHASE2/PHASE3
582 participants

Testing the Combination of Cediranib and Olaparib in Comparison to Each Drug Alone or Other Chemotherapy in Recurrent Platinum-Resistant Ovarian Cancer

Study Type:
INTERVENTIONAL
Last Updated:
2026-07-01
Start Date:
2016-05-03
Completion:
2023-06-12
Conditions
  • Fallopian Tube Clear Cell Adenocarcinoma
  • Fallopian Tube Endometrioid Adenocarcinoma
  • Fallopian Tube Serous Adenocarcinoma
  • Fallopian Tube Transitional Cell Carcinoma
  • Fallopian Tube Undifferentiated Carcinoma
  • Ovarian Clear Cell Adenocarcinoma
  • Ovarian Endometrioid Adenocarcinoma
  • Ovarian Seromucinous Carcinoma
  • Ovarian Serous Adenocarcinoma
  • Ovarian Transitional Cell Carcinoma
  • Ovarian Undifferentiated Carcinoma
  • Primary Peritoneal Serous Adenocarcinoma
  • Recurrent Fallopian Tube Carcinoma
  • Recurrent Ovarian Carcinoma
  • Recurrent Primary Peritoneal Carcinoma
Interventions
  • DRUG
  • PROCEDURE
  • OTHER
Results Available
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