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NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease | MicrobiomeTrials
← Back to trials NCT07398508
NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease Recruiting PHASE1, PHASE2 INTERVENTIONAL
Read the full scientific description See full eligibility requirements Trial Details Start: 2026-08-10Est. Completion: 2030-06-30
Enrollment target: 6 participantsLocations Vagelos College of Physicians and Surgeons, Columbia University, New York, New York, United States Other microbiome trials you might be interested in 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
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
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
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