Welcome to Paid Research Studies




  • Conditions:   Alpha-Mannosidase B Deficiency;   Lysosomal Alpha B Mannosidosis;   Alpha-Mannosidase Deficiency
    Intervention:  
    Sponsor:   Centogene AG Rostock
    Recruiting

  • Conditions:   Immunodeficiency;   Skeletal Abnormalities;   Deafness;   Mental Retardation;   Gingival Hypertrophy;   Facial Dysmorphism
    Intervention:  
    Sponsor:   Centogene AG Rostock
    Recruiting

  • Conditions:   Bone Marrow Failure Syndrome;   Thalassemia;   Sickle Cell Disease;   Diamond Blackfan Anemia;   Acquired Neutropenia in Newborn;   Acquired Anemia Hemolytic;   Acquired Thrombocytopenia;   Hemophagocytic Lymphohistiocytoses;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Common Variable Immunodeficiency;   X-linked Lymphoproliferative Disease;   Severe Combined Immunodeficiency;   Hurler Syndrome;   Mannosidosis;   Adrenoleukodystrophy
    Interventions:   Drug: Thiotepa--single daily dose;   Drug: Thiotepa--escalated dose
    Sponsor:   University of Florida
    Recruiting

  • Conditions:   MLD;   Krabbe Disease;   ALD;   MPS I;   MPS II;   MPS III;   Vanishing White Matter Disease;   GM3 Gangliosidosis;   PKAN;   Tay-Sachs Disease;   NP Deficiency;   Osteopetrosis;   Alpha-Mannosidosis;   Sandhoff Disease;   Niemann-Pick Diseases;   MPS IV;   Gaucher Disease;   GAN;   GM1 Gangliosidoses;   Morquio Disease;   S-Adenosylhomocysteine Hydrolase Deficiency;   Batten Disease;   Pelizaeus-Merzbacher Disease;   Leukodystrophy;   Lysosomal Storage Diseases;   Purine Nucleoside Phosphorylase Deficiency;   Multiple Sulfatase Deficiency Disease
    Interventions:   Other: Palliative Care;   Biological: Hematopoetic Stem Cell Transplantation
    Sponsor:   University of Pittsburgh
    Recruiting

  • Conditions:   Mucopolysaccharidosis Disorders;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Alpha-Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Glycoprotein Metabolic Disorders;   Sphingolipidoses;   Recessive Leukodystrophies;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann-Pick B;   Niemann-Pick C Subtype 2;   Sphingomyelin Deficiency;   Peroxisomal Disorders;   Adrenoleukodystrophy With Cerebral Involvement;   Zellweger Syndrome;   Neonatal Adrenoleukodystrophy;   Infantile Refsum Disease;   Acyl-CoA Oxidase Deficiency;   D-Bifunctional Enzyme Deficiency;   Multifunctional Enzyme Deficiency;   Alpha-methylacyl-CoA Racmase Deficiency;   Mitochondrial Neurogastrointestingal Encephalopathy;   Severe Osteopetrosis;   Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);   Inherited Metabolic Disorders
    Interventions:   Biological: Stem Cell Transplantation;   Drug: IMD Preparative Regimen;   Drug: Osteopetrosis Only Preparative Regimen;   Drug: Osteopetrosis Haploidentical Only Preparative Regimen;   Drug: cALD SR-A (Standard-Risk, Regimen A);   Drug: cALD SR-B (Standard-Risk, Regimen B);   Drug: cALD HR-D (High-Risk, Regimen C);   Drug: cALD HR-D (High-Risk, Regimen D)
    Sponsor:   Masonic Cancer Center, University of Minnesota
    Recruiting

  • Conditions:   Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn;   Alpha-Mannosidosis;   Sanfilippo Mucopolysaccharidoses
    Intervention:   Biological: DUOC-01
    Sponsors:   Joanne Kurtzberg, MD;   The Marcus Foundation
    Recruiting

  • Conditions:   Primary Immunodeficiency (PID);   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Inflammatory Conditions
    Interventions:   Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
    Sponsor:   Paul Szabolcs
    Recruiting