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14 studies found for:    "dyskeratosis congenita" OR "Dyskeratosis Congenita"
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Rank Status Study
1 Active, not recruiting SCT for Dyskeratosis Congenita or SAA
Conditions: Dyskeratosis Congenita;   Aplastic Anemia
Interventions: Drug: Campath 1H;   Drug: Cyclophosphamide;   Drug: Fludarabine;   Procedure: Total Body Irradiation;   Procedure: Stem Cell Transplantation;   Drug: antithymocyte globulin;   Drug: Methylprednisolone
2 Recruiting Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Conditions: Dyskeratosis Congenita;   Aplastic Anemia
Interventions: Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Cyclophosphamide;   Radiation: Total Body Irradiation;   Biological: Stem Cell Transplant;   Drug: Anti-thymocyte globulin
3 Terminated Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Conditions: Fanconi Anemia;   Dyskeratosis Congenita
Intervention: Drug: danazol
4 Recruiting Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Conditions: Dyskeratosis Congenita;   Hoyeraal Hreidarsson Syndrome;   Revesz Syndrome;   Aplastic Anemia
Interventions: Biological: alemtuzumab;   Drug: Fludarabine;   Drug: Cyclosporins;   Drug: Mycophenolate mofetil
5 Completed Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Conditions: Shwachman Syndrome;   Fanconi's Anemia;   Dyskeratosis Congenita;   Thrombocytopenia
Intervention: Drug: filgrastim
6 Completed Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
Conditions: Dyskeratosis Congenita;   Fanconi Anemia;   Myelodysplastic Syndromes;   Pearson Marrow-pancreas Syndrome;   Shwachman-diamond Syndrome
Interventions: Genetic: polymerase chain reaction;   Other: flow cytometry;   Other: immunologic technique;   Procedure: biopsy
7 Suspended
Has Results
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: Thalassemia;   Sickle Cell Disease;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic-granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Schwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Fanconi Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia
Intervention: Drug: Alefacept
8 Recruiting Familial Investigations of Childhood Cancer Predisposition
Conditions: Acute Leukemia;   Adenomatous Polyposis;   Adrenocortical Carcinoma;   AML;   BAP1 Tumor Predisposition Syndrome;   Carney Complex;   Choroid Plexus Carcinoma;   Constitutional Mismatch Repair Deficiency Syndrome;   Diamond-Blackfan Anemia;   DICER1 Syndrome;   Dyskeratosis Congenita;   Emberger Syndrome;   Familial Acute Myeloid Leukaemia;   Familial Adenomatous Polyposis;   Fanconi Anemia;   Familial Cancer;   Familial Wilms Tumor;   Familial Neuroblastoma;   GIST;   Hereditary Breast and Ovarian Cancer;   Hereditary Paraganglioma-Pheochromocytoma Syndrome;   Hodgkin Lymphoma;   Juvenile Polyposis;   Li-Fraumeni Syndrome;   Lynch Syndrome;   MDS;   Melanoma Syndrome;   Multiple Endocrine Neoplasia Type 1;   Multiple Endocrine Neoplasia Type 2;   Neuroblastoma;   Neurofibromatosis Type 1;   Neurofibromatosis Type II;   Nevoid Basal Cell Carcinoma Syndrome;   Non Hodgkin Lymphoma;   Noonan Syndrome and Other Rasopathy;   Overgrowth Syndromes;   Pancreatic Cancer;   Peutz-Jeghers Syndrome;   Pheochromocytoma/Paraganglioma;   PTEN Hamartoma Tumor Syndrome;   Retinoblastoma;   Rhabdoid Tumor Predisposition Syndrome;   Rhabdomyosarcoma;   Rothmund-Thomson Syndrome;   Tuberous Sclerosis;   Von Hippel-Lindau Disease
Intervention:
9 Recruiting Investigation of the Genetics of Hematologic Diseases
Conditions: Bone Marrow Failure Syndromes;   Erythrocyte Disorder;   Leukocyte Disorder;   Hemostasis;   Blood Coagulation Disorder;   Sickle Cell Disease;   Dyskeratosis Congenita;   Diamond-Blackfan Anemia;   Congenital Thrombocytopenia;   Severe Congenital Neutropenia;   Fanconi Anemia
Intervention:
10 Recruiting BMT Abatacept for Non-Malignant Diseases
Conditions: Hurler Syndrome;   Fanconi Anemia;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Shwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia;   Thalassemia Major;   Hemophagocytic Lymphohistiocytosis;   Sickle Cell Disease
Intervention: Drug: Abatacept
11 Recruiting Cancer in Inherited Bone Marrow Failure Syndromes
Conditions: Aplastic Anemia;   Head and Neck Neoplasms;   Bone Marrow Failure
Intervention:
12 Recruiting Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Conditions: Primary Immune Deficiency Disorders;   Hemophagocytic Lymphohistiocytosis;   Inherited Bone Marrow Failure Syndrome;   Hemoglobinopathies;   Metabolic Disorders
Intervention: Biological: BPX-501 and AP1903
13 Recruiting Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Condition: Non-Malignant
Interventions: Procedure: Allogeneic Bone Marrow Transplantation;   Biological: Anti-Thymocyte Globulin;   Drug: Cyclosporine;   Drug: Fludarabine Phosphate;   Other: Laboratory Biomarker Analysis;   Drug: Methotrexate;   Drug: Mycophenolate Mofetil;   Procedure: Peripheral Blood Stem Cell Transplantation;   Drug: Tacrolimus;   Radiation: Total-Body Irradiation;   Drug: Treosulfan;   Procedure: Umbilical Cord Blood Transplantation
14 Recruiting Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa

Study has passed its completion date and status has not been verified in more than two years.