Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
68 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 Unknown  Phase I Study of Vasoactive Intestinal Peptide in Patients With Acute Respiratory Distress Syndrome and Sepsis
Conditions: Sepsis;   Respiratory Distress Syndrome;   Respiratory Distress Syndrome, Adult
Intervention: Drug: vasoactive intestinal peptide
22 Unknown  Study of NTBC for Tyrosinemia I
Condition: Tyrosinemia I
Intervention: Drug: NTBC
23 Unknown  Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Conditions: Gaucher's Disease;   Osteopenia
Interventions: Drug: alendronate sodium;   Drug: calcium carbonate;   Drug: cholecalciferol
24 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
Intervention:
25 Unknown  Phase III Randomized Study of Collagenase in Patients With Residual Stage Dupuytren's Disease
Condition: Dupuytren's Contracture
Intervention: Drug: collagenase
26 Unknown  Study of Bile Acids in Patients With Peroxisomal Disorders
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Bifunctional Enzyme Deficiency;   Adrenoleukodystrophy
Interventions: Drug: chenodeoxycholic acid;   Drug: cholic acid;   Drug: ursodiol
27 Unknown  International Registry for Severe Chronic Neutropenia
Condition: Neutropenia
Intervention:
28 Unknown  Phase III Randomized Study of Oral Acyclovir in Infants With Herpes Simplex Virus Infection Involving the Central Nervous System
Condition: Herpes Simplex
Intervention: Drug: acyclovir
29 Recruiting A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
Conditions: MPS 7;   Sly Syndrome;   Mucopolysaccharidosis;   MPS VII
Intervention: Drug: UX003
30 Unknown  Phase I Study of Alpha-Melanocyte Stimulating Hormone in Patients With Acute Renal Failure
Condition: Acute Renal Failure
Intervention: Drug: alpha-melanocyte stimulating hormone
31 Unknown  Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
Condition: Amino Acid Metabolism, Inborn Errors
Interventions: Behavioral: Protein and calorie controlled diet;   Genetic: Ornithine transcarbamylase vector
32 Unknown  Studies in Porphyria I: Characterization of Enzyme Defects
Condition: Porphyria
Intervention:
33 Unknown  Randomized Study of L-Baclofen in Patients With Refractory Trigeminal Neuralgia
Condition: Trigeminal Neuralgia
Intervention: Drug: L-baclofen
34 Unknown  Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia
Condition: Sickle Cell Anemia
Interventions: Drug: clotrimazole;   Drug: hydroxyurea
35 Unknown  Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis
Condition: Cystic Fibrosis
Interventions: Drug: tauroursodeoxycholic acid;   Drug: ursodiol
36 Unknown  Leuprolide in Determining the Cause of Gonadotropin Deficiency
Condition: Hypogonadism
Interventions: Drug: gonadotropin releasing hormone;   Drug: leuprolide
37 Unknown  Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
Intervention:
38 Unknown  Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
39 Unknown  Study of Depression, Peptides, and Steroids in Cushing's Syndrome
Condition: Cushing's Syndrome
Intervention:
40 Unknown  Randomized Study of Tauroursodeoxycholic Acid in Prophylactic Therapy of Total Parenteral Nutrition Associated Cholestasis in Infants
Condition: Cholestasis
Intervention: Drug: tauroursodeoxycholic acid

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Indicates status has not been verified in more than two years