Study of ATI-450 in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS)
Cryopyrin-Associated Periodic SyndromeThis is a Phase 2 study to investigate the safety and efficacy of ATI-450 for the Maintenance of Remission in Patients with Cryopyrin-Associated Periodic Syndrome (CAPS) Previously Managed with Anti-IL-1 Therapy.
Treatment of Type I Hepatorenal Syndrome (HRS) With Pentoxyfylline
Hepatorenal SyndromePentoxyfylline therapy in addition to the standard of care of albumin, midodrine and octreotide therapy is superior to the standard of care alone in the treatment of Type I hepatorenal syndrome in the first 14 days of hospitalization.
Sorafenib for Hepatopulmonary Syndrome
Hepatopulmonary SyndromeThe main purpose of this clinical trial is to determine the safety and effects of the study drug, sorafenib, in adults diagnosed with hepatopulmonary syndrome (HPS). The study will evaluate how well the drug is tolerated and its effect on the level of oxygen in the blood and the function of the lung vessels.
CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy
Previously Treated Myelodysplastic SyndromesThis pilot clinical trial studies 6, 8-bis (benzylthio) octanoic acid (CPI-613) in treating patients with myelodysplastic syndromes who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of tumor cells and may be an effective treatment for myelodysplastic syndromes that did not respond to previous therapy.
Baminercept, a Lymphotoxin-Beta Receptor Fusion Protein, for Treatment of Sjögren's Syndrome
Primary Sjögren's SyndromeThe purpose of the study is to find out if the experimental study agent, baminercept, is effective in treating patients with Sjögren's syndrome. The study will also determine if the study agent can be safely given to patients with Sjögren's syndrome; examine how it affects symptoms of the disease; and attempt to understand how baminercept affects the underlying mechanisms of Sjögren's syndrome and the immune system.
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
Mucopolysaccharidosis IVAMorquio A Syndrome1 moreThe primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks. Secondary objectives were to investigate the effect of the two doses on exercise capacity for up to 196 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 was assessed.
Efficacy Study of Raloxifene to Induce Ovulation in Polycystic Ovarian Syndrome
Polycystic Ovarian SyndromeThis study examines Raloxifene versus Clomiphene to induce ovulation in women with polycystic ovarian syndrome (PCOS).
Treatment of Port-wine Mark in Sturge-Weber Syndrome Using Topical Timolol
Sturge Weber SyndromePort-wine MarkPrimary Objective: • To assess the possible utility of topical timolol in the management of port-wine mark (PWM) in Sturge-Weber syndrome in children.
Tocilizumab for the Treatment of Behcet's Syndrome
Behcet SyndromeThis is a double-blind placebo controlled study targeting individuals with active Behcet's Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable dose for 4 weeks before enrollment. Prednisone or equivalent (< 10mg/day) permitted if dose stable for 6 weeks prior to enrollment. The study will investigate the safety of tocilizumab for this vasculitic condition in addition to its efficacy. The planned sample size is 30 participants per arm for a total of 60 participants. The study would be for 3 months, with a safety follow up at 2 months after study termination. Study participants will stay on their current treatments and either tocilizumab or placebo infusions will be given every 4 weeks in addition. Patients will be randomized to Actemra IV 8mg/kg Q 4 weeks X 3 doses or placebo.
Safety and Efficacy Study of PD-616 Plus Cytarabine to Treat Acute Myelogenous Leukemia or Myelodysplastic...
Acute Myelogenous LeukemiaMyelodysplastic SyndromeThe purpose of this study is to determine whether PD-616 in combination with low-dose Cytarabine is safe and effective in the treatment of untreated or relapsed/refractory acute myelogenous leukemia (AML) or high-risk myelodysplastic syndrome (MDS).