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NCT ID: NCT01757184 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

ARISE
Start date: January 22, 2013
Phase: Phase 3
Study type: Interventional

This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]). Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.

NCT ID: NCT01756339 Completed - Clinical trials for Community-acquired Bacterial Pneumonia

Efficacy and Safety Study of Oral Solithromycin (CEM-101) Compared to Oral Moxifloxacin in Treatment of Patients With Community-Acquired Bacterial Pneumonia

SOLITAIRE-ORAL
Start date: December 2012
Phase: Phase 3
Study type: Interventional

This study will evaluate the safety and efficacy of an experimental antibiotic, solithromycin, in the treatment of adult patients with community-acquired pneumonia.

NCT ID: NCT01755767 Completed - Clinical trials for Hepatocellular Carcinoma

Study of Tivantinib in Subjects With Inoperable Hepatocellular Carcinoma (HCC) Who Have Been Treated With One Prior Therapy

METIV-HCC
Start date: December 27, 2012
Phase: Phase 3
Study type: Interventional

The purpose of this study is to determine if tivantinib (ARQ 197) is effective in treating patients with MET diagnostic-high hepatocellular carcinoma (liver cancer) who have already been treated once with another therapy.

NCT ID: NCT01753804 Terminated - Clinical trials for Duchenne Muscular Dystrophy

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Start date: September 1, 2012
Phase: N/A
Study type: Observational

To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.

NCT ID: NCT01750190 Completed - CKD Anemia Clinical Trials

A Study of Roxadustat for the Treatment of Anemia in Participants With Chronic Kidney Disease and Not Receiving Dialysis

Start date: November 5, 2012
Phase: Phase 3
Study type: Interventional

The purpose of this study is to determine whether roxadustat is safe and effective in the treatment of anemia in participants with chronic kidney disease and not on dialysis.

NCT ID: NCT01747538 Terminated - Uveitis Clinical Trials

Safety and Efficacy Study of Gevokizumab to Treat Non-infectious Uveitis Controlled With Systemic Treatment

EYEGUARD™-C
Start date: October 2012
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy of gevokizumab in reducing the risk of recurrent uveitic disease in subjects with non-infectious uveitis whose disease is currently controlled with systemic treatment.

NCT ID: NCT01744405 Completed - Chagas Disease Clinical Trials

Study Of Nifurtimox Transfer Into Breastmilk In Lactating Women With Chagas Disease

LACTNFX
Start date: December 2012
Phase: N/A
Study type: Observational

The investigators propose to study the transfer of nifurtimox into breastmilk of lactating women who receive the drug for the treatment of Chagas disease. Breastmilk and blood samples will be obtained from these patients at pre-specified times after they take the clinically indicated medication, and the concentrations in both matrices will be compared to estimate degree of transfer. Estimation of nifurtimox transfer into breastmilk will allow the evaluation of potential degree of exposure of infants breastfed by these women to nifurtimox. This study will help clarify safety of continuing breastfeeding while receiving treatment with nifurtimox for Chagas disease.

NCT ID: NCT01739829 Completed - Type 1 Diabetes Clinical Trials

Open-label Investigation of the Safety and Effectiveness of DIABECELL® in Patients With Type 1 Diabetes Mellitus

Start date: August 2011
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to establish the safety and efficacy of xenotransplantation of DIABECELL® in patients with established type 1 diabetes mellitus

NCT ID: NCT01737398 Completed - Amyloidosis Clinical Trials

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

Start date: March 15, 2013
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of inotersen given for 65 weeks in participants with Familial Amyloid Polyneuropathy (FAP).

NCT ID: NCT01737359 Terminated - Clinical trials for Human Immunodeficiency Virus Infection

A Safety and Efficacy Study of Amdoxovir in HIV-1 Treatment-experienced Subjects.

Start date: December 2012
Phase: Phase 2
Study type: Interventional

This is a double-blind Phase 2a study to test the safety and efficacy of an investigational HIV drug, amdoxovir (300 mg or 500 mg twice daily) compared with tenofovir DF 300 mg once daily in HIV-1 infected antiretroviral therapy-experienced subjects who are currently failing antiretroviral therapy. There are three treatment groups (N=45). Subjects will be randomized to receive either amdoxovir 300 mg twice daily (n=15) or amdoxovir 500 mg twice daily (n=15) or tenofovir DF 300 mg once daily (n=15); each in combination with zidovudine 300 mg twice daily. The study will assess initially amdoxovir (300 mg or 500 mg twice daily) or tenofovir DF 300 mg once daily, both in combination zidovudine 300 mg twice daily plus failing third drug, but then with lopinavir/ritonavir (400 mg/100 mg twice daily) after Week 2. Subjects who received amdoxovir (300 mg or 500 mg twice daily) and benefited from the drug may choose to enroll in the 36-week open-label study.