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NCT ID: NCT01924845 Terminated - Clinical trials for Late-onset Pompe Disease

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Start date: April 2014
Phase: Phase 3
Study type: Interventional

Study 701-301 is a single-arm, open-label, switchover study in patients with late-onset Pompe disease who have been receiving treatment with recombinant human acid alpha-glucosidase (rhGAA) for 48 weeks or longer. Ambulatory patients who have mild to moderate respiratory impairment will switch directly to receive BMN 701 20 mg/kg by IV infusion every other week. All participants will receive active drug. No dose of existing therapy will be missed - experimental drug is started immediately.

NCT ID: NCT01923311 Terminated - HIV Infections Clinical Trials

Two Part Study to Evaluate Pharmacokinetics, Safety, and Antiviral Activity of Elvitegravir Administered With a PI/r Background Regimen for ARV Treatment-Experienced Pediatric Participants

Start date: August 26, 2013
Phase: Phase 2/Phase 3
Study type: Interventional

The primary objectives of this study are to evaluate the safety, tolerability and steady-state PK and confirm the dose of EVG/r in HIV-1 infected, antiretroviral treatment-experienced children 4 weeks to <18 years of age. The study consists of 2 parts: Part A and Part B. Part A will enroll participants with suppressed viremia (HIV-1 RNA < 50 copies/mL) or failing a current antiretroviral (ARV) regimen (HIV-1 RNA > 1,000 copies/mL only for participants in Cohort 2, Part A) to evaluate the steady state PK and confirm the dose of EVG. Part B will enroll participants who are failing a current ARV regimen (HIV-1 RNA > 1,000 copies/mL) to evaluate the safety, tolerability, and antiviral activity of EVG. The study consists of 4 age cohorts with each cohort including 2 parts (Part A and Part B) with the exception of the adolescent age cohort (Cohort 1: 12 to < 18 years old) containing Part B only.

NCT ID: NCT01923142 Terminated - Vitiligo Clinical Trials

Efficacy of Autologous Outer-Root-Sheath Melanocytes Transplantation in the Treatment of Vitiligo

Start date: January 2014
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the hypothesis that, in patients with stable vitiligo unresponsive to conventional treatments including traditional phototherapy and/or topical steroids, the application of a non-cultured autologous hair follicle outer-root-sheath melanocytes suspension in the area affected by the disease followed by targeted ultraviolet B (UVB) phototherapy can lead to a significant skin repigmentation. To assess the effect of the proposed treatment, a within-subject controlled study involving selected symmetric lesion areas localized to the back of the hands will be conducted.

NCT ID: NCT01920477 Terminated - Pemphigus Vulgaris Clinical Trials

Efficacy and Safety of Ofatumumab in Treatment of Pemphigus Vulgaris

Start date: August 13, 2013
Phase: Phase 3
Study type: Interventional

Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters. Ofatumumab is a novel monoclonal antibody (mAb) that specifically binds to the human CD20 antigen, which is expressed only in B lymphocytes. The purpose of this study was to evaluate the efficacy, tolerability, and safety of ofatumumab injection for subcutaneous use (ofatumumab SC) 20 milligrams (mg) administered once in every 4 weeks, (with an additional 20 mg loading dose [i.e. 40 mg total] at both Week 0 and Week 4) in subjects with PV. It was anticipated that with sustained B-cell depletion in the presence of ofatumumab SC, and the resultant reduction of pathogenic anti Dsg (desmoglein) autoantibodies in PV, that clinical remission of the disease would result.

NCT ID: NCT01915303 Terminated - Cushings Disease Clinical Trials

Study of the Efficacy and Safety of Pasireotide s.c. +/- Cabergoline in Patients With Cushing's Disease

Start date: March 6, 2014
Phase: Phase 2
Study type: Interventional

The main purpose of this prospective, multicenter, open-label phase II study, was to evaluate the efficacy and safety of pasireotide alone or in combination with cabergoline in patients with Cushing's disease.

NCT ID: NCT01911325 Terminated - Clinical trials for Squamous Non-small Cell Lung Cancer

Phase II Study of Buparlisib + Docetaxel in Advanced or Metastatic Squamous Non-small Cell Lung Cancer (NSCLC) Patients

Start date: October 2013
Phase: Phase 1
Study type: Interventional

This is a multi-center, open-label Phase Ib dose escalation part followed by a randomized double-blinded placebo controlled Phase II part. The Phase Ib part will determine the Maximum Tolerated Dose (MTD)/Recommended Phase II Dose (RP2D) of buparlisib in combination with docetaxel. Subsequently the MTD/RP2D will be investigated in a Phase II randomized trial in patients with advanced or metastatic squamous NSCLC.

NCT ID: NCT01907100 Terminated - Mesothelioma Clinical Trials

Nintedanib (BIBF 1120) in Mesothelioma

Start date: September 19, 2013
Phase: Phase 2/Phase 3
Study type: Interventional

This is a phase II/III confirmatory study designed to evaluate the safety and efficacy of nintedanib (BIBF 1120) in combination + (pemetrexed / cisplatin) followed by nintedanib (BIBF 1120) versus placebo + pemetrexed / cisplatin followed by placebo for the treatment of patients with unresectable malignant pleural mesothelioma.

NCT ID: NCT01905592 Terminated - Ovarian Neoplasms Clinical Trials

A Phase III Trial of Niraparib Versus Physician's Choice in HER2 Negative, Germline BRCA Mutation-positive Breast Cancer Patients

BRAVO
Start date: February 25, 2014
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare progression-free survival (PFS) in patients with advanced/metastatic breast cancer who have a BRCA mutation when treated with niraparib as compared to those treated with physician's choice

NCT ID: NCT01904253 Terminated - Clinical trials for Small Cell Lung Cancer (SCLC)

A Phase 2 Study Comparing TAS-102 Versus Topotecan or Amrubicin to Treat Small Cell Lung Cancer Following Platinum-Based Chemotherapy

Start date: July 2013
Phase: Phase 2
Study type: Interventional

The purpose of this trial is to compare the effects of TAS-102 with either amrubicin or topotecan (drugs used in Small Cell Lung Cancer) on lung cancer to find out the effects on survival, how much time may pass without disease progression, and the safety of TAS-102.

NCT ID: NCT01902290 Terminated - Asthma Clinical Trials

Study of Efficacy and Safety of Brodalumab Compared With Placebo in Adults With Inadequately Controlled Asthma With High Bronchodilator Reversibility

Start date: May 22, 2013
Phase: Phase 2
Study type: Interventional

The purpose of this study is to determine if brodalumab (AMG 827) is safe and effective compared to placebo as measured by change in Asthma Control Questionnaire (ACQ) composite scores.