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NCT ID: NCT02381457 Completed - Clinical trials for Prader-Willi Syndrome

SNP-based Microdeletion and Aneuploidy RegisTry (SMART)

SMART
Start date: April 2015
Phase:
Study type: Observational [Patient Registry]

This multi-center prospective observational study is designed to track birth outcomes and perinatal correlates to the Panorama prenatal screening test in the general population among ten thousand women who present clinically and elect Panorama microdeletion and aneuploidy screening as part of their routine care. The primary objective is to evaluate the performance of Single Nucleotide Polymorphism (SNP)-based Non Invasive Prenatal Testing (NIPT) for 22q11.2 microdeletion (DiGeorge syndrome) in this large cohort of pregnant women. This will be done by performing a review of perinatal medical records and obtaining biospecimens after birth to perform genetic diagnostic testing for 22q11.2 deletion. Results from the follow-up specimens will be compared to those obtained by the Panorama screening test to determine test performance. Specific test performance parameters will include: PPV, specificity, and sensitivity.

NCT ID: NCT02381418 Completed - Influenza Clinical Trials

Annual Study to Investigate Inactivated Subunit Influenza Vaccine Due to New Virus Strains for the Southern Hemisphere 2015 Season

Start date: March 2015
Phase: Phase 3
Study type: Interventional

Influenza (flu) viruses change continuously, therefore also the parts of viruses used in influenza vaccines can vary from year to year. The current study is a phase IIIa clinical trial with a commercially available vaccine (Influvac®) supplied in pre filled syringes. The objective of this study is to investigate the immunogenicity and safety and tolerability of the changed influenza vaccine virus composition in two groups of subjects in good health: subjects aged >= 18 and <= 60 years and subjects >= 61 years of age (elderly).

NCT ID: NCT02379845 Completed - Clinical trials for Adult Soft Tissue Sarcoma

NBTXR3 Crystalline Nanoparticles and Radiation Therapy in Treating Randomized Patients in Two Arms With Soft Tissue Sarcoma of the Extremity and Trunk Wall

Start date: March 3, 2015
Phase: Phase 2/Phase 3
Study type: Interventional

RATIONALE: Radiation therapy given before surgery of soft tissue sarcoma decreases the size of the tumor mass and the presence of malignant cells in its peripheral region. NBTXR3 and radiation therapy may kill more cancer cells and increase the tumor shrinkage rendering surgery more feasible or easier and achieve better local control of the tumor. PURPOSE: This phase II/III is a prospective randomized, multi-center, open-label and active controlled two arms study in patients with locally advanced soft tissue sarcoma (STS) of the extremity and trunk wall. Patients will be randomized to receive either NBTXR3 as intratumor injection, activated by external beam radiation therapy or external beam radiation therapy alone, as preoperative treatment. Once the radiotherapy treatment is completed, tumor surgery will be performed in all patients.

NCT ID: NCT02377830 Completed - Respiratory Failure Clinical Trials

CYCLE Pilot Randomized Trial

CYCLE Pilot
Start date: March 2015
Phase: Phase 2
Study type: Interventional

Background: Patients in the intensive care unit (ICU) are the sickest in hospital, and need advanced life-support. Survivors of critical illness are very weak and disabled. Up to 1 in 4 have severe leg weakness impairing their quality of life for as long as 5 years after ICU discharge. In-bed cycling involves use of special equipment that attaches to a patient's hospital bed, allowing them gentle exercise while in the ICU. Methods: Adult patients admitted to the ICU who need a breathing machine and are expected to survive their ICU stay are eligible. Patients will randomly receive 30 minutes of in-bed cycling each day they are in the ICU or routine physiotherapy, both delivered by specially trained physiotherapists. Outcomes: Feasibility: The investigators will study whether patients can cycle on most days of their ICU stay, whether patients and their families agree to be a part of the study, and whether investigators can systematically assess patients' strength. Relevance: Effective methods of physiotherapy are needed for critically ill patients to minimize muscle weakness, speed recovery, and improve quality of life. This pilot randomized study is the second of several future larger studies about in-bed cycling in the ICU. Our pilot work includes CYCLE Pilot and CYCLE Vanguard. CYCLE Pilot is an external pilot and enrolled 66 patients from 3/2015 to 6/2016. CYCLE Vanguard is an internal pilot and enrolled 47 patients from 11/2016 to 3/2018. CYCLE Vanguard patients will be analyzed in the main CYCLE RCT (NCT03471247).

NCT ID: NCT02377349 Completed - Clinical trials for Diphtheria-Tetanus-acellular Pertussis Vaccines

Immunogenicity and Safety Study of GlaxoSmithKline (GSK) Biologicals' Boostrix™ Vaccine in Pregnant Women

Start date: October 14, 2015
Phase: Phase 4
Study type: Interventional

The purpose of this study is to assess the immunogenicity and safety of Boostrix™ when compared to a placebo given during 27-36 weeks of gestation in healthy women aged 18-45 years. Infants born to mothers enrolled in this study will be followed-up in two separate clinical studies: 201330 [DTPA (BOOSTRIX)-048 PRI] and 201334 [DTPA (BOOSTRIX)-049 BST: 048].

NCT ID: NCT02374060 Completed - Macular Edema Clinical Trials

PeriOcular and INTravitreal Corticosteroids for Uveitic Macular Edema Trial

POINT
Start date: June 16, 2015
Phase: Phase 3
Study type: Interventional

To evaluate the relative efficacy of three commonly utilized regional corticosteroids for the regional treatment of uveitic macular edema: periocular triamcinolone acetonide; intravitreal triamcinolone acetonide; intravitreal dexamethasone implant. The primary efficacy measure will be percent change in central subfield thickness as measured by OCT at 8 weeks. Participants will continue in the study for 24 weeks in order to evaluate relative effects of the 3 treatment strategies on the duration of treatment effects, requirement for additional injections, and adverse effects. Note: The planned sample size for the POINT Trial was 267 subjects. On 17 July 2017, with 192 subjects enrolled, the Data and Safety Monitoring Committee (DSMC) reviewed the planned interim analysis and recommended that the goals of the trial could be accomplished by completing follow-up of enrolled subjects without the recruitment of additional subjects. Per the DSMC recommendations, recruitment was suspended and follow-up of enrolled subjects was completed according to the protocol.

NCT ID: NCT02372006 Completed - Rhabdomyosarcoma Clinical Trials

Trial of Afatinib in Pediatric Tumours

Start date: April 29, 2015
Phase: Phase 1/Phase 2
Study type: Interventional

Open-label, dose escalation, monotherapy, basket trial with biomarker specific MTD expansion cohort/Phase II part. The trial will consist of 2 parts: 1. Dose finding part to determine the MTD 2. Biomarker specific MTD expansion cohort/Phase II part to assess clinical anti-tumour activity in included tumour types

NCT ID: NCT02371369 Completed - Clinical trials for Tenosynovial Giant Cell Tumor

Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

ENLIVEN
Start date: May 11, 2015
Phase: Phase 3
Study type: Interventional

This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good. The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS). The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2. Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.

NCT ID: NCT02370706 Completed - Myelofibrosis Clinical Trials

Study of the Safety of PIM447 in Combination With Ruxolitinib (INC424) and LEE011 in Patients With Myelofibrosis

Start date: May 21, 2015
Phase: Phase 1
Study type: Interventional

This is a phase Ib study with the primary purpose is to estimate the MTD and/or RDE for the triple combination of PIM447, formerly LGH447, plus ruxolitinib and LEE011 as well as for the doublets, PIM447 plus ruxolitinib, and LEE011 plus ruxolitinib, in patients with myelofibrosis (MF). Each regimen will be assessed for safety, tolerability, pharmacokinetics (PK) and pharmacodynamic effects, and preliminary anti-myelofibrosis activity, including changes in spleen volume, JAK2V617F allele burden, and hematologic response.

NCT ID: NCT02370589 Completed - Ebola Clinical Trials

Study to Evaluate the Immunogenicity and Safety of an Ebola Virus (EBOV) Glycoprotein (GP) Vaccine in Healthy Subjects

Start date: February 2015
Phase: Phase 1
Study type: Interventional

This is a randomized, observer-blind, placebo-controlled trial in male and female subjects ≥18 to <50 years of age. Subjects will be healthy adults based on history, physical examination, and baseline clinical laboratory testing. Approximately 230 eligible subjects will be enrolled into 1 of 13 treatment groups. Treatments will comprise two IM doses at a 21-day interval (Day 0 and Day 21), in alternate deltoids with the test article assigned (i.e., saline placebo, dose of EBOV GP vaccine with or without Matrix-M adjuvant), in a 0.5mL injection volume.