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NCT ID: NCT02215616 Completed - Clinical trials for Huntington's Disease

A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod

LEGATO-HD
Start date: October 28, 2014
Phase: Phase 2
Study type: Interventional

The primary objective of this study is to assess the efficacy of laquinimod as treatment in participants with HD after 52 weeks using the Unified Huntington's Disease Rating Scale Total Motor Score (UHDRS-TMS or TMS).

NCT ID: NCT02215499 Completed - Clinical trials for Narcolepsy, Excessive Daytime Sleepiness

A Phase 1, Single Dose Study of JZP-386 to Evaluate Safety, Pharmacokinetics and Pharmacodynamics

Start date: July 2014
Phase: Phase 1
Study type: Interventional

This study is being conducted to evaluate the safety, tolerability, blood distribution and effectiveness single ascending doses of JZP-386 compared to doses of Xyrem® and placebo.

NCT ID: NCT02215096 Completed - Neoplasms Clinical Trials

Dose-finding Study of GSK2636771 When Administered in Combination With Enzalutamide in Male Subjects With Metastatic Castration-Resistant Prostate Cancer

Start date: November 13, 2014
Phase: Phase 1
Study type: Interventional

This Phase I, open-label, dose-finding, multicenter study is designed to determine the recommended Phase II dose (RP2D) for the combination of an orally administered Phosphatidylinositol-4,5-bisphosphate 3-kinase beta (PI3K-beta) inhibitor (GSK2636771) with enzalutamide. Subjects with phosphatase and tensin homolog (PTEN)-deficient metastatic castration-resistant prostate cancer (mCRPC) who are receiving a stable dose of enzalutamide with a recently demonstrated progression (either by RECIST [Response Evaluation Criteria In Solid Tumors] version 1.1, prostate-specific antigen [PSA] progression, and/or progression in bone) per the Prostate Cancer Working Group 2 (PCWG2) criteria will be enrolled. Eligible subjects will be enrolled in the Dose-Escalation Phase to determine the maximum tolerated dose (MTD) of the combination therapy using a modified 3+3 dose escalation procedure. The safety, pharmacokinetics (PK) and clinical efficacy will also be assessed to guide the selection of the RP2D. The starting dose will be GSK2636771 300 mg once daily in combination with the recommended dose (160 milligram [mg] once daily) of oral enzalutamide. Once the RP2D has been established, additional subjects will be enrolled in the Dose Expansion Phase to further evaluate the safety, PK and preliminary clinical activity. Safety assessments will be performed throughout the study including physical examinations, vital signs, clinical laboratory tests, 12 lead electrocardiograms and monitoring of adverse events. Blood samples will be collected for pharmacokinetic analysis. Subjects will continue treatment until an unacceptable toxicity, disease progression, withdrawal of consent or death occurs. A post-treatment follow-up visit will be performed within 30 days of the last dose of study treatment. Xtandi is a registered trademark of Astellas Pharma Inc

NCT ID: NCT02214160 Completed - Clinical trials for Trifunctional Protein (TFP) Deficiency

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Start date: December 9, 2014
Phase: Phase 2
Study type: Interventional

The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.

NCT ID: NCT02214134 Completed - Clinical trials for Any Stage of Lung Cancer (Any Histotype)

SPECTAlung: Screening Patients With Thoracic Tumors for Efficient Clinical Trial Access

SPECTAlung
Start date: May 22, 2015
Phase:
Study type: Observational [Patient Registry]

SPECTAlung is a program aiming at screening patients with thoracic tumors to identify the molecular characteristics of their disease. The thoracic tumors include lung cancer, malignant pleural mesothelioma, thymoma or thymic carcinoma at any stage. Once the molecular characteristics are identified, there might be the possibility to offer these patients access to targeted clinical trials.

NCT ID: NCT02214121 Completed - Clinical trials for Investigation of Platelet Aggregation in Paediatric Patients With Sickle Cell Disease

A Pharmacokinetic (PK) and Pharmacodynamic (PD) Dose-ranging Phase II Study of Ticagrelor in Paediatric Patients With Sickle Cell Disease

HESTIA 1
Start date: September 11, 2014
Phase: Phase 2
Study type: Interventional

The purpose of this Phase II dose-ranging study is to investigate pharmacokinetic (PK) and pharmacodynamic (PD) properties of various doses of ticagrelor followed by 4 weeks of twice-daily treatment in paediatric patients with sickle cell disease

NCT ID: NCT02213315 Completed - Healthy Volunteers Clinical Trials

A Double-blind, Placebo-controlled, Single-dose Study to Evaluate the PK, IM, and Safety in Japanese Subjects

Start date: July 2014
Phase: Phase 1
Study type: Interventional

A phase 1 randomized, double blind single-dose study to evaluate the PK and immunogenicity of single SC 100 and 150 mg doses of mavrilimumab in healthy adult Japanese subjects.

NCT ID: NCT02213263 Completed - Follicular Lymphoma Clinical Trials

A Study Of PF-05280586 (Rituximab-Pfizer) Or MabThera® (Rituximab-EU) For The First-Line Treatment Of Patients With CD20-Positive, Low Tumor Burden, Follicular Lymphoma (REFLECTIONS B328-06)

Start date: September 30, 2014
Phase: Phase 3
Study type: Interventional

This study will compare the safety and effectiveness of PF-05280586 versus rituximab-EU in patients with CD20-positive, low tumor burden follicular lymphoma. The primary hypothesis to be tested in this study is that the effectiveness of PF-05280586, as measured by the Overall Response Rate, is similar to that of rituximab-EU.

NCT ID: NCT02212444 Completed - Plantar Fasciitis Clinical Trials

Comparing the Effects of Two Foot and Ankle Splints for Foot Pain

Start date: June 2014
Phase: N/A
Study type: Interventional

Plantar fasciitis (severe pain in the heel) is a common problem that has a significant impact on quality of life. There is some evidence to support the use of orthoses and stretches in the conservative management of plantar fasciitis but current orthotic management may not be optimal. More prolonged stretching with night splints may achieve better results but such splints are clinically not well tolerated. Therefore, there is a potential need to apply prolonged stretching during the day and during dynamic tasks such as walking. This has led to the development of other rigid and semi-rigid splints that have shown promising results in small scale clinical trials. There are disadvantages however with the more rigid bracing and orthoses seen in these types of splints. For example, they are often difficult to accommodate with a person's available shoes; this can be particularly difficult for women and thus limits their compliance with the intervention. Further, more rigid bracing can be uncomfortable during fast walking and running and so limits participation in such activities. Recently a novel orthotic, a customised dynamic elastomeric fabric orthoses (DEFO), has been developed. Being made from lycra® based materials the sock-like splint is lightweight and discrete, allowing it to be accommodated easily into most shoe types and potentially better tolerated when worn at night compared to currently available splints. Its design further allows it to be used comfortably during dynamic tasks such as walking and running as supported by initial anecdotal evidence in athletes. To date there has been no evaluation into the effectiveness of the DEFO in the general population with plantar fasciitis. This study will look at the feasibility of conducting a randomised controlled trial into the use of a DEFO compared to an off-the-shelf orthoses as an adjunct to usual care.

NCT ID: NCT02212431 Completed - Cystic Fibrosis Clinical Trials

First Study of Oral Cysteamine in Cystic Fibrosis

Start date: August 2014
Phase: Phase 1/Phase 2
Study type: Interventional

The morbidity and mortality associated with Cystic Fibrosis (CF) are the result of chronic suppurative lung disease. The aggressive use of antibiotics is one of the mainstays of treatment in CF, however, the problems of multiple drug resistance and adverse reactions are major clinical issues. Cysteamine is a licensed drug used in the treatment of cystinosis. In vitro work suggests that cysteamine has properties of potential benefit in CF. Cysteamine is a potent mucolytic, it disrupts biofilms, it is antimicrobial, and synergises with other antibiotic agents. CF is characterised by malabsorption and it is not known whether cysteamine is absorbed in CF, furthermore it is not known if cysteamine enters the bronchial secretions. It is not possible to assume that the pharmacokinetics of cysteamine in patients with CF are the same as those reported for cystinosis. Objectives: to characterise the pharmacokinetic profile of cysteamine in people with CF, to ascertain whether cysteamine enters the bronchial secretions and the tolerability of cysteamine by patients with CF. Method: a single centre, single group open label investigation of the tolerability and pharmacokinetics of oral cysteamine (Cystagon) when administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. Setting: adult CF clinic, Aberdeen Royal Infirmary. Target population: 12 patients aged ≥18years with CF associated lung disease who are clinically stable. Intervention: Oral cysteamine (Cystagon) will be increased from 450mg od to 450mg qds over three weeks, they will remain on 450mg qds for two weeks. Assessment: face to face health outcome assessments will be carried out for all participants at recruitment/baseline, 1, 2, 3, and 5 and 6 weeks. Serial blood cysteamine levels will be measured in the first 24 hours after the first dose. Sputum cysteamine will be quantified after two weeks of full dose cysteamine 450mg qds. Disease specific health status (CFQ-R) will be assessed at baseline and after two weeks of full dose. At each assessment, lung function (FEV1, FVC), adverse reactions and serious adverse events will be ascertained. Blood samples will be taken for measurement of haematological and biochemical parameters. Sputum samples at each assessment will be analysed for microbial load and spinnbarkeit.