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NCT ID: NCT03036488 Active, not recruiting - Clinical trials for Triple Negative Breast Neoplasms

Study of Pembrolizumab (MK-3475) Plus Chemotherapy vs Placebo Plus Chemotherapy as Neoadjuvant Therapy and Pembrolizumab vs Placebo as Adjuvant Therapy in Participants With Triple Negative Breast Cancer (TNBC) (MK-3475-522/KEYNOTE-522)

Start date: March 7, 2017
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of pembrolizumab (MK-3475) plus chemotherapy vs placebo plus chemotherapy as neoadjuvant therapy and pembrolizumab vs placebo as adjuvant therapy in participants who have triple negative breast cancer (TNBC). After a screening phase of approximately 28 days, each participant will receive neoadjuvant study treatment (Pembrolizumab + Chemotherapy OR Placebo + Chemotherapy) based on the randomization schedule for approximately 24 weeks (8 cycles). Each participant will then undergo definitive surgery 3-6 weeks after conclusion of the last cycle of the neoadjuvant study treatment. After definitive surgery, each participant will receive adjuvant study treatment (Pembrolizumab OR Placebo) for approximately 27 weeks (9 cycles). Following adjuvant study treatment, each participant will be monitored for safety, survival and disease recurrence. The primary study hypothesis is that pembrolizumab is superior to placebo, in combination with chemotherapy, as measured by the rate of Pathological Complete Response (pCR) and/or Event-free Survival (EFS), in participants with locally advanced TNBC.

NCT ID: NCT03036280 Active, not recruiting - Alzheimer's Disease Clinical Trials

A 24-Month Study to Evaluate the Efficacy and Safety of Elenbecestat (E2609) in Subjects With Early Alzheimer's Disease_

MissionAD2
Start date: December 29, 2016
Phase: Phase 3
Study type: Interventional

The name of this trial is MissionAD2. This phase 3 study consists of a Core and Open Label Extension (OLE) Phase in participants with Early Alzheimer's Disease (EAD), and will be conducted to evaluate the efficacy and safety of E2609. The Core is a 24-month treatment, multicenter, double blind, placebo controlled parallel group study. The OLE is a 24-month treatment, one group study. The data for end points for the studies E2609-G000-301 (NCT02956486) and E2609-G000-302 (NCT03036280) will be pooled for a combined total of 1900 participants.

NCT ID: NCT03034811 Active, not recruiting - Osteoarthritis Clinical Trials

Persona Partial Knee Clinical Outcomes Study

Start date: February 8, 2017
Phase: N/A
Study type: Interventional

The primary objective of this study is to obtain implant survivorship and clinical outcomes data for the commercially available Persona Partial Knee System.

NCT ID: NCT03032172 Active, not recruiting - Clinical trials for Spinal Muscular Atrophy

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Jewelfish
Start date: March 3, 2017
Phase: Phase 2
Study type: Interventional

This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.

NCT ID: NCT03030053 Active, not recruiting - Mental Processes Clinical Trials

The Effects of Flavonoid Supplementation on Cognition and Neural Mechanisms in Healthy Older Adults

CoCo_Chronic
Start date: February 2016
Phase: N/A
Study type: Interventional

A double-blind, randomised, controlled, parallel arm chronic intervention trial with healthy older adults will be conducted to determine the effect of a flavonoid-rich supplement on cognitive function, peripheral arterial health and brain mechanisms. It is predicted that chronic flavonoid supplementation will result in cognitive benefits and that these may be due to beneficial effects of flavonoids on vascular and brain function.

NCT ID: NCT03025763 Active, not recruiting - Craniosynostosis Clinical Trials

Network Of Clinical Research Studies On Craniosynostosis, Skull Malformations With Premature Fusion Of Skull Bones

Start date: January 13, 2015
Phase:
Study type: Observational

Craniosynostosis (CS) is a common malformation occurring in ~4 per 10,000 live births in which the sutures between skull bones close too early, causing long-term problems with brain and skull growth. Infants with CS typically require extensive surgical treatment and may experience many perioperative complications, including hemorrhage and re-synostosis. Even with successful surgery, children can experience developmental and learning disabilities or vision problems. Most often, CS appears as isolated nonsyndromic CS (NSC). Of the several subtypes of CS, unilateral or bilateral fusion of the coronal suture is the second most common form of CS accounting for 20-30% of all NSC cases. The etiology of coronal NSC (cNSC) is not well understood, although the published literature suggests that it is a multifactorial condition. About 5-14% of coronal craniosynostosis patients have a positive family history, with a specific genetic etiology identified in >25% of cNSC cases, suggesting a strong genetic component in the pathogenesis of this birth defect. The causes for cNSC and its phenotypic heterogeneity remain largely unknown. An international team of investigators will generate large genomic and gene expression datasets on samples from patients with cNSC. State-of-the-art imaging, genetic, and developmental and systems biology approaches will be used to quantitatively model novel pathways and networks involved in the development of cNSC. Novel variant-, gene- and network-level analyses will be performed on the genomic data obtained from cNSC cases, their relatives, and controls to identify novel variants and genetic regions associated with cNCS. Quantitative, analytical, and functional validations of these predictions will provide insights into the etiology and possible therapeutic targets for CS and potentially other bone-related disorders.

NCT ID: NCT03025698 Active, not recruiting - Aplastic Anemia Clinical Trials

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

Start date: September 30, 2017
Phase: Phase 2
Study type: Interventional

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.

NCT ID: NCT03025308 Active, not recruiting - Clinical trials for Rheumatoid Arthritis

Long Term Extension Study to Assess the Safety and Efficacy of Filgotinib in Adults With Rheumatoid Arthritis

FINCH 4
Start date: February 28, 2017
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to evaluate the long-term safety and tolerability of filgotinib in participants who have completed one of the parent studies of filgotinib in rheumatoid arthritis (RA).

NCT ID: NCT03023540 Active, not recruiting - Clinical trials for Charcot-Marie-Tooth Disease, Type IA

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

PLEO-CMT-FU
Start date: March 7, 2017
Phase: Phase 3
Study type: Interventional

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03. Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL). Period 2: All patients continue on twice dose 1 (2X5mL).

NCT ID: NCT03019406 Active, not recruiting - Clinical trials for Glycogen Storage Disease Type II-Pompe's Disease

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa

Mini-COMET
Start date: October 12, 2017
Phase: Phase 2
Study type: Interventional

Primary Objective: To evaluate the safety profile of avalglucosidase alfa in participants with infantile-onset Pompe disease previously treated with alglucosidase alfa. Secondary Objective: To characterize the pharmacokinetic profile of avalglucosidase alfa and to evaluate the preliminary efficacy of avalglucosidase alfa in comparison to alglucosidase alfa.