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NCT ID: NCT02333760 Active, not recruiting - Clinical trials for Wiskott-Aldrich Syndrome

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

WASFUP
Start date: September 2014
Phase: Phase 1/Phase 2
Study type: Interventional

An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.

NCT ID: NCT02326337 Active, not recruiting - Diabetic Foot Ulcer Clinical Trials

Efficacy, Safety and Economic Benefits of Topical Wound Oxygen Therapy in the Treatment of Chronic Diabetic Foot Ulcers

TWO2DFU
Start date: October 2014
Phase: N/A
Study type: Interventional

The purpose of this study is to evaluate the efficacy, safety and economical benefits of Cyclical Pressure Topical Wound Oxygen (TWO2) Therapy in the treatment of chronic diabetic foot ulcers. Subjects will wear a standardized off-loading device and use advanced moist wound therapy (AMWT) dressings. Following a 2 week run-in period with the standardized care and after meeting all the eligibility criteria, half the subjects will use the TWO2 device, while the other half will use a sham device.

NCT ID: NCT02322840 Active, not recruiting - Clinical trials for Mitral Valve Insufficiency

Transcatheter Mitral Valve Replacement System (TMVR) Pilot Study and The Early Feasibility Study of the TMVR Transseptal System

Start date: April 2015
Phase: N/A
Study type: Interventional

PILOT: The study is pilot trial to evaluate the safety and performance of the Twelve TMVR System in very high risk mitral regurgitation patients. EFS: The study is an EFS trial to evaluate the safety and performance of the Medtronic Intrepid™ Transcatheter Mitral Valve Replacement System with Transfemoral Transseptal access in Patients with Severe, Symptomatic Mitral Regurgitation

NCT ID: NCT02321514 Active, not recruiting - Clinical trials for Mitral Valve Regurgitation

Expanded Clinical Study of the Tendyne Mitral Valve System

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

The purpose of this study is to evaluate the safety and performance of the Tendyne Mitral Valve System in the treatment of severe mitral regurgitation in patents with functional disability greater than or equal to NYHA Class II, who are not suitable candidates for surgical replacement with otherwise available devices. Follow-up evaluations will be conducted through 5 years post implantation.

NCT ID: NCT02320435 Active, not recruiting - Solid Tumor Clinical Trials

A Safety and Efficacy Extension Study of Pertuzumab in Patients With Solid Tumors Previously Enrolled in a Hoffmann-La Roche-Sponsored Pertuzumab Clinical Trial

Start date: February 2, 2015
Phase: Phase 3
Study type: Interventional

This is a single-arm, multi-center, open-label extension study designed to provide continued pertuzumab therapy to patients receiving pertuzumab as an investigational medicinal product (IMP) in a Roche-sponsored global study and who continue to receive pertuzumab at the end of the Parent study, as well as to collect long-term safety and efficacy data of pertuzumab therapy. Patients with solid tumors who have not experienced progressive disease in the Parent study and, in the investigator's opinion, may potentially benefit from continued pertuzumab treatment, will continue to receive pertuzumab until disease progression, unacceptable toxicity, investigator/patient decision, patient non-compliance, patient death, patient request to withdraw, or study termination by the Sponsor, whichever occurs first.

NCT ID: NCT02319837 Active, not recruiting - Prostate Cancer Clinical Trials

Safety and Efficacy Study of Enzalutamide Plus Leuprolide in Patients With Nonmetastatic Prostate Cancer (EMBARK)

Start date: December 17, 2014
Phase: Phase 3
Study type: Interventional

The purpose of this study is to assess enzalutamide plus leuprolide in patients with high-risk nonmetastatic prostate cancer progressing after radical prostatectomy or radiotherapy or both. The randomized / blinded portion of the study is now completed following primary endpoint analyses. The study remains ongoing in open label format.

NCT ID: NCT02319226 Active, not recruiting - Clinical trials for Graft vs Host Disease

Toward Immune Biomarkers for Tolerance and GvHD in Humans

BioGvHD
Start date: May 2014
Phase:
Study type: Observational

Graft-versus-Host Disease (GVHD), is the most frequent and severe complication of allogeneic hematopoietic stem cell transplantation (HSCT). Much of our knowledge on the pathophysiology of GVHD has been gained from experimental models but far less from the study of the disease in humans. Recent developments in basic biology open new avenues to the development of biomarker sets that could predict GVHD severity and prognosis that could be tested and validated through well-designed multicenter clinical trials. The main goal of this project is to further our understanding of the pathogenic mechanisms of human GVHD on one hand, and of functional immune tolerance on the other. Furthermore, this study aims at setting up a clinically relevant biomarker set in human GVHD and immune tolerance in a discovery cohort. The objectives of this project are: 1. To define phenotypic, functional and molecular correlates of acute GVHD early after HSCT/at its onset 2. To study thymic reconstitution and the T-cell repertoire after HSCT during period 2 3. To identify functional and molecular correlates of immune tolerance in long-term survivors of HSCT 4. Preparing for biomarker validation into a clinical trial We propose a prospective analysis of a cohort of 680 patients transplanted from an HLA-identical sibling donor at Saint Louis hospital. Analyses will be performed during 3 critical, clinically relevant, periods. 1. Period 1: Analysis at the onset of GVHD, or at the time of engraftment 30 days after HSCT in patients not developing GVHD. An additional blood sample will also be analyzed 90 days after HSCT. 2. Period 2: Thymic function analysis using measurements of T-cell receptor excision circles (TREC) will be performed at 6 and 12 months post-transplant for all patients. T-cell receptor analysis on sorted T-cell populations will be performed by NGS. 3. Period 3: In "tolerant" patients (patients more than 2 years after HSCT not requiring immunosuppressive treatment), or in patients still requiring immunosuppressive therapy after 2 years. We will also analyze the corresponding immune parameters for each donor. The longitudinal design of this study will allow us to provide an integrated view of GVHD pathophysiology and mechanisms of immune tolerance in human. Prospectively identified phenotypic, molecular or functional biomarkers will then be tested, in a subsequent study, from biological materials prospectively collected within the French wide CryoStem cohort. Thus, as the final task of this project, we will perform statistical analyses taking into account confounding clinical variables influencing the outcome (i.e. GVHD-related death or tolerance). Preparing for a clinical trial will need moving from classical Bioinformatics analyses into clinically relevant statistical analyses that include sequential biological measurement in the discovery set cohort. Main points that will be taken into accounts for this task are the followings; 1. Transplant-related mortality (TRM) can be estimated in the range of 20%; 2year post-allogeneic HSCT 2. TRM is mostly (even if totally) due to GVHD and its associated immune deficiency 3. GVHD cumulative incidence can be estimated in the range of 40% 4. 80 patients will be prospectively studied and 30 patients will be analyzed (cross sectional study) for part 3 only. 5. Since GVHD-related mortality and tolerance are mutually exclusive situation the optimal calculation for the validation cohort can be expected 6. This calculation will be the basis for the proposal of an interventional clinical trial.

NCT ID: NCT02316106 Active, not recruiting - Multiple Myeloma Clinical Trials

A Study to Evaluate 3 Dose Schedules of Daratumumab in Participants With Smoldering Multiple Myeloma

Start date: May 20, 2015
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate three daratumumab dose schedules in participants with Smoldering Multiple Myeloma.

NCT ID: NCT02313389 Active, not recruiting - Clinical trials for Primary Central Nervous System Lymphoma

Maintenance Treatment Versus Observation in Elderly Patients With PCNS Lymphoma

BLOCAGE-01
Start date: September 2015
Phase: Phase 3
Study type: Interventional

Hypothesis Our hypothesis is that maintenance chemotherapy will prolong complete remission obtained after a standard induction chemotherapy with an acceptable toxicity in the elderly. Rationale - Treatment of the elderly is challenging, indeed age over 60 is associated both with a poor prognosis and a high risk of treatment induced neurotoxicity with devastating consequences on quality of life. Therefore it has become standard practice to treat elderly in first line with high-dose methotrexate (MTX) based polychemotherapy alone, avoiding whole brain radiotherapy (WBRT) or deferring it for recurrence. - There is a clear need to improve disease control after induction chemotherapy. Since consolidation with WBRT or intensive chemotherapy with autologous stem cell rescue are either poorly effective and/or too toxic in the elderly population, maintenance chemotherapy is an interesting alternative approach. Several agents, such as high-dose MTX, temozolomide (TMZ), rituximab, with a reported activity in PCNSL and acceptable safety profile, as single agent or combined, are good candidates for maintenance

NCT ID: NCT02311959 Active, not recruiting - Breast Cancer Clinical Trials

Therapeutic Nipple Sparing Mastectomy.

MAPAM-01
Start date: February 9, 2015
Phase: Phase 3
Study type: Interventional

This is a phase III, multicentric, single arm study with a sequential design using the exact conditional Poisson test. The primary objective of this prospective study is to evaluate whether selected breast carcinoma patients could be treated with nipple sparing mastectomy (NSM) with an acceptable low local recurrence rates. 450 patients will be included over a period of 4 years and will be followed for a 5 years period (twice a year). All patients must have a complete preoperative evaluation (bilateral mammography, bilateral breast ultrasonography +/- MRI), and a histopathological diagnostic proof of carcinoma (invasive or in situ, all histopathological types, first treatment or relapse). The study procedure is represented by nipple areola skin-sparing mastectomy followed by immediate breast reconstruction. After surgery, early and late complications, including specific complications (suffering of the nipple-areolar complexe and suffering of the skin flaps), will be evaluate. An esthetic evaluation (by patient and clinician) and a quality of life evaluation will be realized throughout the study. A complementary study, prospective, non-interventional, will be proposed to patients who will benefit from the same surgical technique (NSM) followed by immediate reconstruction, but in the context of preventive surgery (prophylactic surgery for patients mutated or high genetic risk). The aim of this complementary study, which will include a maximum of 60 patients, is to evaluate associated morbidity in prophylactic NSM. Data will be collected over a 3 months period after surgery.