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In the study the investigators plan to include 100 early affected HD patients (5 ≤ UHDRS ≤ 40) in a randomized, double-blind, controlled study in 2 centers (France and the Netherlands). Patients will receive either triheptanoin at 1g/kg of body weight per day (n = 50), or a contr…

Over 50% of the more than 270,000 childhood cancer survivors in the U.S. have been treated with anthracyclines and thus are at risk of developing cardiotoxicity. The impact of exercise training on LV structure has been extensively studied. Left ventricular hypertrophy and cardiac…

This is a dose-escalation to determine the MTD and/or RPII for combinations of ibrutinib (PCI-32765) plus lenalidomide/rituximab in patients with relapsed/refractory mantle cell lymphoma.

At present, widespread use of the human milk-based caloric supplement (cream) has not occurred, particularly in infants with bronchopulmonary dysplasia (BPD), and further data are needed to support its adoption as a standard care practice. The investigators hypothesize that infa…

This is an open, randomized, rehabilitation-controlled study in subjects with complete Traumatic Spinal Cord Injury, where the active treatment consists of a surgical implantation of SC0806 (a biodegradable device with heparin-activated FGF1 and nerve implants).

Bacterial vaginosis (BV) is the most common vaginal infection in women. It is caused by a vaginal bacterial imbalance. Treatment with antibiotics is the standard of care but there is a high rate of recurrence. Almost half of women successfully treated suffer a recurrence within t…

Primary Objective: To assess the activity of Afatinib in patients with persistent or recurrent uterine serous carcinoma overexpressing HER2/neu with the frequency of patients who survive progression-free for at least 6 months after initiating therapy. Secondary Objectives: To ass…

This is a multicenter study assessing the efficacy of different therapeutic strategy in patients with advanced sarcomas.

Drug-eluting stents (DES) have long been recommended as the default device for patients undergoing percutaneous coronary intervention (PCI). Drug-Coated Balloon (DCB) angioplasty is similar to plain old balloon angioplasty procedurally, but there is an anti-proliferative medicati…

This study evaluates the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-881 in Gliomas, that harbor an IDH1 and/or IDH2 mutation.

Our goal is to temporarily implant the following groups for 540 +/- 30 days: 1. Forearm FAST electrodes 1. Five human partial hand amputees (amputated at the level of the hand) with 2 FAST electrodes in the ulnar nerve and 2-5 FAST electrodes in the median nerve. 2. Five …

This study will implement and evaluate a mentoring program designed to promote positive youth development and reduce adverse outcomes among maltreated adolescents with open child welfare cases. Teenagers who have been maltreated are at heightened risk for involvement in delinquen…

A phase I/II study of pazopanib in combination with temozolomide in patients with newly diagnosed glioblastoma multiforme after surgery and RT-CT (PAZOGLIO study)

This is a prospective, non-randomized, non-blinded observational study. The overarching goal is to discover new disease-associated genes in children, while establishing a specific focus on disorders where molecular characterization is most likely to lead to novel therapies. This …

This is an open label multi center trial to determine the safety and efficacy of ceritinib in combination with nivolumab in ALK-positive NSCLC patients

This randomized phase II/III trial studies how well afatinib dimaleate with cetuximab works and compares it with afatinib dimaleate alone in treating patients with newly diagnosed stage IV or recurrent (has come back), epidermal growth factor receptor (EGFR) mutation positive non…

Objectives: Primary Objective To provide compassionate access to the Miltenyi device for CD34+ cell infusions to patients who experience poor graft function after stem cell transplantation (SCT).

This is a Phase I study to determine the maximum tolerated dose (MTD) and/or recommended phase II dose of D2C7-IT (D2C7 Immunotoxin) when delivered intratumorally by convection-enhanced delivery (CED) to recurrent World Health Organization (WHO) grade III and IV malignant glioma …

Background: \- Radiation can cause the parotid salivary glands to make less saliva (dry mouth). This can cause problems like infections and tooth decay. Researchers hope a new drug can help people with dry mouth caused by radiation. Objectives: \- To examine the safety of AAV2…

Desmoid tumors (DT) are rare tumors (2-4 cases/million/year) that originate from musculoaponeurotic structures. Although they are benign tumors with no metastatic potential, DT are considered as locally aggressive tumors, with local invasiveness and tissue destruction, leading to…