
Safety, Tolerability and Pharmacokinetics of Milademetan Alone and With 5-Azacitidine (AZA) in Acute...
Acute Myelogenous LeukemiaMyelodysplastic SyndromeThis study will take place in parts: Dose Escalation (Part 1): Participants receive milademetan alone with different dose schedules Dose Escalation (Part 1A): Participants receive milademetan in combination with 5-azacytidine (AZA), with different dose schedules The recommended dose for Part 2 will be selected. Dose Expansion (Part 2): After Part 1A, participants will receive the recommended Part 2 dose schedule. There will be three groups - those with: refractory or relapsed acute myelogenous leukemia (AML) newly diagnosed AML unfit for intensive chemotherapy high-risk myelodysplastic syndrome (MDS) End-of-Study Follow-Up: Safety information will be collected until 30 days after the last treatment. This is the end of the study. The recommended dose for the next study will be selected.

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency...
Glucose Transporter Type 1 Deficiency SyndromeThe primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.

A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
Down SyndromeThis study will evaluate the safety, tolerability, efficacy, and pharmacokinetic and pharmacodynamic activity of 3 different dosages of RO5186582 compared with placebo. A total of approximately 46 participants will be enrolled, in order to have at least 32 evaluable, and will be randomly assigned to 1 of 4 treatments in a 1:1:1:1 ratio, with 9 children per treatment arm. The target ratio between 6-8 years and 9-11 years age groups is approximately 1:1 in each treatment arm, with a minimum of 3 children per age group in each treatment arm.

Platelet-rich Plasma (PRP) Injection for Treating Shoulder Subacromial Impingement Syndrome
Shoulder Impingement SyndromeThe purpose of this study is to determine whether injection of platelet-rich plasma derived from patient's blood is effective in treatment of shoulder subacromial impingement syndrome as compared to the current protocol of methylprednisolone injection.

Blinded, Randomized Study of Gabapentin (Neurontin®) and Gabapentin Enacarbil (Horizant™) in Restless...
Restless Leg SyndromeThe study will compare the safety, effectiveness and tolerability of gabapentin (Neurontin) versus gabapentin enacarbil (Horizant) as treatment restless leg syndrome.

Subcutaneous Nerve Stimulation for Failed Back Surgery Syndrome (FBSS) Patients
Failed Back Surgery SyndromePost-laminectomy SyndromeThe purpose of this study is to assess the safety and efficacy of Subcutaneous Nerve Stimulation (SQS) (also known as Peripheral Nerve Stimulation [PNS]) in the reduction of chronic, intractable post-surgical back pain in adults.

Study of Azacitidine With or Without Birinapant in Subjects With MDS or CMMoL
Myelodysplastic Syndrome (MDS)Chronic Myelomonocytic Leukemia (CMML)This is a randomized double blind placebo controlled study of azacitidine with or without birinapant in subjects with higher risk Myelodysplastic syndrome, secondary MDS or myelomonocytic leukemia (CMMoL) who are naïve, to azacitidine therapy. Pre-clinical and mechanistic studies support that azacitidine may modulate pathways that enable birinapant-mediated anti-tumor activity.

Feasibility Study of Unfractionated Heparin in Acute Chest Syndrome
Acute Chest SyndromeSickle Cell DiseaseThe purpose of this study is to determine the feasibility of performing a larger multicenter phase III trial to assess the effects of unfractionated heparin (UFH) in acute chest syndrome (ACS). Prespecified feasibility criteria consists of the ability to enroll potential study participants, which includes the timely notification of hospitalized patients with ACS, the capacity to consent eligible individuals, and the ability to appropriately randomize eligible patients within 24 hours of diagnosis. Additional feasibility objectives involve ensuring appropriate eligibility criteria, proper administration of the study drug, and the ability to completely and accurately collect clinical data of interest. The final aim of our pilot study is to provide preliminary data, with respect to treatment effect and variance, to allow sample size calculation in a larger trial given the lack of data available to help guide this process. The investigators hypothesize that the use of UFH in ACS will result in a decrease in the duration of hospitalization and improve other clinical outcomes, such as the duration of hypoxemia and duration of moderate to severe pain.

Registry for Participants With Short Bowel Syndrome
Short Bowel SyndromeThis is a global prospective, observational, multi-center registry to evaluate the long-term safety profile for participants with short bowel syndrome (SBS) who are treated with teduglutide in a routine clinical setting. The registry will also evaluate the long-term clinical outcomes in participants with SBS. SBS participants treated and not treated with teduglutide will be enrolled.

Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
Myelodysplastic SyndromeTransfusional Iron OverloadThe objective of the study is to evaluate and compare the frequency and severity of GI adverse events in different dose administration regimens. The patient population consists of low or intermediate (int-1) risk myelodysplastic syndrome (MDS) patients with transfusional iron overload. The study patients are randomized to either a morning dose of 20 mg/kg/day deferasirox or an evening dose of the same. Patients are then followed up for 6 months for any GI events and are assessed using patient reported outcomes tools e.g. a patient diary.