Phoenix, AZ Paid Clinical Trials

This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia. This Registry will be open for enrollment for 7 years and all enrolled participants will be followed prospectively for a minimum of 2 years, and up to 9 years. Data will be collected from participating Registry Physicians, participants, and, where appropriate, parents/guardians who have provided informed consent or assent (where relevant) and authorization pursuant to applicable laws and regulations. Data should include demographic, clinical, and treatment data; and other data of relevance to the management of patients with PK deficiency. Annual chart review and data entry are expected in order to enhance longitudinal understanding of PK deficiency; however, no specific protocol schedule of assessment is required by this Registry protocol. Read Less

Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how safe and effective Upadacitinib is in treating pediatric participants with UC. Adverse events and change in disease activity will be assessed. Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active UC and is being developed for moderate- to severely active UC in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: an 8-week open-label induction phase which means that the study doctor and patients know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 44-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 260 week open-label extension (OLE) of Period 1. Approximately 110 pediatric participants with moderate to severely active UC will be enrolled at up to 100 sites worldwide. Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will be followed up for 30 days after each phase (i.e. after induction, maintenance, OLE) and only if a participant doesn't continue into the next phase. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires. Read Less

The goal of this clinical trial is to evaluate if the study drug CIN-102 (deudomperidone) can help reduce the symptoms associated with diabetic gastroparesis in adult patients. The main questions it aims to answer are: To evaluate the efficacy of CIN-102 on symptoms of gastroparesis when given to patients with diabetic gastroparesis compared to a placebo To evaluate the safety and tolerability of CIN-102 when given to patients with diabetic gastroparesis compared to a placebo Participants will go through the following schedule: Screening period (1-2 visits) Lead-in period (1 visit) Will complete a Gastric Emptying Breath Test (GEBT) Will complete daily diary and other Patient Reported Outcomes (PROs) as described in the protocol to assess eligibility for continued study participation 12-week treatment period (5 visits) Study drug taken twice daily by mouth Will complete daily diaries and other PROs as described in protocol 1 week follow-up (1 visit) Researchers will compare the effects of the following treatments: Drug- CIN-102 Dose 1 Drug- CIN-102 Dose 2 Drug- Placebo Read Less

An antibody is a substance your body makes to fight off infection. This study will explore the safety and antibody response of a vaccine to prevent severe diarrhea caused by a germ called Clostridoides difficile (C. diff). Three new formulations of the C. diff vaccine will be used in this study, in addition to a C. diff vaccine formulation that has been studied in previous clinical trials. The purpose of this study is to understand if giving the new C. diff vaccine formulations helps people make as many antibodies as giving the previously studied C. diff vaccine formulation. The study is divided into 2 phases. Phase 1 will evaluate 3 new formulations of the C. diff vaccine and 2 dosing schedules spread out over 2 months or 6 months. The Phase 1 portion of the study is seeking participants: who are healthy adults of 65 to 84 years of age who have not had a C. diff infection before who have not received a C. diff vaccine or C. diff monoclonal antibody therapy before. All participants in Phase 1 will receive study injections with active vaccine or placebo at each vaccination visit, depending on the vaccine group to which they are assigned. A placebo does not contain any active ingredients. Participants in Phase 1 will attend at least 9 study visits and will take part in the study for approximately 18 months. Based on the results of Phase 1, 1 or 2 of the new C. diff vaccine formulations will be chosen for further study in Phase 2. Phase 2 will evaluate the safety and effects of the new C. diff vaccine formulation(s) chosen in Phase 1. The Phase 2 portion of the study is seeking participants: who are healthy adults ≥65 years of age who have not had a C. diff infection before who have not received a C. diff vaccine or C. diff monoclonal antibody therapy before. All Phase 2 participants will receive active C. diff vaccine or placebo at each vaccination visit. Participants in Phase 2 will attend at least 6 and up to 11 study visits and will take part in the study for approximately 4.5 years. Read Less

The purpose of this study is to measure the benefit of adding abemaciclib to chemotherapy (irinotecan and temozolamide) for Ewing's sarcoma that has come back or did not respond to treatment. This trial is part of the CAMPFIRE master protocol, which is a platform to speed development of new treatments for children and young adults with cancer. Your participation in this trial could last 11 months or longer, depending on how you and your tumor respond. Read Less

The purpose of this study is to evaluate the efficacy, safety, drug levels, and drug effects of ozanimod in pediatric participants with moderately to severely active Crohn's Disease. Read Less

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. Read Less

This is a global, multi-center, prospective, randomized, blinded, controlled pivotal study. Clinical and radiological evaluation will be performed preoperatively and postoperatively up to 24 months; and endpoint success will be determined at 24 months postoperatively. Overall a maximum of 1017 subjects will be enrolled and treated. The purpose of the study is to provide safety and effectiveness data of Infuse™ in A Transforaminal Lumbar Interbody Fusion (TLIF) procedures and to obtain indication expansion for Infuse™ use in one and two level TLIF procedures. Read Less

The primary objective of this study is to characterize real-world clinical outcomes of Deep Brain Stimulation (DBS) using retrospective review of de-identified patient records. Read Less

This study is an open-label, multi-center, non-randomized pivotal Phase 3 study to assess the efficacy and safety of PET imaging with [18F]PI-2620 for detection of tau deposition in subjects with Alzheimer's disease (AD) and controls during lifetime when compared to histopathology obtained after death and completion of brain autopsy. Read Less

The purpose of the dose escalation phase is to evaluate the safety profile of escalating doses and dose schedules of NXP800. In the expansion phase the preliminary efficacy in subjects with ARID1a mutated ovarian clear cell and ovarian endometrioid cancers will be estimated. Read Less

The purpose of this trial is to measure the efficacy, safety, and tolerability of darigabat (25 milligrams [mg] twice daily [BID]) compared with placebo in participants with panic disorder. Read Less