International Intestinal Failure Registry

Date Added
August 4th, 2021
PRO Number
Pro00112520
Researcher
Candi Jump

List of Studies


Keywords
Digestive System, Metabolism, Nutrition, Rare Diseases
Summary

The International Intestinal Failure Registry (IFR) is an initiative of the Intestinal Rehabilitation and Transplant Association (IRTA) and The Transplantation Society (TTS) and will be managed by these organizations. The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.

The primary objective of this project is to create a large international database of children with intestinal failure to characterize their management and outcome and guide the development of best practices and evidence-based management.

Institution
MUSC
Recruitment Contact
Candi Jump
843-792-5021
jump@musc.edu

Gabapentin for Restoring GABA/glutamate Homeostasis in Co-occurring Bipolar and Cannabis Use Disorders: A Randomized, Double-blind, Placebo-controlled, Parallel-group, MRI Study

Date Added
August 3rd, 2021
PRO Number
Pro00112593
Researcher
James Prisciandaro

List of Studies


Keywords
Depression, Mental Health, Psychiatry, Substance Use
Summary

This research study evaluates the effects of anFDA-approved medication Gabapentin in individuals with Bipolar Disorder who smoke marijuana. Participants in the study will will be assigned to take either Gabapentin or a matched placebo. Study medication will be taken for 17 days. There will be 5 study visits, with 2 MRI brain imaging scans completed. Questionnaires and clinical interview measures will be completed at study visits along with consistent assessment of potential side effects from study medication.

Institution
MUSC
Recruitment Contact
Sara Hix
843-792-0572
hixs@musc.edu

Evaluation of Trauma Center-Based, Technology-Enhanced Stepped Care Intervention for Adolescent Traumatic Injury

Date Added
August 3rd, 2021
PRO Number
Pro00111879
Researcher
Tatiana Davidson

List of Studies


Keywords
Adolescents, Mental Health
Summary

Pediatric traumatic injury is the leading cause of death and morbidity among US adolescents and are associated with mental health and health risk outcomes, including posttraumatic stress and depression, deficits in physical recovery, social functioning and quality of life, which if unaddressed, may contribute to increased use of health care services. In 2015 our team launched the Trauma Resilience and Recovery Program (TRRP) at Medical University of South Carolina, a scalable and sustainable, technology-enhanced, multidisciplinary stepped model of care – one of the few in the US - that provides early intervention and direct services to improve access to evidence-based mental health care after traumatic injury for children, adults and families. We have found this model of care to be feasible and acceptable to adolescent patients (ages 12-17) at each level of service. TRRP includes 3 major steps: (1) in-hospital education, brief risk reduction session, and tracking patients' emotional recovery via an automated text-messaging system, (2) a 30-day screen via telephone to identify patients who are good candidates for psychological treatment, and (3) providing referral to best-practice telehealth-based or in-person assessment and treatment. We have partnered with three accredited Level I and II pediatric trauma centers, Prisma-Health Upstate, Children's of Alabama, and Boston Children's Hospital, and propose a multi-site hybrid 1 effectiveness-implementation randomized controlled trial with 300 adolescent (ages 12-17) traumatic injury patients to assess the extent to which TRRP promotes improvement in quality of life and emotional recovery and gather preliminary data on the potential for TRRP to be implemented in other Level I trauma centers. This study will provide valuable data on the efficacy, preliminary effectiveness and potential for implementation of an innovative, cost-effective, sustainable technology-enhanced intervention designed to address the unique needs of adolescent injury patients and mitigate short- and long-term impact of injury on mental health, quality of life, and overall well-being.

Institution
MUSC
Recruitment Contact
Olivia Eilers
843-792-0979
eilerso@musc.edu

First-in-Human, Phase 1/1b, Open-label, Multicenter Study of Bifunctional EGFR/TGF-β Fusion Protein BCA101 Monotherapy and in Combination Therapy in Patients with EGFR-Driven Advanced Solid Tumors

Date Added
July 27th, 2021
PRO Number
Pro00111442
Researcher
John Kaczmar

List of Studies


Keywords
Cancer, Drug Studies, Men's Health, Women's Health
Summary

This study is for patients who have advanced cancers, such as head and neck cancer, colorectal cancer, breast cancer, and others. This study is testing a new treatment for these types of cancer. The new test drug is called ficerafusp alfa. Pembrolizumab is an approved drug by the United States Food and Drug Administration (FDA) for the diseases described in this study and will be used as approved. The type of cancer a patient has will determine whether he/she will get ficerafusp alfa alone or ficerafusp alfa in combination with pembrolizumab. Participants will receive either ficerafusp alfa alone, ficerafusp alfa in combination with pembrolizumab or ficerafusp alfa, depending on your cancer until the cancer gets worse, they experience bad side effects, or until they withdraw consent, or until the Investigator considers it is in his/her best interest to discontinue the study drug.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu

Randomized, Phase III Study of Early Intervention With Venetoclax and Obinutuzumab Versus Delayed Therapy With Venetoclax and Obinutuzumab in Newly Diagnosed Asymptomatic High-Risk Patients With Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL): EVOLVE CLL/SLL Study

Date Added
July 22nd, 2021
PRO Number
Pro00111458
Researcher
Brian Hess

List of Studies


Keywords
Cancer, Cancer/Leukemia, Cancer/Lymphoma
Summary

This study is for newly diagnosed asymptomatic high-risk patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The purpose of this study is to find out if starting treatment with the investigational products, venetoclaz and obinutuzumab (V-O) early (before symptoms occur) affect how long you live compared to the usual approach of starting treatment after showing symptoms. Participants can expect to receive treatment for up to 12 months, until the cancer gets worse or until the side effects are too great. After study completion, participants will continue to be followed for up to 10 years.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu

Combined Deep Brain Stimulation and Functional Magnetic Resonance Imaging

Date Added
July 20th, 2021
PRO Number
Pro00111668
Researcher
Gonzalo Revuelta

List of Studies


Keywords
Brain, Movement Disorders, Parkinsons
Summary

The purpose of this research study is to identify brain activation patterns in response to deep brain stimulation (DBS). To participate in this study, participants are required to have had a DBS implant or to be healthy controls without a history of a neurological disorder. Participants will undergo screening, a motor assessment session and a 30-minute Magnetic Resonance Imaging (MRI) session where their DBS device will be turned on and off in a cycled pattern. This study will be separated over 3 visits (Visit 1 for screening, Visit 2 for motor assessments and Visit 3 for the MRI scans). The total study duration will be approximately 5 hours.

Institution
MUSC
Recruitment Contact
Daniel Lench
843-792-9115
lenchd@musc.edu

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease

Date Added
July 13th, 2021
PRO Number
Pro00107924
Researcher
Shayla Bergmann

List of Studies


Keywords
Blood Disorders
Summary

Vaso-occulsive crisis is a complication of Sickle cell disease in which the red blood cells (RBC) change shape, causing congestion within the blood vessels that leads to pain and tissue damage.

The study medication FT-4202, an oral tablet, is believed to reduce the rate of sickle cell polymerization and improve RBC membrane function, thereby reduction sickling of RBCs and their hemolysis (breakdown of red cells) that causes vascular obstruction and anemia.

This study will consist of a 52-week, randomized (volunteers are selected by chance to receive study either study medication or placebo) , placebo controlled (a placebo is a look-alike pill that contains no active medication). There will be 17 study visits.

The study is followed by a 52-week open label extension study in which all participants will receive study medication. There will be 11 study visits.

Institution
MUSC
Recruitment Contact
Karen Hawkins
843-792-0560
hawkink@musc.edu

Clinical Feasibility of Low Intensity Focused Ultrasound Pulsation for the Treatment of Generalized Anxiety Disorder

Date Added
July 6th, 2021
PRO Number
Pro00109178
Researcher
Mark George

List of Studies


Keywords
Anxiety
Summary

The purpose of this research is to test out a new way of treating generalized anxiety disorder (GAD) with low intensity focused ultrasound pulsation (LIFUP). If the subject agree to participate in the research study, the participation will last approximately 10.5 hours over seven (7) days. The visits are as follows: 1- consent and MRI, 2- and 3- focused ultrasound will be delivered outside of the MRI environment, 4- MRI plus focused ultrasound, 5- assessments 1 week post focused ultrasound, and 6- one month follow-up assessments. Research studies are voluntary and include only people who agree to take part.

Institution
MUSC
Recruitment Contact
Sarah Huffman
843-792-8672
huffmans@musc.edu

Optimal Perioperative Therapy for Incidental Gallbladder Cancer (OPT-IN)- A Randomized Phase II/III Trial

Date Added
July 5th, 2021
PRO Number
Pro00112123
Researcher
William Hawkins

List of Studies

Keywords
Cancer/Gastrointestinal, Gall Bladder
Summary

This study is for patients with gallbladder cancer. The purpose of this study is to compare the usual treatment (surgery plus chemotherapy after) to using chemotherapy both before and after surgery.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
(843) 792-9321
hcc-clinical-trials@musc.edu

A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome

Date Added
June 25th, 2021
PRO Number
Pro00112028
Researcher
Caroline Buchanan

List of Studies

Keywords
Genetics
Summary

The Drug Product ZYN002 is a transdermal CBD gel. CBD is the primary non-euphoric cannabinoid contained in the Cannabis sativa L. plant. The CBD contained within ZYN002 is a pharmaceutically produced Active Pharmaceutical Ingredient (API) that is chemically identical to the CBD present in Cannabis. ZYN002 is currently being evaluated in clinical trials in children and adolescents with Fragile X Syndrome (FXS), autism spectrum disorder, 22q11.2 deletion syndrome, and developmental and epileptic encephalopathies. The safety and efficacy of ZYN002 in the treatment of behavioral symptoms in children and adolescents with FXS has been evaluated in three studies: Study ZYN2-CL-009, a completed open-label, multiple-center, multiple-dose study (n=20); Study ZYN2-CL-016, a completed randomized, double-blind, placebo-controlled, multiple-center study (n=212 randomized); and Study ZYN2-CL-017, an ongoing open-label extension and expanded access study to assess the long-term safety and tolerability of ZYN002 (n=240). The present protocol for ZYN2-CL-033 (RECONNECT) is designed to evaluate the efficacy and safety of ZYN002 for the treatment and behavioral symptoms in children and adolescents with genetic evidence of full mutation FXS. Qualified subjects that complete ZYN2-CL-033 will have the opportunity to roll over to the open label ZYN2-CL-017 study.

Institution
Self Regional Healthcare
Recruitment Contact
Sarah English
8646726912
senglish@ggc.org



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