Sharing Our Stories: The Realities of Living with Ehlers Danlos Syndromes

Date Added
August 7th, 2025
PRO Number
Pro00144466
Researcher
Michelle Nichols

List of Studies


Keywords
Autoimmune disease, Chronic Fatigue, Genetics, Joint, Non-interventional, Pain, Rheumatoid
Summary

Sharing Our Stories: The Realities of Living with Ehlers Danlos Syndromes (EDS) is a research study to help better understand what it is like to live with EDS. Adults with EDS will complete a one-time video interview with a member of the research team. We hope to better understand the challenges adults with EDS face and learn from them things that make it more difficult and easier to receive care, what their needs are and recommendations for improving an earlier diagnosis, care, and treatment.

Institution
MUSC
Recruitment Contact
Michelle Nichols
843-876-6463
nicholmg@musc.edu

A Phase I, First-in-human, Open-label, Dose Escalation Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BNT317 in Patients With Advanced Solid Tumors

Date Added
August 7th, 2025
PRO Number
Pro00143120
Researcher
John Kaczmar

List of Studies


Keywords
Cancer, Drug Studies
Summary

This study is for patients that have been diagnosed with advanced solid tumors. This study is testing an investigational drug called BNT317. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to evaluate the safety, tolerability, absorption of BNT317. BNT317 is administered via intravenous (IV) infusion. Participants can continue to receive this study drug until it no longer gives them benefit. Researchers will continue to follow-up with patients long-term.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
8437929321
hcc-clinical-trials@musc.edu

A Phase 3 Open-Label, Randomized Study of PDS0101 Plus Pembrolizumab vs. Pembrolizumab Alone in First Line Treatment of Immune Checkpoint Inhibitor (ICI) Naive Subjects with Unresectable Recurrent and/or Metastatic (R/M) Human Papillomavirus 16 (HPV16)-Related Head and Neck Squamous Cell Carcinoma (HNSCC)

Date Added
August 8th, 2025
PRO Number
Pro00145400
Researcher
John Kaczmar

List of Studies


Keywords
Cancer, Cancer/Head & Neck, Drug Studies, Men's Health, Women's Health
Summary

The purpose of this study is to test the effectiveness and safety of the combination of an investigational vaccine, PDS0101, given by subcutaneous (beneath the skin) injection and the standard of care, Pembrolizumab (KEYTRUDA), given intravenously ("IV;" into the vein) compared to pembrolizumab (KEYTRUDA) alone for those with unresectable recurrent or metastatic head and neck cancer and high risk papillomavirus infection. "Investigational" means the combination of the study vaccine and pembrolizumab being tested has not been approved by the US Food and Drug Administration (FDA) nor any other Health Authority.

Institution
MUSC
Recruitment Contact
HCC clinical trails office
843-792-9321
hcc-clinicaltrials@musc.edu

A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate/Ruxolitinib versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation

Date Added
August 11th, 2025
PRO Number
Pro00146236
Researcher
Jonathan Alexander

List of Studies

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

This phase 3 study is recruiting patients who are at risk of graft-versus-host disease (GVHD) after a bone marrow transplant. This study will measure the safety and effectiveness of a prevention treatment combination called Tacrolimus/Methotrexate/Ruxolitinib compared to Standard of Care (SOC), Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil. Ruxolitinib (Rux) is an approved treatment for GVHD. This study is divided into two parts. The first part, called the run-in phase, will investigate the best dose of Ruxolitinib. The second part of the study will compare the SOC combination therapy with the investigational combination therapy (which will include Ruxolitinib). Participants will be randomly assigned to one of the two groups (like flipping a coin). The study will enroll up to 572 patients nationwide and 5 at MUSC. The participants can expect to be involved in the study for at least 24 months. The main risk is that medical treatments often cause side effects. The most common side effects expected from the investigational combination therapy are high cholesterol, increased liver enzymes, low platelet levels, and low red blood cell counts. There is no direct benefit for them in participating in this study.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
8437929321
hcc-clinical-trials@musc.edu

A Randomized Phase II Study of Venetoclax and HMA-based Therapies for theTreatment of Older and Unfit Adults With Newly Diagnosed for FLT-3 Mutated Acute Myeloid Leukemia (AML): a MyeloMATCH Treatment Trial

Date Added
August 19th, 2025
PRO Number
Pro00146654
Researcher
Praneeth Baratam

List of Studies

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

This phase 2 study is enrolling patients who have acute myeloid leukemia (AML) with certain biomarkers. This study is being done to see the effectiveness of different combinations of drugs to treat AML. It will involve 3 groups of patients receiving different combinations of Gilteritinib, Azacitidine and Venetoclax. Gilteritinib is an investigational drug, Azacitidine and Venetoclax are FDA approved. The main purpose of the study is to see if the amount of leukemia in the patient's body can be lowered by adding the drug Gilteritinib to the Standard of Care of treating AML with Azacitidine and Venetoclax. The study will include approximately 147 patients. The patients will be randomized into the three groups (like flipping a coin), Group 1 will receive just Azacitidine + Venetoclax, Groups 2 and 3 will also receive Gilteritinib but Group 2 will receive it for more time within a treatment cycle. Patients will complete screening after participating in this treatment trial or SOC therapies. Patients will continue treatment until disease progression, unacceptable toxicity, study closure, death, or withdrawal of consent. The main risk is that the study drugs may not be as good as the usual approach for their cancer or condition at shrinking or stabilizing their cancer. Patients may have none, some, or all of the side effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit for them in participating in this study.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
8437929321
hcc-clinical-trials@musc.edu

An Open-label, Multicenter Trial to Assess the Safety and Tolerability of Lumateperone in the Treatment of Pediatric Patients with Schizophrenia, Bipolar Disorder, or Autism Spectrum Disorder

Date Added
August 21st, 2025
PRO Number
Pro00140167
Researcher
Thomas Uhde

List of Studies


Keywords
Autism
Summary

Currently, there are no FDA-approved medications for the treatment of irritability associated with Autism Spectrum Disorder (ASD). This study is designed to look at the usefulness and safety of lumateperone (CAPLYTA) for the treatment of irritability associated with ASD among pediatric participants between the ages of 5 to 17 years. The study will last approximately 26 weeks and the participants will receive the study drug, lumateperone.

Institution
MUSC
Recruitment Contact
Jelissa Suarez
843-876-9262
suarezj@musc.edu

Quantification of phosphorylated alpha-synuclein in patients with Parkinson's disease and REM sleep behavior disorder (Syn-Q)

Date Added
August 27th, 2025
PRO Number
Pro00143143
Researcher
Christine Cooper

List of Studies


Keywords
Parkinsons
Summary

This study aims to study the natural progression of P-SYN quantification by conducting skin biopsies in patients with Parkinson's Disease (PD) and REM sleep behavior disorder (RBD). Participation will be over an 18-month period and will include skin biopsies and clinical examinations every six months. Subjects will be male or female between the ages of 50-100 and have a diagnosis of PD or RBD.

Institution
MUSC
Recruitment Contact
Sandra Wilson
8437924616
MovementResearch@musc.edu

A Phase 3, Multicenter, Randomized, Subject and Evaluator Blinded, Controlled Study Evaluating the Safety and Efficacy of NTX-001 Compared to Standard of Care (Neurorrhaphy) in the Treatment of Upper Extremity Transected Nerves Requiring Surgical Repair

Date Added
August 27th, 2025
PRO Number
Pro00145325
Researcher
Charles Daly

List of Studies

Keywords
Nerve, Surgery
Summary

The study is a 48-week clinical study to compare study medication NTX-001 used intraoperatively to promote nerve regeneration compared to the standard of care on surgery for acute nerve damage in the hand on subjects between the ages of 18 to 80 years old. Subjects who present with sever nerve damage of the hand and need surgery to repair the nerve damage within 48 hours of injury. After surgery, subjects will complete 12 total study visits: 6 in person and 6 additional telehealth visits. During the in-person clinic visits, subjects will be assessed by physical examinations, vital signs, safety laboratory assessment and complete questionnaires. Subjects with motor/mixed motor injury will also complete an EMG and NCV assessments with an Occupational Therapist at 2 of the in-person visits. During the telehealth visits, subjects will complete questionnaires.

Institution
MUSC
Recruitment Contact
Lisa Mock
843-876-2211
mockl@musc.edu

Prospective changes in patient-reported and objective functioning following reduction of cannabis use

Date Added
September 2nd, 2025
PRO Number
Pro00145652
Researcher
Rachel Tomko

List of Studies


Keywords
Mental Health, Psychiatry, Substance Use
Summary

Adults (ages 18+) who use cannabis and are interested in reducing their use will be enrolled in an 8-week treatment program. All participants will receive counseling (1 goals session with a therapist followed by 7 weekly computerized cognitive-behavioral therapy sessions). Detailed cannabis assessments (biological and self-report) will be conducted throughout treatment and at 1- and 4-months post-treatment completion. Daily electronic diaries will be administered via text message to record detailed logs of cannabis use quantity and frequency. Participants are also asked at different points throughout the study to wear a Fitbit to monitor their sleep.

Institution
MUSC
Recruitment Contact
Kevin Branson
843-792-0493
bransonk@musc.edu

Accelerated, Left Prefrontal Transcranial Magnetic Stimulation for Functional Seizures; An Open-Label Exploration of Feasibility, Tolerability, and Preliminary Efficacy

Date Added
September 2nd, 2025
PRO Number
Pro00144502
Researcher
Mark George

List of Studies


Keywords
Mental Health, Psychiatry
Summary

As growing research suggests noninvasive brain stimulation techniques have the potential to adjunct current treatments or treat Seizure-Type Functional Neurologic Disorder (FND-seiz), also known as Psychogenic Non-Epileptic Seizures (PNES), we aim to evaluate whether a form of accelerated intermittent theta burst transcranial magnetic stimulation (a-iTBS-rTMS), is a practical and well-tolerated treatment for people with this disorder. Transcranial Magnetic Stimulation or TMS uses magnetic pulses to stimulate a part of the brain involved in mood and thinking, the left dorsolateral prefrontal cortex, which has established benefits in disorders known to coincide in patients with FND-seiz, such as depression.

As an open-label, early feasibility study, enrolled participants will receive 6 to 10 treatment sessions each day over 3 to 5 days, with the goal of completing 30 total sessions. This approach was selected because similar protocols have already been shown to be safe and effective in other conditions, and the shortened treatment schedule in comparison to other protocols may make participation easier for people living with FND-seiz. The main goal of the study is to see how many participants can safely and comfortably complete at least 20 of the 30 TMS sessions.

The researchers will also evaluate changes in seizure frequency, quality of life, mood, post-traumatic stress symptoms, physical health, social functioning, and overall satisfaction with treatment. These outcomes will be measured before treatment and again four weeks afterward. The researchers also aim to explore whether people with overlapping conditions, such as depression or PTSD, respond differently to the treatment. Finally, given the overlap between epilepsy and FND-seiz, not all TMS providers are comfortable treating patients with FND-seiz when TMS is indicated for other conditions, thus the researchers aim to outline a protocol to ensure safety and increase TMS access for FND-seiz patients.

Institution
MUSC
Recruitment Contact
Joseph Chasen
(843) 637-1358
chasenj@musc.edu



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