This project is an extension of the CDC-funded FORWARD (Fragile X Online Registry With Accessible Research Database) study. From its inception in 2010, the goal of the FORWARD study has been to characterize the natural history of fragile X syndrome (FXS). This current extension project is known as FORWARD-MARCH (Multiple Assessments for Research CHaracterization) because it will include multiple assessments to characterize behavioral, adaptive, and cognitive function in greater depth and thereby further improve understanding of the natural history of FXS. FORWARD-MARCH continues the mission of FORWARD to better understand the natural history of FXS in order to improve the lives of children and adolescents with FXS and the lives of their families. FORWARD-MARCH will also better define trajectories of development in FXS that will be useful in understanding the long-term effects of an intervention relative to the natural history of FXS.

FORWARD-MARCH builds upon the foundation of the FORWARD study. The FORWARD study included 24 participating FXS specialty clinics throughout the US that are members of the FXCRC (Fragile X Clinical & Research Consortium). The FORWARD study worked closely with the Centers for Disease Control and Prevention (CDC), the National Fragile X Foundation (NFXF), and other stakeholders in the FXS community. FORWARD-MARCH will also involve a contractor, Chickasaw Nation Industries (CNI), funded through a contract with the CDC. CNI will assist in data collection and management.

Between September 2022 and August 2026, FORWARD-MARCH expects to enroll at least 600 individuals with fragile X syndrome who were born between 2003-2017. The majority of these individuals will already be FORWARD study participants, enabling researchers to conduct longitudinal analyses incorporating previously collected data. Cognitive, behavioral, and adaptive function will be assessed using parent or caregiver-completed surveys and in-person clinical assessments. After completion of data collection, deidentified data will be securely maintained at CDC and will be an important long-term resource for analyses of the natural history of FXS.

Previous phases of the FORWARD study, conducted between 2012 and 2022, have received IRB review and approval by the institutions of each participating clinic. These previous phases of the study did not require review by a CDC IRB, as CDC had no participant contact and did not have access to personal identifying information (PII). The extension of the FORWARD study covered in this protocol (FORWARD-MARCH, 2022-2026) will continue to be reviewed and approved by the institutions of each participating clinic conducting data collection. However, review and approval are also being sought from the CDC IRB because PII will be maintained on CDC servers and because CDC's contractor, CNI, will regularly have access to PII and interact directly with study participants. A reliance agreement allowing CNI to rely on CDC's IRB is being developed and will be executed before data collection is begun. To clarify which aspects of the protocol involve CDC and CNI staff (rather than just clinic staff), sections 3,4 and 5 of this protocol document each end with a subsection that specifically focuses on the role of CDC and CNI staff.

The study will compare the effectiveness of endoscopic surveillance and endoscopic eradication therapy (EET) for the management of Barrett's esophagus (BE) and low-grade dysplasia (LGD).

Over 2400 people who have sickle cell disease and are between the ages of 15 and 45 have been enrolled into the National Registry (SCDIC-I) of patients with Sickle Cell Disease (SCD). A rich resource of natural history data, the SCDIC-I Registry has longitudinal data collected yearly since 2016 from patient surveys (e.g. self reported pain incidences, sleep, barriers to care, experiences during and after pregnancy), medical record abstraction (e.g. medications, transfusion history, co-morbidities) and laboratory results. The 150 patients (or 1200 among the 8 sites) will be selected from both MUSC adult and pediatric SCD clinics starting at 12 years of age; those not previously enrolled in the SCDIC National Registry will be offered the possibility to enroll in SCDIC-II.
We will look at the following:
1- Compare the effect of new SCD medications – crizanlizumab, voxelotor, and L-glutamine – on clinical outcomes in individuals with SCD.
2 - Identify genetic and genomic predictors of response to crizanlizumab, voxelotor, and L-glutamine
3 - Integrate study data into the CureSCi metadata catalog (MDC) to enhance future cross-study analyses.

The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF pulmonary exacerbations during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean ppFEV1 (pulmonary function testing) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms. The study will compare treatment of an intravenous aminoglycoside and β-lactams (AG) versus intravenous β-lactams only (non-AG) in people with CF diagnosed with a pulmonary exacerbation who will be treated for Pa.

This study is for patients that have been diagnosed with locally advanced head and neck squamous cell carcinoma. The purpose of this research study is to evaluate the effectiveness of using a combination of pembrolizumab and olaparib when given before and after standard chemoradiation therapy in treating locally advanced head and neck squamous cell carcinoma. Pembrolizumab and olaparib are drugs that are approved for treatment of different cancers including lung, head and neck, breast and prostate cancer. However, FDA has not approved use of these two drugs together in treating head and neck cancer.

Treatment will be offered in three phases. In the induction phase, participants will receive a single infusion of pembrolizumab and will take olaparib tablets twice daily for total of 21 days. Participants will move to the chemoradiation phase, where they will receive radiation therapy and chemotherapy per routine standard care, for a total of 7 weeks. Chemoradiation therapy is done on a daily basis (excluding weekends), and chemotherapy therapy will involve a cisplatin infusion once weekly. At the conclusion of this phase, participants start the maintenance phase, which involves treatment with pembrolizumab and olaparib in cycles that are 42-days long. Treatment will include a single pembrolizumab infusion during each cycle and taking olaparib tablets twice daily during each cycle. Total number of cycles to be completed in the maintenance phase are 8 cycles. Participants can expect to be in this study for about 6.5 years.

The purpose of this study is to develop and test if upper limb task practice and muscle activity training improve upper limb function in stroke survivors. Participants will be asked to come to the laboratory 3 times a week for 6 weeks to receive upper limb task practice and/or muscle activity training. Participants will also come to the laboratory for additional 3 visits for assessments of upper extremity function. The total duration of the study will be 2.5 months.

The need to advance knowledge and increase access to treatment for co-occurring posttraumatic stress disorder (PTSD) and substance use disorders (SUD) is a priority for service members and Veterans. PTSD and SUD often co-occur and are two of the most common and debilitating mental health disorders among service members and Veterans. However, few providers are trained in this approach and therefore are not prepared to deliver best practice care to Veterans who need integrated PTSD/SUD treatment.
This project will directly address this critical gap in provider training to improve the care of Veterans with comorbid PTSD/SUD, by providing development and testing of a web-based provider training course for concurrent treatment of PTSD and SUD Using prolonged exposure (COPE).

This study includes a longitudinal mixed methods study of 360 YYA with diabetes in SC, CO, and WA conducted with SFS 1 participants. SFS 2 will have two parts: (1) an intense, longitudinal study consisting of two 14-day assessment periods spaced 9 months apart which will include CGM, EMA, accelerometry, and surveys in all participants (the EMA study); and (2) a concurrent events qualitative study with a subset of participants (30 T1D, 15 T2D) who will complete 13 one-on-one, semi-structured interviews throughout the 9-month assessment period (the qualitative study).

This study aims to evaluate the efficacy and safety of lebrikizumab when used in combination with topical corticosteroid (TCS) treatment, compared with placebo, in pediatric participants with moderate-to-severe atopic dermatitis. Participants found to be eligible according to all of the study entry criteria will be randomly assigned in a 2:1 ratio to receive either lebrikizumab or placebo. This study can last up to 32 weeks, with 4 study periods. Screening Period: up to 4 weeks (≤30 days), TCS Standardization Period: 2 weeks, Treatment Period: 16 weeks, Post-Treatment Safety Follow-up Period: 12 weeks.

This small stroke rehabilitation study will test the feasibility of a new method to personalize a home exercise program for arm/hand recovery. The research study will last ~8 weeks. Participants will be included if they are adults who have experienced a stroke that has caused one arm/hand to become weak, are able to come to the MUSC main campus 3 times for ~2hr arm movement evaluations, and are willing and able to engage in 60 minute telerehabilitation video visits with an occupational therapist 1-2 times per week for 6 weeks. We anticipate that the results of this study will enable occupational therapists and stroke survivors to, together, design home exercise programs that are meaningful, motivating and effective.