Observational Studies

Enrolling

Full Study Name: Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) Data Hub Protocol

Purpose:  The purpose of this study is to collect information about individuals with neuromuscular disease to better understand the disease progression and ultimately improve the medical care, quality of life, and survival of those with neuromuscular disease.

Participants: Participants of any age with ALS, DMD, BMD, FSHD, LGMD, Pompe, and SMA. Must be a patient at UCSF. 

Study Length: If you decide to take part in the MOVR Data Hub, information will be collected about you, your health, and other potentially relevant information by the MDA Care Center. Each person who participates in the MOVR Data Hub will have his/her information collected for as long as the person is being seen at an MDA Care Center and information is still being collected, unless and until the person requests that the information no longer be provided to the MOVR Data Hub.

Sponsor: Muscular Dystrophy Association (MDA)

Compensation: You will not be paid for being in this study.

Principal Investigator: Jill Goslinga, MD, MPH

I'm Interested

Enrolling

Full Study Name: Characterizing Microbial Markers Predictive for ALS Onset and Progression (ALS-CMOP)

Purpose:  This study is evaluating whether certain microbial species, genes, and metabolites from the gut microbiome may contribute to an increased risk and more rapid progression of ALS. Patients enrolled in this study will also be simultaneously enrolled in MOVR. 

Participants: Patients with ALS, their spouse (or other adult that shares a living space with the patient), and georgraphically-matched healhty volunteers will be able to join this study. 

Study Length: The study includes 2 visits. The first will be a zoom or phone call to go over the consent form and answer any questions you may have regarding the study. If you agree to participate, we will send you a stool sample collection kit to your home. The second visit will be a zoom or phone call during which we will ask you questions regarding your lifestyle and diet, and you will collect a stool sample the same day. You will not receive any new or experimental treatments as part of the study. While active participation will take about 2 hours of your time, we will continue to access your medical records to track your progression indefinitely.

Sponsor: Department of Defense

Compensation: In return for your time and effort, you will be provided a total of $50 in Amazon gift cards.
You will receive a $25 Amazon gift card for completing the lifestyle questionnaire with a study team member and you will receive a $25 Amazon gift card once we receive your stool sample.

Principal Investigator: Catherine Lomen-Hoerth, MD, PhD

I'm Interested

Enrolling

Full Study Name: Stratifying sALS patients-using Molecular-, cellular-, and physiological profiling-for personalized treatment matching and drug development (COMBINE ALS)

Purpose:  In this study, the researchers are collecting blood samples and skin biopsy samples to learn more about subgroups of the Amyotrophic Lateral Sclerosis (ALS) patient population. Patients enrolled in this study will also be simultaneously enrolled in MOVR. 

Participants: Persons with a diagnosis of ALS and healthy volunteers will be asked to participate in this study. Patients must be seen at UCSF.

Study Length: You would be in this study for about 5 years and visit the research site 4 times per year of your study participation. You will be asked to wear daily a Garmin watch provided to you for the duration of the study.

Compensation: You will not be paid for being in this study.

Sponsor: Target ALS

Principal Investigator: Catherine Lomen-Hoerth, MD, PhD

I'm Interested 

Enrolling

Full Study Name: ASSESS ALL ALS – Longitudinal Biomarker Study for Symptomatic ALS and Healthy Control Participants  

Purpose:  To study people diagnosed with ALS and healthy participants to further our understanding of the disease and potential biomarkers of disease progression. The information collected in this study may contribute to future research and development of new treatments for ALS and similar neurological diseases. 

Participants: People with ALS and healthy volunteers 

Study Length: up to 2 years (7 in person or remote visits) 

Sponsor: National Institutes of Health

Principal Investigator: Laura Rosow, MD

I'm Interested

Enrolling

Full Study Name: PREVENT ALL ALS – Longitudinal Biomarker Study for Participants Who are At Risk for ALS

Purpose:  To study people at risk for developing ALS and broaden our understanding of causes of underlying early disease changes. The information collected in this study may result in the development of treatments that target the earliest changes in ALS and lead to possible disease prevention.

Participants: People who are asymptomatic ALS gene carriers or have a family history of ALS

Study Length: up to 3 years (6 remote visits/3 yearly in-person visits)

Sponsor: National Institutes of Health

Principal Investigator: Laura Rosow, MD

I'm Interested​

Active - Enrolling 

Full Study Name: A Global Real-World Study to Assess HyQvia Use and Outcomes in Patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Switching to HyQvia

Purpose:  The purpose of this research is to collect real world information regarding people with Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who are currently or plan to start taking HyQvia®.  

Participants: Adult participants with CIDP who have had a stable disease course for at least 12 weeks. Participants will either have just switched to HyQvia (within 6 weeks prior to screening), or plan to start HyQvia within 6 weeks after screening.

Study Length: We expect that your taking part in this research will last 12 months. Your study visits will coincide with your regular clinic visits.

Compensation: You will not be paid for being in this study.

Sponsor: Takeda Development Center Americas, Inc

Principal Investigator: Pritikanta Paul, MD

I'm Interested​

Active - Enrollment Closed

Full Study Name: A Multicenter Prospective Longitudinal Study of Clinical Outcomes, Disease Course, Health-Related Quality of Life, and Health Care Resource Utilization in Adult Patients With Multifocal Motor Neuropathy

Purpose:  The purpose of the study is to provide a detailed view of the impact of Mulitfocal Motor Neuropathy (MMN) and MMN treatment on patients in the real world. This protocol aims to collect data from participants with MMN to characterize the disease’s course and management and the humanistic and economic burden on patients.

Participants: Patients who have a diagnoisis of MMN and are not currently participating in a clinical trial

Study Length: Participants will be followed for 2 years and complete questionnaires every 3 months at their regularly scheduled clinic visits.

Compensation: You will not be paid for being in this study.

Sponsor: argenx BV

Principal Investigator: Madina Tugizova, MD

Enrolling

The brachial neuritis registry is a prospective registry to understand the natural progression of brachial neuritis and its impact on quality of life. If appropriate, patients can be contacted to participate in clinical trials.

Eligibility: 

• Dx of Brachial Neuritis
• Seen at UCSF Brachial Neuritis Center

I'm Interested

Full Study Name: PLS Natural History Study

Purpose:  The purpose of this study is to develop a natural history dataset and biorepository of early PLS and well-established PLS cases for future clinical trials.

Participants: Diagnosis of Primary Lateral Sclerosis (PLS), adults ≥ 25 years old, ability to independently walk without an assistive device at Baseline visit, symptoms that started within the last 15 years, no history of ALS or PLS in the immediate family and no family history of hereditary spastic paraplegia (HSP)

Study Length: The study will take 2 years and include 3 in-person study visits at UCSF Mission Bay and 3 telephone calls. 

Sponsor: The ALS Association (ALSA), Mitsubishi Tanabe Pharma, The Spastic Paraplegia Foundation (SPF)

Principal Investigator: Catherine Lomen-Hoerth, MD, PhD