Enrolling

Full Study Name: Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis 

Purpose: This expanded access research study is being done to determine if RAPA-501 cells can be safely administered to participants with high-risk ALS and evaluate whether RAPA 501 might result in a therapeutic effect to slow progression of disease.

Participants: Adult pariticpants with a diagnosis of ALS. Patients cannot be eligibile for the Phase 2/3 Rapa-501 study, including SVC < 50%.

Study Length: This study will have a screening period (28 days or less). If eligible, you will undergo an apheresis procedure within 28-days in order to collect immune cells from your blood that are needed to manufacture the RAPA-501 cell therapy. It will take approximately one-month from the apheresis date to manufacture the RAPA-501 cell therapy and begin your RAPA-501 cell infusions. Once you start the RAPA-501 cell therapy, the treatment and follow-up period will be approximately 8-months. Therefore, the total amount of time that you may be involved in the research is approximately eight months (spanning from the beginning of screening until the end of follow-up).

There are 7 on-site study visits and 6 virtual visits.

Sponsor: Rapa Therapeutics

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study - up to $1500 total.

Principal Investigator: Laura Rosow, MD

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The HEALEY Platform Trial is a perpetual adaptive trial. Future regimens will be added as a new study drugs become available. Register for the Community Q&A Webinars to stay connected to trial news and updates.  

Learn more about the platform trial.

Current Active Regimens - No Longer Enrolling 

Regimen F: Trial of ABBV-CLS-762 (No Longer Enrolling, Enrollment Goals Met)
Developed by Calico in collaboration with AbbVie, ABBV-CLS-7262 targets eIF2B, a key regulator of the integrated stress response (ISR), a pathway activated in people with ALS. In neurons exposed to cellular stressors, inhibition of the ISR by ABBV-CLS-7262 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules. This effect of ABBV-CLS-7262 is of clinical interest because TDP-43 containing stress granules are thought to lead to TDP-43 inclusions, a hallmark of ALS pathology.

Regimen G: Trial of DNL343 (No Longer Enrolling, Enrollment Goals Met)
DNL343 is a novel investigational ALS therapy that targets eIF2B, a central regulator of the integrated stress response (ISR). The ISR appears to be overactive in ALS, leading to the formation of stress granules containing TDP-43. Buildup of TDP-43 is harmful and leads to neuronal degeneration. In the lab, inhibition of the ISR by DNL343 dissolves TDP-43 containing stress granules and decreases ISR biomarkers. The safety, pharmacokinetics, and pharmacodynamics of DNL343 have been characterized in both healthy participants and people with ALS, in a Phase 1 (N=47) and a Phase 1b (N=29) study, respectively, with dosing for up to 28 days. Results from both studies demonstrated that once-daily oral dosing with DNL343 was generally well tolerated and exhibited extensive Cerebrospinal Fluid (CSF) penetration. In addition, robust inhibition of biomarkers associated with the ISR pathway was observed in blood samples from study participants.

No Longer Enrolling

Regimen A: Trial of Zilucoplan 
Developed by UCB

Regimen B: Trial of Verdiperstat
Developed by Biohaven Pharmaceuticals

Regimen C: Trial of CNM-Au8
Developed by Clene Nanomedicine

Regimen D: Trial of Pridopidine
Developed by Prilenia Therapeutics

Regimen E: Trial of Trehalose (SLS-005)
Developed by Seelos Therapeutics

Learn more about the study drugs

Enrolling

Full Study Name: A Phase 1/2 Study to Evaluate the Safety, Pharmacokinetics and Biodistribution of an Imaging agent, 18F-OP-801 (18F Hydroxyl
Dendrimer), After Intravenous Administration to Patients with Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease (AD), Multiple Sclerosis (MS), Parkinson’s Disease (PD), and Healthy Volunteers (HV)

Purpose: This research study is designed to test an investigational drug, OP-801. ‘Investigational’ means that OP-801 has not been approved by the FDA as an imaging agent. OP-801 is an imaging agent used to detect inflammation in the central nervous system in patients with nervous system disorders such as ALS.

Participants: Up to 51 people will take part in this study at approximately 5 study centers. At the beginning of the study, 5 healthy volunteers will be enrolled. If the drug does not cause bad side effects, 10 ALS patients and 10 age-matched healthy volunteers will be enrolled. A total of 10 ALS patients is the most that would be able to enter the study across all sites. There will also be 10 AD (Alzheimer’s Disease), 15 MS (multiple sclerosis), and 1 PD (Parkinson’s Disease) enrolled across all centers. UCSF will enroll up to 20 subjects.

Study Length: If you choose to be in this study and are determined eligible, you will be enrolled to receive one dose of the study drug on Day 1. In addition, the main study procedures include medical history review, physical exams, blood draws, MRI, administration of OP-801, and PET scans. You will be in this study approximately 75 days and visit the research site approximately 3 times.

Sponsor: Ashvattha Therapeutics, Inc.

Compensation: In return for your time and effort, you will be paid up to $2200 for taking part in this study. You will receive $600 for completing the screening visit, $1200 for completing the Day 1 dosing visit, and $400 for completing the follow up visit.

Principal Investigator: Vinil Shah, MD

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Upcoming

Full Study Name: A phase 2, randomized, double-blind, placebo-controlled parallel group study of VHB937 in Amyotrophic Lateral Sclerosis (ALS) over 40 weeks followed by an Open-label Extension (ASTRALS)

Purpose: The purpose of the study is to find out whether the study treatment VHB937 is safe and slows disease progression in people with ALS who have had symptoms for no more than 2 years.

Participants: Adult participants with ALS. First signs of muscle weakness < 24 months, ALSFRS ≥ 30, SVC ≥ 60%

Study Length: Unless you decide to stop study treatment, you will be in the study as long as your Study Doctor feels that you are benefiting from your participation in the study or the study is cancelled. Before starting study treatment (screening period), you will visit the Study Doctor up to 3 times. Once you start the study treatment, you will visit the site about 11 times over the initial 40 weeks. During the study you will receive an intravenous infusion of study treatment every 4 weeks, while your safety and disease progression will be assessed every 12 weeks. If you decide to stop study treatment, you may continue visiting the site every 12 weeks until you decide to stop these visits. After that point, your Study Doctor will still continue checking on your health status via phone calls every 12 weeks to you, your relatives, or your personal doctor.

Sponsor: Novartis

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study.

Principal Investigator: Jill Goslinga, MD, MPH

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Enrolling

Full Study Name: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2‑Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy

Purpose: The purpose of this study is to look at the effect (efficacy) and the safety of a drug called “efgartigimod” in people with active idiopathic inflammatory myopathy, commonly known as myositis. People with the disease subtypes of dermatomyositis, immune-mediated necrotizing myopathy, and polymyositis (including antisynthetase syndrome) will be included in the study.

Participants: Adults subjects with IIM with active disease. Must be receiving permitted background treatment for IIM, including oral corticosteroids and/or immunosuppressants.  Participants cannot be recieving IVIg or PLEX.

Study Length: The study will last about 66 weeks.

You will visit the study site about 15 times over 66 weeks. At these visits, you will have a physical examination and an electrocardiogram, blood will be drawn about 636 milliliters during the study, plus an optional 10 milliliters for future biomarker research on markers related to myositis, and another optional 14 milliliters for testing about the role of “interferons” in myositis, and you will fill out some questionnaires and complete tests to check your myositis symptoms. Sometimes, a urine sample will be collected. You will spend about 3 to 4 hours at the study site for visits. The examinations performed may vary.

During this time, you will be required to have visits to assess your health, disease, and study treatment 17-18 times. These visits may be in the clinic or, if it is allowed by your Study Doctor, some of these visits may be able to take place in your home or at another site with a study nurse/study health care provider coming to see you rather than you having to go to the clinic. If you have home visits, there may be use of telehealth during the visits.

Sponsor: argenx BV

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study.

Principal Investigator: Min Kang, MD

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Upcoming

Full Study Name: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA‑201) in Participants with Generalized Myasthenia Gravis

Purpose: This study is being done to test a drug called CABA-201. We will test CABA-201 with people who have generalized myasthenia gravis (MG). CABA-201 is a personalized therapy that is made from your own T cells, atype of white blood cell in your immune system. This type of cell therapy is also known as chimeric antigen receptor T (CAR T) cell therapy. In this study, we will collect some T cells from your blood intravenously (through your vein) and genetically change them to find and remove the B cells in your body, including the B cells that are involved in causing your disease. These new T cells will then be re infused into your body intravenously. The study team is trying to determine the safety of CABA-201 in treating your condition.

Participants: Adult participants with generalized Myasthenia Gravis aged ≥18 and ≤70 years. Patients cannot be treated with Eculizumab or Rituximab within specified timeframes prior to screening.

Study Length: The screening, pre-treatment, treatment, and post-treatment follow-up portions of the study will take place over 3 years. The long-term safety follow-up portion will last for at least an additional 12 years after the post-treatment follow up. In total, the study will last for at least15 years. You will be asked to attend about 46 visits over the course of the entire study, but this may vary depending on circumstance. There will only be one infusion of the study drug, and it will occur on site. The CABA-201 infusion will be given in the hospital , and you will remain hospitalized for at least 10 days following the infusion. Long-term follow-up visits may be conducted remotely if a previous in-person visit has determined that there are no more CABA-201 cells left in your body, otherwise they will be done in-person until there are no more remaining CABA-201 cells.

Sponsor: Cabaletta Bio, Inc

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study.

Principal Investigator: Min Kang, MD

Active - Closed to Enrollment

Full Study Name: A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants with Generalized Myasthenia Gravis (gMG)

Purpose: The purpose of this study is to learn how well batoclimab works and how safe it is, when compared with placebo (an inactive material that looks like batoclimab but does not have any active drug). 

Participants: gMG, being treated or have been treated with corticosteroids and/or cholinesterase inhibitors OR non-steroidal immunosuppressants,

Study Length: If you choose to enter this study, you will participate in the study for up to approximately 84 weeks (1 ½ years) and you will be asked to attend up to 34 study visits. The study includes a screening period of up to 5 weeks, two 12-week treatment periods (referred to as Period 1 and Period 2), and a 52-week long-term extension period (referred to as Period 3).

Sponsor: Immunovant Sciences GmbH

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study.

Principal Investigator: Min Kang, MD

Active - Closed to Enrollment

Full Study Name: A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of ABC008 in the Treatment of Subjects with Inclusion Body Myositis

Purpose: Inclusion Body Myositis or IBM is an inflammatory autoimmune disorder of skeletal muscle resulting in progressive limb weakness and loss of function. This means harmful immune cells attack muscle, resulting in muscle weakness. These harmful immune cells have a molecule on their surface called KLRG1. The study drug ABC008 has been designed to bind to KLRG1 and then to reduce the number of these harmful immune cells present in people with IBM.

Participants: Adults subjects diagnosed with IBM 40 years or older. Able to arise from a standard armchairwith use of arms but without support from another person or device.

Study Length: Your participation in this study will last about 85 weeks, with an extended period of evaluation that can vary. This includes a screening period of up to 5 weeks, a study treatment period of up to 76 weeks, and a 4-week follow-up visit for both Part A and Part B.

During this time, you will be required to have visits to assess your health, disease, and study treatment 17-18 times. These visits may be in the clinic or, if it is allowed by your Study Doctor, some of these visits may be able to take place in your home or at another site with a study nurse/study health care provider coming to see you rather than you having to go to the clinic. If you have home visits, there may be use of telehealth during the visits.

Sponsor: Abcuro, Inc.

Compensation: You will not be paid for being in this study; however, you will receive reasonable reimbursement for travel expenses as required for each study visit during your participation in this study.

Principal Investigator: Laura Rosow, MD

Full Study Name: A Phase 3b, multicenter, open-label, single-arm study to evaluate the safety, tolerability, and efficacy of zilucoplan in participants with generalized myasthenia gravis switching from intravenous complement component 5 inhibitors to subcutaneous zilucoplan

Purpose:  The purpose of this study is to assess the safety, tolerability (body’s ability to handle), and the effectiveness of the study drug, zilucoplan, when administered after switching from IV treatment with C5 inhibitors. The study drug will be administered as a subcutaneous (SC) (under the skin) injection.

Participants: gMG, Patients need to be switching from IV complement component C5 inhibitor.  Patients receive eculizumab or ravulizumab up until screening and receive their final dose at the screening visit – then wait 2-8 weeks for baseline

Study Length: The study consists of the following periods:

• Screening Period (up to 2 weeks or 8 weeks, depending on the C5 inhibitor the participant has been on treatment with): 2-week Screening period for study participants switching from eculizumab or 8-week Screening period for study participants switching from ravulizumab
• Main Study Treatment Period (12 weeks) including a Baseline Visit on Day 1
• Extension Study Treatment Period (optional) after the Main Study Treatment Period until zilucoplan may be available by prescription or until further communication by the sponsor.
• Safety Follow-up (SFU) Visit (40 days after the last dose of study drug)

Sponsor: UCB Bio

Principal Investigator: Min Kang, MD 

Full Study Name: An open-label, crossover study to evaluate rozanolixizumab self-administration by study participants with generalized myasthenia gravis.

Purpose:  Phase 3 crossover study to evaluate self-administration of rozanolixizumab by study participants with gMG

Participants: gMG, has myasthenic crisis or impending crisis

Study Length: 19 weeks, majority visits in-person. 6 weeks dosing at home. SC self-administration of Rozanolixizumab weekly.

Sponsor: UCB Bio

Principal Investigator: Min Kang, MD 

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