Epilepsy
Study Name: STARS – Stand Together Against Prolonged Seizures
Study Design: Clinical research study evaluating an investigational treatment for prolonged seizures.
Study Stage: Actively Recruiting
Who Can Participate: Patients who experience prolonged epileptic seizures, such as:
- Focal seizures lasting 3 minutes or more
- Flurries of absence or myoclonic seizures lasting 5 minutes or more
- A focal or myoclonic seizure lasting at least 90 seconds that progresses into a generalized tonic-clonic seizure (total duration 3+ minutes)
Age Range: 18 years and older
Why Participate:
- Help advance treatment options for long-lasting seizures and contribute to epilepsy research.
- Participants may transition into the open-label phase to continue receiving the study drug
Principal Investigator: Vijayalakshmi Rajasekaran, MD
Clinical Trial ID: NCT05077904 (https://clinicaltrials.gov/study/NCT05077904)
Contact: Dane Prince, Email: princede2@upmc.edu, Phone: 2624906818
Study Name: X-ACKT: XEN1101 for Primary Generalized Tonic-Clonic Seizures
Study Design: Clinical research study evaluating an investigational treatment for generalized tonic-clonic seizures.
Study Stage: Actively Recruiting
Who Can Participate: Patients with primary generalized tonic-clonic seizures (PGTCS) that meet the following:
- Recurrent primary generalized tonic-clonic seizures (NO focal-onset)
- Experience ≥ 3 generalized seizures in the past 8 weeks
- Currently taking 1–3 antiseizure medications and stable for ≥1 month
Age Range: 18 years and older
Why Participate:
- Help advance treatment options for generalized seizures and contribute to epilepsy research.
- Participants may transition into the open-label phase to continue receiving the study drug
Principal Investigator: Vijayalakshmi Rajasekaran, MD
Clinical Trial ID: NCT05667142 (https://clinicaltrials.gov/study/NCT05667142)
Contact: Dane Prince Email: princede2@upmc.edu, Phone: 2624906818
Study Name: RISE 3: BHV-7000 in Focal (Partial) Epilepsy
Study Design: Clinical research study evaluating an investigational treatment for adults with focal-onset (partial) seizures.
Study Stage: Upcoming
Who Can Participate: Patients who experience focal onset seizures and meet the following:
- Experience ≥ 4 observable focal seizures within a month
- Currently taking 1–3 antiseizure medications and stable for ≥1 month
- Have been diagnosed with focal onset epilepsy for at least one year
Age Range: 18 to 75 years old
Why Participate:
- Contributing to research may advance new treatment options for people with difficult-to-treat focal seizures
- Participants may transition into the open-label phase to continue receiving the study drug
Principal Investigator: James Castellano, MD, PhD
Clinical Trial ID: NCT06309966 (https://clinicaltrials.gov/study/NCT06309966)
Contact: Dane Prince, Email: princede2@upmc.edu, Phone: 2624906818
Neuroimmunology/Multiple Sclerosis
Study Name: FREXALT- Master protocol of two independent, randomized, double-blind, Phase 3 studies comparing efficacy and safety of frexalimab (SAR441344) to teriflunomide in adult participants with relapsing forms of multiple sclerosis
Study Design: Clinical research study evaluating an investigational treatment for relapsing Multiple Sclerosis.
Study Stage: Actively Recruiting
Who Can Participate:
- The participant must have been diagnosed with RMS according to the 2017 revision of the McDonald diagnostic criteria
- The participant has an EDSS score ≤5.5 at the first visit
- The participant must have at least 1 of the following prior to screening:
- ≥1 documented relapse within the previous year OR
- ≥2 documented relapses within the previous 2 years, OR
- ≥1 documented Gd enhancing lesion on an MRI scan within the previous year
Age Range: 18-55 years
Why Participate:
- Help advance treatment options for MS and contribute to MS research.
- Participants may transition into the open-label phase to continue receiving the study drug
Principal Investigator: Ingrid Loma-Miller, MD
Clinical Trial ID: NCT06141473 (Study Details | NCT06141473 | Efficacy and Safety Studies of Frexalimab (SAR441344) in Adults With Relapsing Forms of Multiple Sclerosis | ClinicalTrials.gov)
Contact: Courtney Skasik, Email: skasikca@upmc.edu, Phone: 717-395-2614
Study Name: Prospective Investigation of Multiple Sclerosis in the Three Rivers Region (PROMOTE)
Study Design: Long-term, prospective, observational research study of multiple sclerosis
Study Stage: Actively Recruiting
Who Can Participate:
For MS patients:
- Diagnosis of multiple sclerosis or related disorders.
For healthy controls:
- No known personal history of multiple sclerosis or related disorders
- No other chronic diseases
Age Range: 7 years or older
Why Participate:
- Help researchers learn why MS affects people differently
- Contribute to developing more personalized treatments and care
Principal Investigator: Zongqi Xia, MD, PhD
Clinical Trial ID: NCT02994121 (https://clinicaltrials.gov/study/NCT02994121)
Contact: Zongqi Xia, Email: msstudy2@pitt.edu, Phone: 412-383-5377
Neuromuscular
Study Name: ALS/MND Natural History Study Data Repository
Study Design: The ALS/MND Natural History Data Repository/Registry is a multi-site multi-protocol clinic-based natural history study of ALS and other motor neuron disorders (MND). The study track’s the disease’s natural history in patients with ALS and/or MND through longitudinal data collected from clinic visits.
Study Stage: Actively Recruiting
Who Can Participate: All people with a confirmed diagnosis of ALS or other MNDs who attend a participating clinic are eligible for inclusion in the data repository.
Age Range: 18 years and older
Why Participate: Participation in this study will help researchers better understand the natural history of ALS and other motor neuron diseases (MNDs), including the wide variability in disease progression. This knowledge will help guide current treatment and care approaches and support future studies focused on identifying biomarkers for diagnosis and disease progression, as well as developing therapeutic interventions to improve patient care and health outcomes.
Principal Investigator: Tawfiq Al Lahham, MD
Clinical Trial ID: NCT05966038 (https://clinicaltrials.gov/study/NCT05966038)
Contact: Jacquelynn Krugh, Research Manager/Coordinator Email: jonesjr12@upmc.edu Phone: 412-864-2873
Study Name: A Global Prospective Observational Registry of Patients with Pompe Disease
Study Design: An Observational Registry to assess clinical outcomes in patients with Pompe disease, including patients with late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD), regardless of current or previous therapy. Data is collected regarding standard of care Pompe treatments as well as untreated Pompe patients directly from participants or from the medical record. No additional study activities are required, yearly patient questionnaires are optional.
Patients will be followed prospectively for at least 5 years from the date of signed informed consent.
Study Stage: Actively enrolling
Who can participate: Patients with late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD), regardless of current or previous therapy
Age Range: n/a
Why Participate: To contribute to the body of knowledge regarding “real-world” outcomes for people with Pompe disease, regardless of treatment status.
Principal Investigator: Paula Clemens, MD
Clinical Trial ID: NCT06121011 (Study Details | NCT06121011 | A Global Prospective Observational Registry of Patients With Pompe Disease | ClinicalTrials.gov)
Contact: Keira Fuener, Study coordinator Email: fuenerkr@upmc.edu Phone: 412-648-7324
Study Name: A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients with Late-Onset Pompe Disease.
Study Design: This is a non-interventional, multi-center, multi-national, study for adolescent and adult participants with Late-Onset Pompe Disease (LOPD) to characterize the occurrence of antibodies (total and neutralizing) to adeno-associated virus serotype 8 (AAV8) and acid alpha glucosidase (GAA).
Study Stage: Active, not recruiting
Who can participate: Participant is male or female with diagnosis of LOPD
Age Range: ≥ 16 years and < 70 years.
Why Participate: Help advance Adeno-associated virus (AAV)-based gene therapy for Pompe disease patients.
Principal Investigator: Paula R. Clemens, MD.
Clinical Trial ID: NCT06150820
Contact: Gabriela A. Niizawa, Research Manager I E-mail: niizawaga@upmc.edu I Phone: 412 383 9775
Study Name: ESPRIT - The first study of oral substrate reduction therapy for Pompe disease
Study Design: The ESPRIT study is to evaluate an investigational treatment (S-606001) in adult participants with Late-onset Pompe Disease (LOPD) as an add-on to ERT. The study will be conducted as a multinational, multicenter, randomized, double-blind, placebo controlled, parallel-group study. Approximately 45 participants with LOPD are planned to be enrolled in North America and Europe.
Study Stage: Actively Recruiting
Who can participate: Participants with LOPD:
- Currently receiving ERT
- Able to walk ≥ 75 meters. Use of assistive devices (eg, cane, walker, rollator) is allowed.
Age Range: ≥ 18 years and ≤ 65 years of age
Why Participate: Help advance treatment options for LOPD and contribute to Pompe disease research.
Participants may transition into the open-label phase to continue receiving the study drug
Principal Investigator: Paula R. Clemens, MD
Clinical Trial ID: NCT07123155
Contact: Gabriela A. Niizawa, Research Manager I E-mail: niizawaga@upmc.edu I Phone: 412 383 9775
Study Name: SAVANT (Safety assessment of AValgulucosidase Alfa as a Novel Treatment)
Study Design: This is a non-interventional, multicenter, longitudinal study of safety with prospectively collected data in participants with Pompe disease who are receiving enzyme replacement therapy (ERT) with avalglucosidase alfa.
Study Stage: Active, not recruiting
Who can participate: Participants with Pompe disease treated or planning to be treated with avalglucosidase alfa.
Age Range: All ages.
Why Participate: Help to understand the benefits and/or potential problems or side effects occurring from treatment with avalglucosidase alfa (Nexviazyme®).
Principal Investigator: Paula R. Clemens, MD.
Clinical Trial ID: NCT06150820
Contact: Gabriela A. Niizawa, Research Manager I E-mail: niizawaga@upmc.edu I Phone: 412 383 9775
Study Name: Pompe Disease Registry
Study Design: The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.
Study Stage: Actively enrolling
Who can participate: All patients with a confirmed diagnosis of Pompe disease. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Age Range: n/a
Why Participate: To contribute to the body of knowledge regarding “real-world” outcomes for people with Pompe disease, regardless of treatment status.
Principal Investigator: Paula Clemens, MD
Clinical Trial ID: NCT00231400 (Study Details | NCT00231400 | Pompe Disease Registry Protocol | ClinicalTrials.gov)
Contact: Keira Fuener, Study coordinator Email: fuenerkr@upmc.edu Phone: 412-648-7324
Study Name: A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of remibrutinib in patients with generalized Myasthenia Gravis, followed by an open-label extension phase
Study Design: Clinical research to evaluate the efficacy, safety and tolerability of remibrutinib in patients with generalized Myasthenia Gravis (gMG), who are on stable standard-of-care treatment. Trial will include a double-blind 6-month study drug vs placebo phase, followed by a 60-month open label phase. Remibrutinib or placebo will be taken in pill form twice a day for the duration of the study treatment period.
Study Stage: Active
Who can participate: Adult patients with gMG, who meet eligibility requirements regarding current health status and stable standard treatments for gMG. Subjects must be able to swallow pills and agree to use of highly effective birth control while on study.
Age Range: 18-75 years old
Why Participate: To contribute to knowledge regarding the safety and efficacy of a potential new treatment for gMG.
Principal Investigator: Fang Sun, MD
Clinical Trial ID: NCT06744920 (Study Details | NCT06744920 | A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis | ClinicalTrials.gov)
Contact: Keira Fuener, Study coordinator Email: fuenerkr@upmc.edu Phone: 412-648-7324
Study Name: A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients with Generalized Myasthenia Gravis (MG)
Study Design: This is a randomized, double-blind, placebo-controlled, multi-center phase 3 trial to evaluate the efficacy, safety and tolerability of autologous T-cells expressing a chimeric antigen receptor (CAR) directed to B-Cell maturation antigen (BCMA) in patients with antibody positive gMG. The cell product (Descartes-08) will be infused in weekly infusions for 6 weeks, and Myasthenia Gravis symptoms will be assessed at prescribed time points for follow-up.
Individual participant duration lasts approximately 14 months: 6 months in Part 1 and 8 months in Part 2. Part 1 is composed of 2 months of screening followed by 4 months of double-masked treatment, Part 2 is open-label treatment for 6 weekly infusions with follow-up until Month 8.
Study Stage: Active, enrollment closes on June 12, 2026.
Who can participate: Adult patients with gMG, who have Acetylcholine receptor autoantibody (anti-nAChR) titer or anti-AChR cluster antibody above the reference laboratory upper normal limit (UNL) and who meet eligibility requirements regarding current health status and stable standard treatments for gMG.
Age Range: Must be at least 18 years old
Why Participate: To contribute to knowledge regarding the safety and efficacy of a potential new treatment for gMG.
Principal Investigator: Fang Sun, MD
Clinical Trial ID: NCT06799247 (Study Details | NCT06799247 | Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis | ClinicalTrials.gov)
Contact: Keira Fuener, Study coordinator Email: fuenerkr@upmc.edu Phone: 412-648-7324
Movement Disorders
PARKINSON’S DISEASE (PD) STUDIES:
Parkinson’s Progression Markers Initiative (PPMI)
Sponsor: Michael J Fox Foundation
Site PI: Lana Chahine, MD
Coordinator: Sherri Mosovsky, MPH, CPH
Objective: To identify biomarkers that can track the progression of Parkinson's disease to help develop and test new therapies.
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
SPARX3
Sponsor: NIH/NINDS
Site PI: Alexandra B. Gil, PT, PhD
Coordinator: Deena Ratner, MPPM
Description: Phase 3, multi-site, randomized, evaluator-masked, study of endurance treadmill exercise on changes in the Movement Disorder Society-Unified Parkinson Disease Rating Scale (MDS-UPDRS) Part III score at 12 months among persons with early-stage Parkinson’s disease.
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
PD Gene Therapy Trial
Sponsor: Ask Bio
Site PI: Valerie Suski, DO
Coordinator: Sherri Mosovsky, MPH, CPH
Description: Phase 2, Open-Label, Multi-Stage Study to Optimize the Intraputaminal Administration of AB-1005 (AAV2-GDNF) Using a Prescriptive Infusion Algorithm (PIA).
Candidates: PD diagnosis of over 10 years
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
Antioxidant, gamma-glutamylcysteine (GGC) in PD
Sponsor: University of Pittsburgh
Site PI: Pravat Mandal, PhD and Abby Olsen, MD, PhD
Coordinator: Nazia Abdul Rasheed Pillar, clinic55@pitt.edu
Goal: To investigate the effect of the antioxidant GGC, a precursor to glutathione, on brain health, motor function, oxidative stress, and cognitive function.
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
HUNTINGTON’S DISEASE (HD) STUDIES:
ENROLL-HD
Sponsor: CHDI
Site PI: Valerie Suski, DO
Coordinator: Patricia Conlon, BSOT
Description: A prospective Registry Study in a Global HD Cohort
Candidates: Gene positive and Gene Unknown patients
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
GENERATION-HD2
Sponsor: Roche
Site PI: Valerie Suski, DO
Coordinator: Patricia Conlon, BSOT
Description: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding, Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals with Prodromal and Early Manifest Huntington’s Disease.
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
SPK-1001
Sponsor: Roche
Site PI: Valerie Suski, DO
Coordinator: Patricia Conlon, BSOT
Description: A Phase I/II, Randomized, Sequential, Dose Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of a One-Time Bilateral, Intraparenchymal Infusion of SPK-1001 into the Caudate and Putamen in Participants with Huntington’s Disease.
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
MULTIPLE SYSTEM ATROPHY (MSA) STUDIES:
TOPAS
Sponsor: TEVA
Site PI: Valerie Suski, DO and Abby Olsen, MD, PhD
Coordinator: Deena Ratner, MPPM
Description: A Multi-Centered, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Phase 2 Study of TEV-56286 (emrusolmin) for the Treatment of Patients with Multiple System Atrophy.
Candidates: clinical probable and clinical possible MSA-C and MSA-P
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
MASCOT
Sponsor: Lundbeck
Site PI: Valerie Suski, DO and Abby Olsen, MD, PhD
Coordinator: Deena Ratner, MPPM
Description: Phase 3, Interventional, Randomized, Double-Blind, Placebo-Controlled, Optional Open-Label Extension Trial of Lu AF82422 (amlenetug) in Participants with Multiple System Atrophy.
Candidates: clinical probable and clinical possible MSA-C and MSA-P
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
LEWY BODY DISEASE
BEHOLD (BioEnergetic Hallmarks of Lewy Body Dementia)
Sponsor: National Institute on Aging (National Institutes of Health)
PI: Sarah Berman, MD, PhD
Coordinator: Jan Ciccozzi
Description: Lewy Body Dementia (LBD) study of the involvement of mitochondrial and bioenergetic dysfunction in LBD.
Candidates: Lewy Body Dementia, REM Sleep Behavior Disorder (RBD), Mild Cognitive Impairment, and Alzheimer’s disease
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
Dementia with Lewy Bodies (DLB) Consortium
Sponsor: National Institute on Aging (National Institutes of Health)
Site PI: Sarah Berman, MD, PhD
Coordinator: Cary Zik
Description: Longitudinal study of DLB and Parkinson’s Disease Dementia
Candidates: Parkinson Disease Dementia, Dementia with Lewy Bodies (DLB), Mild Cognitive Impairment with Lewy Bodies
If interested, please email mosovskysl@upmc.edu or call 412-692-4659
Stroke
Study Name: The Pivotal Study of RapidPulseTM Aspiration System as Frontline Approach for Patients with Acute Ischemic Stroke due to Large Vessel Occlusions (“RapidPulse Pivotal Study”)
Study Design: Global, multi-center, single-arm, prospective, adaptive, open-label clinical study against a literature derived performance goal (PG).
Study Stage: The RapidPulse™ Aspiration System (RPAS) is an investigational device that is designed to remove occlusive thrombus from the cerebral vasculature using precisely pulsed aspiration. RapidPulse™ Aspiration System is intended to improve revascularization results during aspiration thrombectomy in patients with acute ischemic stroke.
Device trials are not staged.
Who Can Participate: Male and females, ages 18 to 80 years old, presenting with acute ischemic stroke in the anterior and posterior cerebral circulation, that can be treated within 8 hours of AIS symptom onset using the current thrombectomy devices. Participants should be generally healthy prior to the stroke.
Age Range: Male and females, ages 18 to 80 years old.
Why Participate: To demonstrate the safety and effectiveness for the RapidPulseTM Aspiration System in the treatment of large vessel occlusions in patients experiencing an Acute Ischemic Stroke.
The direct aspiration first-pass technique (ADAPT) is being increasingly used in current clinical practice and involves the use of large-bore aspiration catheters that are guided to the proximal end of the thrombus and then put under negative pressure (by using vacuum aspiration systems or a syringe) to produce suction on the thrombus. The clot is thus trapped at the tip or aspirated through the aspiration catheter. The aim of this study is to therefore investigate the safety and effectiveness of endovascular treatment of Acute Ischemic Stroke using the RapidPulseTM Aspiration System with the ADAPT technique.
Site Principle Investigator: Bradley Gross, MD
Global Principle Investigator: Raul Nogueira, MD
Clinical Trial ID: NCT 06029491, https://rapidpulsemed.com/clinical-trials
Contact: Abdullah Al Qudah, MD, Email: alqudaham@upmc.edu
Study Name: ATHENA – A Prospective, Randomized, Dual-Arm, Multi-center Study to Assess the Safety and Effectiveness of Mechanical Thrombectomy Using the ANACONDA ANA5 Device in Combination with a Stent Retreiver
Study Design: Prospective, randomized, dual-arm, multi-center study. Randomization to either ANA or Control will be performed in a 1:1 ratio.
Study Stage: Enrollment pause as of 25 October 2025
Who Can Participate:
- Acute cerebral ischemia
- Indicated for neurovascular thrombectomy procedure
- NIHSS ≥ 8 and ≤ 25
- Treatable within 24hrs of stroke onset
- M1 or dominant or proximal M2 occlusion
- Pre-ictal mRS 0-2
- CT ASPECTS 6-10
Age Range: 22-85
Why Participate: Help to demonstrate that the use of ANA does not adversely impact the safety and effectiveness of a conventional stent retriever MT procedure
Principle Investigator: Alhamza Al-Bayati, MD
Clinical Trial ID: NCT 06370182
Contact: Danielle Moss, Email: mossdn@upmc.edu, Phone: 878-261-6389
Study Name: RESPECT-ETB – A multicentric, randomized, doub;e-blind, parallel, placebo-controlled phase III study to assess the safety and efficacy of sovateltide in patients with acute cerebral ischemic stroke
Study Design: This is a multicentric, randomized, double-blind, parallel, placebo-controlled phase III clinical study
Study Stage: Actively enrolling
Who Can Participate:
- Stroke ischemic in origin
- NIHSS ≥8 and < 20
- First dose given within 24hrs of stroke onset
- Not receiving any endovascular therapy or surgical intervention
Age Range: 18-80
Why Participate: Help with treatment options by adding a therapeutic in the market that repairs cerebral damage and alleviates neurological deficit
Principle Investigator: Jussie Correia Lima, MD
Clinical Trial ID: NCT 05691244
Contact: Danielle Moss, Email: mossdn@upmc.edu, Phone: 878-261-6389
