Clinical Trials

Clinical Trials

Clinical Research Trials at Holy Cross 

Currently, motor neuron diseases including ALS and PLS do not have a cure. The Phil Smith Neuroscience Institute at Holy Cross understands the importance of providing research and trial opportunities for our patients. Research is crucial to advance the science and develop effective drug and treatment options for motor neuron disease. Here at the Phil Smith ALS Clinic, we participate in various types of research, and are happy to discuss research options during your clinic visit. 

Holy Cross Health is a participant in the Healey ALS Platform Trial, an initiative led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. A "platform trial" is a clinical trial in which multiple treatments are evaluated simultaneously. New treatments are added as they become available, thereby decreasing the gap in time from identification of a therapy to testing. This model, already proven successful in the cancer field, will greatly accelerate development of effective and breakthrough treatments for people with ALS by allowing researchers to test more drugs, increase patient access to trials, and reduce the cost by quickly and efficiently evaluating the effectiveness of multiple therapies. The Healey Center platform trial is the first for ALS in the US and has enrolled participants faster than any ALS trial before. Lead Investigators: Gustavo Alameda, MD, FAAN & Nithya Mathai, DNP,APRN,FNP-C.

Current Active Trials

Healey Platform Trials regimens:

  • A- Zilucoplan - CLOSED 
  •  B- Verdiperstat - CLOSED 
  •  C- CNM-Au8 GOLD - CLOSED 
  •  D- Pridopidin - CLOSED 
  •  E- Trehalose (IV) Infusion- ACTIVE
    • *Trehalose EAP – for patients who do not qualify for Healy Platform Trial. 
  • F- Enrolling
  • G- Enrolling

PTC Trial – Enrolling

BIOGEN 303 SODl gene and families - This study is looking for ALS subjects and their family members who have tested positive for the SOD1 gene. If someone has a family history of ALS, we would be more then happy to test them for this SOD1 gene.

Trehalose EAP access - sod1 patients only.

CLENE EAP- Enrolling.

Observational Studies:

Observational Studies are research opportunities that allow for persons living with ALS to contribute to helping to find a cure for ALS. There is no drug involvement in observational trials. 

LabPals: Enrolling - Testing for Biomarkers in the persons living with ALS 

Precision Labs: Enrolling - Testing biomarkers in blood and csf specimens in persons living with ALS.

Research Contacts 

Ashley Stepler | - Lead Research Coordinator
Ph: 954-542-3442

Silenny Mejia-Almerio, BS | – Research Coordinator
Ph: 954-542-3443