The MDA/ALS Center of Hope provides an important setting for research towards understanding, treating, and curing ALS. That is why the ALS Hope Foundation supports an extensive clinical research program at the Center of Hope. Without research, there would be no progress in our fight against ALS. Clinical Research is a broad term, encompassing clinical trials (studies on the effect of a medicine on the disease or its symptoms) and clinical research that examines other aspects of the illness, such as assistive technology or quality of life. Clinical research at the Center complements our basic research program, in the hope that promising therapeutics identified at the lab bench can be tested for efficacy in the clinic.
For more information on the clinical trials being conducted at Temple's ALS clinic, please contact our Research Nurse Coordinator, Kathleen Hatala, via email at Kathleen.Hatala@tuhs.temple.edu.
To learn more about each of these trials, please go to clinical trials.gov and search for ALS trials.
Clinical Research: Ongoing Projects
Cytokinetics CY5022 FORTITUDE
FORTITUDE-ALS is a Phase 2, double-blind, randomized, placebo-controlled, parallel group, dose ranging study of CK-107 in patients with ALS. The trial will enroll approximately 450 patients with ALS in the US and Canada. The primary efficacy endpoint is the change from baseline in the percent predicted SVC at 12 weeks. CK-2127107 is a small molecule activator of the fast skeletal muscle troponin complex, a sarcomere-directed therapy intended to improve skeletal muscle function in conditions associated with muscle weakness and/or fatigue. Although derived from a different structural class, it has the same mechanism of action as tirasemtiv (CK-2017357). Learn more about Cytokinetics.
Amylyx AMX0035 CENTAUR
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum. The drug is a combination of two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA). Each compound has exhibited strong efficacy in several cellular and animal models of ALS. Furthermore, PB and TUDCA have been individually tested in clinical trials of ALS and both showed safety and tolerability and preliminary signs of efficacy. In preclinical trials, Amylyx has shown a synergistic effect between the two compounds, suggesting that the combination may be more effective in people with ALS than either compound alone. The trial will test the safety and tolerability of AMX0035, as well as functional outcomes. Analysis of biomarkers of cell function, neuronal damage, and inflammation will be included as a major part of the trial, along with a new measure of muscle strength that has been shown to be tightly correlated with disease progression.
Mallinckrodt Acthar PENNANT
This Phase 2B clinical study will enroll patients ages 18 to 75 with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ two years prior to the screening visit. Subjects will be randomized on a 2:1 basis to receive subcutaneous H.P. Acthar Gel daily or matching placebo daily for 36 weeks. The efficacy of H.P. Acthar Gel will be assessed using standard measures of functional decline, including change from baseline in the ALS Functional Rating Scale-Revised, assessed after 36 weeks of therapy. Approximately 195 patients will be enrolled across multiple sites.
Flex Biopharma FLX-787
This is a double-blind placebo controlled study of FLX-787-ODT in low dose and high dose in muscle cramps in people with motor neuron diseases (including ALS, PLS, PMA) over 28 days of run in with 28 days of treatment. The endpoint will be frequency of cramps along with secondary endpoints that examine cramp free days, cramp duration and severity, and screens for quality of life and functions. Safety data will also be collected. The clinic will also be participating in the trial for Charcot-Marie-Tooth (CMT).
While ALS primarily affects the motor system, up to 40% of PALS have cognitive impairment with predominantly executive dysfunction, while approximately 20% of PALS show behavioral changes, predominantly apathy and disinhibition. These cognitive and behavioral changes add significantly to the burden and distress of caregivers who now have to face the challenges of managing behavioral changes in addition to physical disability. Given this increasing burden, there is a critical need for validated interventions that can help the caregiver manage these behaviors. However, there is no standard and/or validated intervention currently available, nor has there been investigation directed at helping caregivers to manage these behavioral changes in ALS. The overall goal of our study is to characterize the relationship of caregiver burden to cognitive and behavioral impairment in ALS and to develop and perform a feasibility study of a pilot individualized intervention as a way to reduce caregiver burden. Ultimately, this study will provide the necessary background to develop, inform, and guide future targeted and larger scale interventions directed to both PALS and caregivers.
EEG-Based Brain-Computer Interface
By using EEG (brain wave) signals from the scalp to create a signal, the Brain-Computer Interface (BCI) allows people to make selections from a computer screen. This study is intended to evaluate both the complexity of the system and the degree to which each participant will be able to communicate using BCI. Trials will consist of asking the subject to follow a series of simple instructions and to complete certain tasks while using the BCI in our clinic or offices. We are also working to simplify the system as a prelude to home based use.
In a disease like ALS in which the cause is unknown, no animal models of ALS can substitute for understanding how it affects humans. We are currently collecting blood, urine, cerebrospinal fluid, and autopsy materials from people with ALS and other motor neuron diseases to look for clues in the human tissue. All samples are tied to de-identified clinical information in a database to help maximize the usefulness of this precious resource. The anonymous database includes demographic information, environmental exposures, and medical history. This will increase availability of human tissue for research with pertinent corresponding clinical information.