ALS Clinical Trials

Listed below are the clinical research studies currently underway or in planning stages at the Johns Hopkins ALS Clinic. 

Research Studies currently enrolling participants:

A Phase 1, Double-Blind, Placebo-Controlled, Dose-Escalation Study of the Safety, Tolerability, and Pharmacokinetics of ISIS 333611 Administered Intrathecally to Patients with Familial Amyotrophic Lateral Sclerosis Due to Superoxide Dismutase 1 Gene Mutations
Jeff Rothstein, MD, PhD – Principal Investigator

Brain metabolism in amyotrophic lateral sclerosis (ALS): A 3 Tesla magnetic resonance spectroscopy study
Mona Mohamed, MD – Principal Investigator

Safety and Tolerability of the Ketogenic Diet in ALS
Vinay Chaudhry, MD – Principal Investigator

A Single and Repeat Dose Escalation Study of the Safety, Pharmacokinetics and Pharmacodynamics of GSK1223249 in Patients with ALS.
Andrea Corse, MD – Principal Investigator

Generation and Characterization of Cell Lines for ALS
Nicholas Maragakis, MD – Principal Investigator
(Currently only enrolling patients with confirmed familial ALS)
Contact: Betsy Mosmiller (p) 410-502-0495 | emosmil1@jhmi.edu

Skin biopsies to generate cell lines for study of ALS
Nicholas Maragakis, MD – Principal Investigator

A Longitudinal Study of Cognition and Behavior in ALS
Chiadi Onyike, MD, MHS – Principal Investigator

A Multicenter Study for the Validation of ALS Biomarkers
Nicholas Maragakis, MD – Principal Investigator

Upcoming Trials (not yet enrolling participants):

No Longer Enrolling Participants (ongoing studies):

A Phase II, Double-Blind, Randomized, Placebo-Controlled, Three-Way Crossover, Pharmacokinetic and Pharmacodynamic Study of CK-2017357 in Patients with Amyotrophic Lateral Sclerosis (ALS)
Nicholas Maragakis, MD – Principal Investigator

Electrical impedance myography as an outcome measure in ALS clinical trials
Nicholas Maragakis, MD – Principal Investigator

Quality of Life and Caregiver Burden in ALS
Richard Kimball, MSN/MPH APRN PhD(c) – Principal Investigator

A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients with Amyotrophic Lateral Sclerosis (ALS)
Jeff Rothstein, MD, PhD – Principal Investigator

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For information about these clinical trials, please contact
Lora Clawson, MSN, CRNP
Director of ALS Clinical Services
410-955-8511
lclawson@jhmi.edu

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Information about other ALS-specific research trials can be found on these websites:

MUSCULAR DYSTROPHY ASSOCIATION
http://www.mda.org/research/ctrials.aspx

ALS ASSOCIATION
http://www.alsa.org/research/drugdev.cfm

 NATIONAL INSTITUTES OF HEALTH
www.clinicaltrials.gov