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Neuromuscular

Neuraltus Pharmaceuticals, Inc. is continuing their study of NP001 in people with ALS.  This is an injectable experimental drug designed to minimize the contribution of inflammation to the development or progression of the disease by changing the action of the blood cells responsible for inflammation in the body.  Analysis of data from the phase I study of NP001 showed that people with elevated levels of the inflammation biomarker CRP may have experienced enhanced respiratory function due to action of NP001.  Subjects must be able to attend up to 5 consecutive dosing days per month over a 6-month period, and must be tested for elevated inflammation at baseline in order to participate.  For additional information, contact Jane Bordeau, RN at 404-727-1679 (jrbord@emory.edu).

The current clinical trial sponsored by Genentech, Inc. is expanding to include a multiple-ascending-dose segment. This will involve 3 cohorts of subjects who will be given increasing doses of the Genentech experimental drug GDC-0134, based upon the safety of prior dosing regimens explored in the single-ascending-dose portion currently underway. (The current trial information is in the table below) Participants will be asked to complete a 4-5 day inpatient stay in the clinical research unit where they will receive the first doses of drug. Drug wll then be sent home with the subject, and they will return several times over the next 6-7 weeks for safety checks. Some cohorts may also be asked to take a dose of either caffeine or midazolam (a sedative medication), to assess processing of GDC-0134 by the liver. Eligibility requirements are similar to those for the single-ascending-dose portion. If you are interested, please contact Jane Bordeau (jrbord@emory.edu

ALSO COMING SOON - probably in early 2017, is a study with Amylyx pharmaceuticals.  More info can be found HERE.

The table below will gives you information about all the trials and studies currently happening at Emory ALS Center.

Research Study Name Currently Recruiting Treatment Trial? Who Is Eligible to Participate? Brief Summary Contact Information Links for More Information
A Single-Ascending Dose Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Patients with ALS NO YES
  • Patients with a diagnosis of ALS
  • Vital capacity >60%
  • Able to swallow pills; no feeding tube
  • Not taking nutritional/herbal supplements
  • Non-smoker
  • No history of eye disease (ex. glaucoma)
This Phase 1 placebo-controlled study will test the safety, tolerability, and pharmacokinetics of an investigational oral agent in patients with ALS. Participants will spend 4 days in the hospital and will have several other study visits over a 15-day period.

Jane Bordeau

(404) 727-1679

jrbord@emory.edu

GDC-0134
ANSWER ALS YES NO
  • Patients with ALS or PLS
Participants donate blood (and optional spinal fluid) and undergo testing of strength and cognition. Includes visits every 3 months for a year, then phone assessments. Stem cells will be created from each patient, and the cells will be studied to identify causes and subtypes for targeted treatments.

Arish Jamil

(404) 727-1273

arish.jamil@emory.edu

ANSWER ALS
CRiALS: Clinical research in ALS YES NO
  • Patients with ALS
  • OR
  • Family members of ALS patients
  • OR
  • Healthy volunteers
A genetic research program where participants give a blood sample for DNA and (optionally) a small skin sample to create stem cells for further research. 

Arish Jamil

(404) 727-1273

arish.jamil@emory.edu

PROJECT MINE
Single and Multiple Dose Study of BllB067 (Isis-SOD1Rx) in Adults with ALS YES YES
  • Patients with a diagnosis of sporadic ALS (Part A)
  • Patients with familial ALS due to an SOD1 gene mutation (Part B)
  • Vital capacity >50% predicted
This is a Phase 1 placebo-controlled study to test the safety, tolerability, and pharmacokinetics of an investigational drug that is injected into the spinal fluid 

Anna Partlow

(404) 778-3181

apartlo@emory.edu

BllB067
LABB: A Multicenter Study for the Discovery and Validation of ALS Biomarkers NO NO
  • Patients with a diagnosis of ALS
  • Vital capacity >50%predicted
  • Able and willing to undergo a spinal tap
The purpose of this study is to discover blood and spinal fluid markers of ALS disease progression. 

Meraida Polak

(404) 778-3807

mpolak@emory.edu

LABB
Methodology Study of Novel Outcome Measures to Assess Progression of ALS YES NO
  • Patients with a diagnosis of ALS
  • Onset of ALS symptoms <2 year prior
To identify outcome measures and biomarkers for use in ALS research studies.

Anna Partlow

(404) 778-3181

apartlo@emory.edu

METHODOLOGY