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ALS Clinical Research Update
Clinical trials are the only way to find a cure for ALS and motor neuron disease. While this has not yet happened, scientists are closer than ever before. |
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News and Announcements
Early Stage Planning for US Brainstorm Trial
Massachusetts General Hospital, the University of Massachusetts and Brainstorm Cell Therapeutics are in theearly stages of planning to design a phase II clinical trial of Mesenchymal Stromal Stem cells secreting Neurotrophic factors in the United States. The following required steps need to be established before a US trial can begin:
* development of a trial design;
* identification of treatment production facilities;
* FDA approval of the new trials.
ALS Association/NEALS PALS Webinars Series
NEALS offers live, online seminars (or "webinars") for people with ALS and other motor neuron disease. Experts explain the scientific rational and design of current ALS clinical research. Our webinars also offer participants the opportunity to ask questions directly to the experts running trials. Below is the information for our upcoming ALS Association/NEALS Patient Webinars:
Imaging Biomarkers Research Studies
April 16th, 2012
3pm-4pm EST
Nazem Atassi, MD, MMSc (Massachusetts General Hospital)
James Berry, MD, MPH (Massachusetts General Hospital, Harvard Medical School)
Register
Ethics and Informed Consent
April 30th, 2012
3pm-3:40pm EST
James Russell, DO (Lahey Clinic)
Nazem Atassi, MD, MMSc (Massachusetts General Hospital)
Nuedexta for Bulbar Symptoms
June 5th, 2012
4pm-5pm EST
Richard Smith, MD (Center for Neurological Study)
Jeremy Shefner, MD, PhD (SUNY Upstate Medical Center)
Phase II Trial of Nuedexta to Launch
Nuedexta is a drug approved by the FDA for improving emotional lability in ALS and other disorders. Based on patient experience, a new NEALS trial will be starting soon to evaluate whether this drug improves bulbar functions such as swallowing and speaking. Information regarding inclusion criteria and study sites will be posted on the NEALS website when available.
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Enrolling ALS Trials
Phase I and II Trials
Phase II High Fat/High Calorie Trial
A phase II nutritional diet trial looking at safety, tolerability, and preliminary efficacy trial investigating the ideal amount and type of calories to be consumed by ALS patients daily. Participants must be currently tolerating tube feedings through the use of either a g-tube or j-tube. Participants will be randomly assigned to consume one of three diets: high fat and high calorie, high calorie, or normal diet. The trial consists of five in-person clinic visits and four telephonic visits over five months: four months of tube feeds with an additional month for follow-up. This trial is currently enrolling at 12 sites throughout the United States.
Biomarker Studies
A Multicenter Study for the Discovery and Validation of Biomarkers in ALS
This biomarkers study is actively recruiting participants at six NEALS sites. Blood samples and cerebrospinal fluid (CSF) from patients with ALS will be collected. Through comparison of these samples, the researchers will learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to diagnose ALS more quickly and develop new therapies.
Familial ALS Trials
Arimoclomol in SOD1 Positive Familial ALS
An amplifier of heat-shock protein, Arimoclomol is being investigated in a phase II/III randomized, double-blind, placebo-controlled trial in patients with SOD1 positive familial ALS. Researchers are assessing safety, tolerability, and efficacy of Arimoclomol and hypothesize that it will slow disease progression. This trial is currently enrolling 80 participants at two sites: the University of Miami and Massachusetts General Hospital.
Other Interventional Trials
Trial of Resistance and Endurance Exercise in ALS
How can exercise be combined with other treatments? Standard of care has been considered to be stretching and range of motion exercises; there has been little, if any, research into other forms of exercise in ALS patients. In this pilot study, 60 participants with ALS will be assigned to treatment in 1 of 3 arms: resistance exercise, endurance exercise, or stretching and range of motion exercise. Researchers are looking to learn about which is safe and tolerable for patients with ALS. The trial will enroll at four locations: Johns Hopkins University, Massachusetts General Hospital, Carolinas Medical Center, and Washington University in St. Louis, Missouri.
Braingate2: Feasibility Study of an Intracortical Neural Interface System
BrainGate2 is a pilot clinical study looking to (1) obtain preliminary safety information about a device called BrainGate2 Neural Interface System and to (2) demonstrate the feasibility of people with paralysis of the arms and legs using this device to control a computer cursor and other assistive devices with their thoughts. The 13 month study is invasive and requires surgery. Participants must live within a three-hour drive of one of the study sites in Boston, MA, Providence, RI, or Palo Alto, CA. For more information about this study please visit the website at http://www.braingate2.org or contact Dr. Leigh Hochberg at (617) 726-4218 or by email at neurotechnology@partners.org.
Stem Cell Trials
Neural Stem Cell Transplantation
Sponsored by NeuralStem, 12 participants received injections of human spinal cord-derived neural stem cells in the lumbar spinal cord at Emory University in Atlanta, GA. The next participants will receive injections into the cervical (neck region) spinal cord. This phase I trial is primarily designed to investigate safety in patients with ALS. Participants must live in close proximity to Atlanta.
Brainstorm Stem Cell Trial
Brainstorm Cell Therapeutics is performing a Phase I/II clinical trial at the Hadassah Medical Organization in Israel, and is currently enrolling ALS participants in a trial of autologous transplantation of Mesenchymal Stromal Stem cells secreting Neurotrophic factors (MSC-NTF), taken from the patient's own bone marrow. The cells are delivered into the muscles or into the spinal fluid.
For a full list of enrolling trials, go here. |
Enrollment Closures
Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen
The Phase II Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen has recently completed enrollment. Sixty subjects have been enrolled at 9 NEALS sites across the US. Each participant will take one active study drug (creatine 30gm, tamoxifen 40mg, or tamoxifen 80mg) and one placebo for 38 weeks. This trial has a unique design which allows selecting the most promising treatment to be tested in a future larger trial. Trial results are anticipated to be announced in the beginning of 2013.
For more information on the Selection Trial, click here. Questions can be directed to Kate Jackson at kjackson13@partners.org or 617-724-3871.
ISIS Administered Intrathecally to Patients with Familial ALS
For more information on the ISIS study, click here. Questions can be directed to Katy Mahoney at kmahoney17@partners.org 617-643-7434.
Cytokinetics has Closed Two Phase II Trials
Cytokinetics announced that they are no longer recruiting participants in two phase II, randomized, double-blind, placebo-controlled trials investigating dosage of CK-2017357, a drug that enhances muscle contraction. One trial examined low, mid, and high doses both without (Cohort A) and with (Cohort B) concurrent use of Riluzole. Results from Cohort A show that CK-2017356 was well-tolerated when dosed daily in ALS patients. In Cohort B, patients reduce their Riluzole to 50 mg for seven days before taking daily oral doses of either placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for fourteen days, and to remain at that dose of Riluzole while randomized.
Cytokinetic's titration study is a three week dose escalation study to see if subjects can tolerate 500 mg CK-2017357 in daily divided doses. Participants reduce their use of Riluzole to 50 mg for seven days before being randomized to receive twice daily doses of CK-2017357, or placebo, totaling 250 mg, 375 mg, and 500 mg, each for seven days. Researchers are examining the optimal dosage to display maximum tolerability and clinical effects, with the goal of arriving at an appropriate dosing schedule to use in a planned Phase 3 trial.
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ALS Association/ NEALS Trial Expert
Devon Punch is the "ALS Association/NEALS Clinical Trial Expert." She is available to help people with motor neuron disease and their caregivers navigate the NEALS website and answer questions about clinical research during normal business hours eastern standard time. ALS Trial Expert (877) 458-0631 alstrials@partners.org |
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