July 10, 2014
Institutional Review Board, Hospital for Special Surgery
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Before enrolling in a clinical trial or research study, the investigator will explain the purpose of the trial/study, its expected benefits, any possible risks or side effects, and what your role will be. If you want to join the trial/study, you must sign informed consent documents. You can leave a trial/study at any time without penalty.
For further information, see Understanding Clinical Trials/Research Studies.
Eliz Agopian, MD
Nicole Kassebaum
Lauren Langford
Dora Leung, MD
Erin Manning
Janki Panchal
Leah Pillosph, RN
Rahul Remanan
Mona Shahbazi
Alexander Shtilbans, MD, PhD, MS
An unblinded, randomized controlled clinical trial comparing DPS treatment group to
standard of care (control) group. The purpose of this study is to determine if surgical implantation of the NeuRx® Diaphragm Pacing System™ (DPS) improves survival or diaphragm function in individuals with ALS and breathing muscle weakness. Subjects will be randomly assigned in a 2:1 ratio to DPS or standard of care treatment group, respectively. In addition to the screening/randomization visit all study subjects will have on-site study visits at 3, 6, 9, 12, 15, and 18 months. Phone call assessments will be completed monthly after the screening visit, between study visits, and continue indefinitely after the 18 month study visit to assess for any adverse events and optimize standard of care treatment for any study subject reported breathing symptoms. Vitality status will be assessed during the entire study duration.
Inclusion Criteria
1. Age 21 years or older.
2. Sporadic or familial ALS diagnosed as definite, probable or possible ALS as defined by revised El Escorial criteria.
3. Evidence of hypoventilation at Screening with at least one of the following:
a. Maximal static inspiratory pressure (MIP) <60 cm H20.
b. Upright or supine forced vital capacity (FVC) <50% predicted for gender, age, and height.
4. Both hemi-diaphragms must be able to be stimulated with a recordable compound muscle action potential
(CMAP _ 0.1milivolts amplitude and distal latency _ 11 milliseconds) with standard phrenic nerve conduction studies.
5. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole
for at least 30 days, prior to randomization (riluzole-naïve subjects are permitted in the study).
6. Capable of providing informed consent and following trial procedures.
7. Geographically accessible to the site.
8. Negative urine pregnancy test at Screening in women of child bearing potential (WOCBP).
(Women who are post-menopausal or who have had a hysterectomy are deemed not of child bearing potential).
9. Women of child bearing potential must use an adequate form of contraception: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for _ 3 months, barrier method in conjunction with spermicide, or another adequate method.
Exclusion Criteria
1. Upright forced vital capacity (FVC) _ 45% of predicted for gender, age, and height.
2. Any prior use of non-invasive ventilation (NIV) outside of sleep (nighttime or naps).
3. Any pulmonary or cardiac disorder or other medical disorder that would be a contraindication for general anesthesia or DPS hardware implantation in the chest.
4. Implanted electrical device such as a pacemaker or cardiac defibrillator.
5. Known diaphragm abnormality such as hiatal hernia or para-esophageal hernia of abdominal contents into the thoracic cavity.
6. Participation in another treatment research study for people with ALS.
7. Exposure to any other agent currently under investigation for the treatment of people with ALS (off-label use or investigational) within 30 days of the Screening Visit.
8. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, psychiatric, renal disease, or other medically significant illness.
9.Pregnant women or women currently breastfeeding.
Lindsay Kaplan, BS
646.797.8657
KaplanL@hss.edu