Diaphragm Pacing System (DPS) in Participants with Amyotrophic Lateral Sclerosis (ALS)
Study type: Interventional What is this?
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study phase: II What is this?
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.
- Jacksonville, Florida: 13-006347
NCT ID: NCT01938495
Sponsor Protocol Number: 13-006347
About this study
The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function.
The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival.
The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.
See eligibility criteria
- Age 21 years or older.
- Sporadic or familial ALS diagnosed as definite, probable or possible ALS as defined by revised El Escorial criteria.
- Evidence of hypoventilation at Screening with at least one of the following:
- Maximal static inspiratory pressure (MIP) <60 cm H20.
- Upright or supine forced vital capacity (FVC) <50% predicted for gender, age, and height.
- 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.
- 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).
- Capable of providing informed consent and following trial procedures.
- Geographically accessible to the site.
- 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).
- 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.
- Upright forced vital capacity (FVC) ≤ 45% of predicted for gender, age, and height.
- Any prior use of non-invasive ventilation (NIV) outside of sleep (nighttime or naps).
- Any pulmonary or cardiac disorder or other medical disorder that would be a contraindication for general anesthesia or DPS hardware implantation in the chest.
- Implanted electrical device such as a pacemaker or cardiac defibrillator.
- Known diaphragm abnormality such as hiatal hernia or para-esophageal hernia of abdominal contents into the thoracic cavity.
- Participation in another treatment research study for people with ALS.
- 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.
- Clinically significant history of unstable or severe cardiac, oncologic, hepatic, psychiatric, renal disease, or other medically significant illness.
- Pregnant women or women currently breastfeeding.
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.