This trial is active, not recruiting.

Condition amyotrophic lateral sclerosis
Treatment bipap
Sponsor University of Kentucky
Collaborator National Institute of Neurological Disorders and Stroke (NINDS)
Start date August 2004
End date July 2008
Trial size 220 participants
Trial identifier NCT00116558, R01NS045087


The purposes of the study are to determine the energy balance and evaluate the nutritional status of patients with ALS, and to investigate the use of NIPPV as respiratory support to treat patients with ALS.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Intervention model parallel assignment
Masking open label
Primary purpose treatment
non-invasive positive pressure ventilation (NIPPV) - Early Intervention with NIPPV NIPPV involves mechanically assisted or generated breaths delivered through a tightly fitting nasal or facial mask
(No Intervention)

Primary Outcomes

ID sensitive early respiratory failure measure; ID NIPPV acceptance & tolerance predictors & develop compliance policy; Validate respiratory questionnaires.
time frame: duration of the trial
Guidelines to standardize NIPPV and nutritional interventions during the conduct of multicenter clinical drug studies.
time frame: duration of the trial

Secondary Outcomes

Develop acceptance & compliance statistics for Phase 3; Estimate survival increase with early NIPPV use for Phase 3
time frame: duration of the trial
Develop equations to accurately predict TDEE based on clinical measures that are validated against the DLW method.
time frame: duration of the trial
Identify optimal method to measure Energy Intake (EI). Formulate criteria for intervention with PEG based on maintaining energy balance
time frame: duration of the trial

Eligibility Criteria

Male or female participants from 18 years up to 80 years old.

Inclusion Criteria: - Willing and able to give informed consent. - If women of childbearing age, must be non-lactating and surgically sterile or using an effective method of birth control (double-barrier or oral contraception) and have a negative pregnancy test. - Minorities: All races and ethnic backgrounds. - Disease inclusion: Clinically possible/definite/probable, or laboratory supported probable, sporadic or familial ALS according to the revised Escorial criteria. - Onset of progressive weakness within 60 months prior to study. - Willing to return for visits as scheduled and adhere to protocol requirements. FVC Criteria - NIPPV Arm: Best-sitting FVC between 50% and 95% of predicted normal. - Nutrition Arm: Best-sitting FVC >50% of predicted normal. Exclusion Criteria: - Untreated or unstable hyper or hypothyroidism, untreated or unstable asthma, unstable angina, advanced liver or renal disease, advanced cancer, untreated diabetes or chronic obstructive pulmonary disease (COPD). - Diagnosis of other motor neuron or other neurological disorder that mimics ALS. - Diagnosis of other neurodegenerative disease (i.e., Parkinson's disease, Alzheimer's disease, etc.) - Inflammatory bowel disease or malabsorption syndrome. - Use of NIPPV, mechanical ventilation, or tracheostomy at the time of consent. - Inability to adhere to study visit schedule or lack of reliable caretaker if participant requires one. - Pregnant or lactating woman.

Additional Information

Official title Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
Principal investigator Edward Kasarskis, MD, PhD
Description Studies to date indicate that percutaneous endoscopic gastrostomy (PEG)—insertion of a feeding tube in the stomach—and non-invasive positive pressure ventilation (NIPPV)—mechanically assisted or generated breaths delivered through a tightly fitting nasal or facial mask—improve survival in amyotrophic lateral sclerosis (ALS), even when introduced late in the disease. Dr. Kasarskis and his research team believe early intervention with these treatments may improve patients outcome even further. However, many issues regarding the early use of these treatments prevent the design of a phase III clinical trial to test this hypothesis. Common to both NIPPV and nutrition is a lack of a reliable indicator of early respiratory or nutritional insufficiency. With respect to nutrition, reliable and cost effective methods are needed to determine a patient's energy (i.e. caloric) requirements at different stages of the illness to establish a basis for recommending PEG on the adequacy of energy intake. For NIPPV, factors that influence acceptance and tolerability of this therapy, and measurements of early respiratory dysfunction need to be identified. The purposes of this trial are to develop and validate strategies to improve tolerability of NIPPV, identify factors that influence acceptance of NIPPV, and evaluate measures of early respiratory failure, other than percentage of forced vital capacity (FVC). Researchers will also develop and validate methods to determine energy balance in and evaluate the nutritional status of patients with ALS that will be applicable to a multi-center phase III study of nutrition and NIPPV. The study will be conducted at 11 other sites across the country. Some study sites will focus on the nutritional aspects of the trial while the other sites will focus on NIPPV treatment. A total of 220 patients will be studied over 2 years. Enrollment will end on June 30, 2007.
Trial information was received from ClinicalTrials.gov and was last updated in January 2008.
Information provided to ClinicalTrials.gov by National Institute of Neurological Disorders and Stroke (NINDS).