Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation (NIPPV)
Recruitment status was Active, not recruiting
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Amyotrophic Lateral Sclerosis |
Device: BiPAP |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation |
- ID sensitive early respiratory failure measure; ID NIPPV acceptance & tolerance predictors & develop compliance policy; Validate respiratory questionnaires. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
- Guidelines to standardize NIPPV and nutritional interventions during the conduct of multicenter clinical drug studies. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
- Develop acceptance & compliance statistics for Phase 3; Estimate survival increase with early NIPPV use for Phase 3 [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
- Develop equations to accurately predict TDEE based on clinical measures that are validated against the DLW method. [ Time Frame: duration of the trial ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 220 |
| Study Start Date: | August 2004 |
| Estimated Study Completion Date: | October 2008 |
| Estimated Primary Completion Date: | July 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: intervention group
NIPPV
|
Device: BiPAP
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: Nutrition |
Detailed 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.
Eligibility| Ages Eligible for Study: | 18 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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.
Contacts and Locations| United States, Colorado | |
| University of Colorado | |
| Denver, Colorado, United States, 80262 | |
| United States, Florida | |
| University of Miami | |
| Miami, Florida, United States, 33136 | |
| United States, Kentucky | |
| University of Kentucky | |
| Lexington, Kentucky, United States, 40536 | |
| United States, Michigan | |
| Henry Ford Hospital | |
| Detroit, Michigan, United States, 48202 | |
| United States, New York | |
| Columbia University | |
| New York, New York, United States, 10032 | |
| Beth Israel | |
| New York, New York, United States, 10003 | |
| SUNY | |
| Syracuse, New York, United States, 13210 | |
| United States, Pennsylvania | |
| Pennsylvania State University | |
| Hershey, Pennsylvania, United States, 17033 | |
| Drexel University | |
| Philadelphia, Pennsylvania, United States, 19103 | |
| United States, Texas | |
| University of Texas-San Antonio | |
| San Antonio, Texas, United States, 78229 | |
| United States, Utah | |
| University of Utah | |
| Salt Lake City, Utah, United States, 84132 | |
| United States, Vermont | |
| University of Vermont | |
| Burlington, Vermont, United States, 54505 | |
| Principal Investigator: | Edward Kasarskis, MD, PhD | University of Kentucky |
More Information
Additional Information:
No publications provided
| Responsible Party: | Edward Kasarskis, MD, PhD ,, University of Kentucky |
| ClinicalTrials.gov Identifier: | NCT00116558 History of Changes |
| Other Study ID Numbers: | R01NS045087 |
| Study First Received: | June 29, 2005 |
| Last Updated: | January 8, 2008 |
| Health Authority: | United States: Federal Government |
Keywords provided by National Institute of Neurological Disorders and Stroke (NINDS):
|
amyotrophic lateral sclerosis ALS Lou Gehrig's disease nutrition |
NIPPV non-invasive positive pressure ventilation percutaneous endoscopic gastrostomy PEG |
Additional relevant MeSH terms:
|
Amyotrophic Lateral Sclerosis Sclerosis Motor Neuron Disease Spinal Cord Diseases Central Nervous System Diseases Nervous System Diseases |
Neurodegenerative Diseases TDP-43 Proteinopathies Neuromuscular Diseases Proteostasis Deficiencies Metabolic Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on June 18, 2013