Amyotrophic Lateral SclerosisNCT00116558
Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation
A study testing Early NIPPV and Standard NIPPV in people with Amyotrophic Lateral Sclerosis, run by Edward Kasaraskis at 12 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Early NIPPV and Standard NIPPV
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 153 participants
- Where
- 12 sitesUnited States
- Study dates
- 1 Aug 2004to Jan 2012
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 9Show fewer
- FVC Criteria
- NIPPV Arm: Best-sitting FVC between 50% and 95% of predicted normal.
- Nutrition Arm: Best-sitting FVC >50% of predicted normal.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
University of Vermont
Burlington, Vermont, 54505
University of Colorado
Denver, Colorado, 80262
Henry Ford Hospital
Detroit, Michigan, 48202
Pennsylvania State University
Hershey, Pennsylvania, 17033
University of Kentucky
Lexington, Kentucky, 40536
University of Miami
Miami, Florida, 33136
Beth Israel
New York, New York, 10003
Columbia University
New York, New York, 10032
Drexel University
Philadelphia, Pennsylvania, 19103
University of Utah
Salt Lake City, Utah, 84132
University of Texas-San Antonio
San Antonio, Texas, 78229
SUNY
Syracuse, New York, 13210
About this study
Official title: Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
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.
Read the 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. The last patient was followed clinically until June 30, 2008. The study remained open for sample analysis, data analysis, and assessment of vital status until the completion of funding on November 30, 2009.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Early NIPPV, Standard NIPPV
- Keywords
- amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, nutrition, NIPPV, non-invasive positive pressure ventilation, percutaneous endoscopic gastrostomy, PEG
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Early NIPPVDevice
Also called BiPAP (Bilevel Positive Airway Pressure)
Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC).
Standard NIPPVDevice
Also called BiPAP (Bilevel Positive Airway Pressure)
Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC).
| Study group | Role | What they receive |
|---|---|---|
| Early NIPPV Intervention | Experimental | Participants with >80% predicted forced vital capacity (FVC). Receives: Device: Early NIPPV |
| Standard of Care NIPPV | Active comparator | Participants with 50-74% predicted forced vital capacity (FVC). Receives: Device: Standard NIPPV |
| Standard of Care NIPPV and Nutritional Monitoring | Active comparator | Participants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status. Receives: Device: Standard NIPPV |
What is being measured
The primary outcome is the main question the study is designed to answer.
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment.
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Measured: 6 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2005 (estimated)
- Study start1 Aug 2004
- Primary completionJan 2012
- Study completionJan 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 153 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edward Kasaraskis
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Edward Kasaraskis, University of Kentucky
- Investigators
- Edward Kasarskis, MD, PhD, Principal investigator, University of Kentucky
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00116558
- Sponsor's protocol ID
- R01NS045087
- Other IDs
- R01NS045087
- Status last verified
- Jul 2019
- First submitted
- 29 Jun 2005
- Last updated
- 31 Jul 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Kasarskis EJ, Mendiondo MS, Matthews DE, Mitsumoto H, Tandan R, Simmons Z, Bromberg MB, Kryscio RJ; ALS Nutrition/NIPPV Study Group. Estimating daily energy expenditure in individuals with amyotrophic lateral sclerosis. Am J Clin Nutr. 2014 Apr;99(4):792-803. doi: 10.3945/ajcn.113.069997. Epub 2014 Feb 12.
PubMed 24522445Scagnelli CN, Howard DB, Bromberg MB, Kasarskis EJ, Matthews DE, Mitsumoto HM, Simmons Z, Tandan R; ALS Nutrition-NIPPV Study Group. Hydration measured by doubly labeled water in ALS and its effects on survival. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):220-231. doi: 10.1080/21678421.2017.1413117. Epub 2017 Dec 15.
PubMed 29243507Kasarskis EJ, Mendiondo MS, Wells S, Malguizo MS, Thompson M, Healey M, Kryscio RJ; ALS Nutrition/NIPPV Study Group. The ALS Nutrition/NIPPV Study: design, feasibility, and initial results. Amyotroph Lateral Scler. 2011 Jan;12(1):17-25. doi: 10.3109/17482968.2010.515225.
PubMed 21271789
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Treatment of Amyotrophic Lateral Sclerosis With Nutrition and Non-Invasive Positive Pressure Ventilation
- ClinicalTrials.gov ID
- NCT00116558
- Acronym / sponsor ID
- R01NS045087
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, nutrition, NIPPV, non-invasive positive pressure ventilation, percutaneous endoscopic gastrostomy, PEG
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 153 participants (actual)
- Lead sponsor
- Edward KasaraskisUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- 1 Aug 2004 (actual)
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 30 Jun 2005 (estimated)
- Results
- Posted 31 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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. The last patient was followed clinically until June 30, 2008. The study remained open for sample analysis, data analysis, and assessment of vital status until the completion of funding on November 30, 2009.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Early NIPPV Intervention | Experimental | Device: Early NIPPVParticipants with >80% predicted forced vital capacity (FVC). |
| Standard of Care NIPPV | Active comparator | Device: Standard NIPPVParticipants with 50-74% predicted forced vital capacity (FVC). |
| Standard of Care NIPPV and Nutritional Monitoring | Active comparator | Device: Standard NIPPVParticipants with 50-95% forced vital capacity (FVC), and normal or impaired amyotrophic lateral sclerosis functional rating scale (ALSFRS) scores. Participants in this group will receive standard of care NIPPV therapy but will also undergo detailed analysis of nutritional status. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Early NIPPVAlso: BiPAP (Bilevel Positive Airway Pressure)Early intervention with non-invasive positive pressure ventilation (NIPPV) at 80% forced vital capacity (FVC). | Device | Early NIPPV Intervention |
| Standard NIPPVAlso: BiPAP (Bilevel Positive Airway Pressure)Standard of care non-invasive positive pressure ventilation (NIPPV) at 50% forced vital capacity (FVC). | Device | Standard of Care NIPPV, Standard of Care NIPPV and Nutritional Monitoring |
Primary outcome
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment.
Time frame: 6 weeks
Percentage of patients attempting to use NIPPV therapy within six weeks of initial offer.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 24522445Kasarskis EJ, Mendiondo MS, Matthews DE, Mitsumoto H, Tandan R, Simmons Z, Bromberg MB, Kryscio RJ; ALS Nutrition/NIPPV Study Group. Estimating daily energy expenditure in individuals with amyotrophic lateral sclerosis. Am J Clin Nutr. 2014 Apr;99(4):792-803. doi: 10.3945/ajcn.113.069997. Epub 2014 Feb 12.
- Background: PubMed 29243507Scagnelli CN, Howard DB, Bromberg MB, Kasarskis EJ, Matthews DE, Mitsumoto HM, Simmons Z, Tandan R; ALS Nutrition-NIPPV Study Group. Hydration measured by doubly labeled water in ALS and its effects on survival. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):220-231. doi: 10.1080/21678421.2017.1413117. Epub 2017 Dec 15.
- Results publication: PubMed 21271789Kasarskis EJ, Mendiondo MS, Wells S, Malguizo MS, Thompson M, Healey M, Kryscio RJ; ALS Nutrition/NIPPV Study Group. The ALS Nutrition/NIPPV Study: design, feasibility, and initial results. Amyotroph Lateral Scler. 2011 Jan;12(1):17-25. doi: 10.3109/17482968.2010.515225.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edward Kasarskis, MD, PhD principal investigator
University of Kentucky
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Early NIPPV and Standard NIPPV in people with Amyotrophic Lateral Sclerosis, run by Edward Kasaraskis at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Acceptance Rate of Early Non-invasive Positive Pressure Ventilation (NIPPV) Treatment. (time frame: 6 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00116558, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jul 2019.