Amyotrophic Lateral SclerosisPhase 2NCT02794857
Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation
A Phase 2 study testing NP001 in people with Amyotrophic Lateral Sclerosis, run by Neuraltus Pharmaceuticals, Inc. at 22 sites in 2 countries.
Trial summary
This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo. Read more about this study
- Who is it for?
- Ages 21 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- NP001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 138 participants
- Where
- 22 sitesin 2 countries
- Study dates
- 29 Aug 2016to 12 Dec 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 21 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
- Forced vital capacity greater than or equal to 65% of that predicted for age and height
- Onset of ALS-related weakness less than 3 years prior to first dose of study drug
- Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
- Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent
- For females: Not be of childbearing potential or agree to use adequate birth control during the study
Who cannot join
- Life expectancy of less than 6 months
- Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
- Active pulmonary disease
- Gastrostomy
- Stem cell therapy
- Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening
- Unstable medical condition other than ALS
Trial locations and contacts
22 sites in 2 countries.
22 sites in 2 countries
United States21 sites
Emory University, Department of Neurology
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
Carolinas Medical Center, Neurosciences Instutite-Neurology
Charlotte, North Carolina, 28207
Cleveland Clinic Foundation-Cleveland Clinic Hospital
Cleveland, Ohio, 44195
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Duke Neurological Disorders Clinic at Morreene Road
Durham, North Carolina, 27705
Houston Methodist Neurological Institute
Houston, Texas, 77030
Mayo Clinic Florida
Jacksonville, Florida, 32224
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Kentucky, Albert B. Chandler Medical Center
Lexington, Kentucky, 40536-0293
Cedars-Sinai Medical Center
Los Angeles, California, 90048
University of Miami Miller School of Medicine
Miami, Florida, 33136
Clinical & Translational Science Institute, University of Minnesota
Minneapolis, Minnesota, 55414
Columbia University Medical Center
New York, New York, 10032
University of California, Irvine, Department of Neurology
Orange, California, 92862
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
Phoenix, Arizona, 85013
Providence Brain & Spine Institute, ALS Center
Portland, Oregon, 97213
University of Texas Health Sciences Center San Antonio
San Antonio, Texas, 78229
Forbes Norris MDA/ALS Research Center, CPMC
San Francisco, California, 94115
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation
This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.
Read the detailed description
This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- NP001, Placebo
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation, C-Reactive Protein
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NP001Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| NP001 | Experimental | NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6 Receives: Drug: NP001 |
| Placebo | Placebo comparator | Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
Measured: Baseline and 6 months
Secondary outcomes (3)
Change in pulmonary function as measured by slow vital capacity readings
Measured: Baseline and 6 months
Time to tracheotomy
Measured: Up to 6 months
Change in levels of blood inflammatory biomarkers
Measured: Baseline, 3 and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2016 (estimated)
- Study start29 Aug 2016
- Primary completion12 Dec 2017
- Study completion12 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 138 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gil Block, MD, PhD, Study director, Neuraltus Pharmaceuticals, Inc.Robert G. Miller, MD, Principal investigator, California Pacific Medical CenterJonathan Katz, MD, Principal investigator, California Pacific Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02794857
- Sponsor's protocol ID
- NP001-10-003
- Status last verified
- May 2018
- First submitted
- 24 May 2016
- Last updated
- 7 May 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Forrest BD, Goyal NA, Fleming TR, Bracci PM, Brett NR, Khan Z, Robinson M, Azhir A, McGrath M. The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis. Biomedicines. 2024 Oct 16;12(10):2367. doi: 10.3390/biomedicines12102367.
PubMed 39457678
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation
- ClinicalTrials.gov ID
- NCT02794857
- Acronym / sponsor ID
- NP001-10-003
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Central Nervous System Diseases, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases, Inflammation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 138 participants (actual)
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Aug 2016 (actual)
- Primary completion
- 12 Dec 2017 (actual)
- Study completion
- 12 Dec 2017 (actual)
- First posted
- 9 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.
Detailed description
This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NP001 | Experimental | Drug: NP001NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6 |
| Placebo | Placebo comparator | Drug: PlaceboNormal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6 |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
- Forced vital capacity greater than or equal to 65% of that predicted for age and height
- Onset of ALS-related weakness less than 3 years prior to first dose of study drug
- Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
- Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent
Key exclusion criteria (7 listed)
- Life expectancy of less than 6 months
- Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
- Active pulmonary disease
- Gastrostomy
- Stem cell therapy
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 |
|---|---|---|
| NP001 | Drug | NP001 |
| Placebo | Drug | Placebo |
Primary outcome
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire
Time frame: Baseline and 6 months
3 secondary outcomes
Change in pulmonary function as measured by slow vital capacity readings
Time frame: Baseline and 6 months
Time to tracheotomy
Time frame: Up to 6 months
Change in levels of blood inflammatory biomarkers
Time frame: Baseline, 3 and 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2018.
- Derived from this study: PubMed 39457678Forrest BD, Goyal NA, Fleming TR, Bracci PM, Brett NR, Khan Z, Robinson M, Azhir A, McGrath M. The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis. Biomedicines. 2024 Oct 16;12(10):2367. doi: 10.3390/biomedicines12102367.
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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.
Gil Block, MD, PhD study director
Neuraltus Pharmaceuticals, Inc.
Robert G. Miller, MD principal investigator
California Pacific Medical Center
Jonathan Katz, MD principal investigator
California Pacific Medical Center
22 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NP001 in people with Amyotrophic Lateral Sclerosis, run by Neuraltus Pharmaceuticals, Inc. at 22 sites in 2 countries.
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 6 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 7 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Canada. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change from baseline in score on ALS Functional Rating Scale-Revised (ALSFRS-R) questionnaire (time frame: Baseline and 6 months). 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 NCT02794857, which also lists 1 linked publication.
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 7 May 2018.