Amyotrophic Lateral SclerosisPhase 2NCT01281631
A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing NP001 in people with Amyotrophic Lateral Sclerosis, run by Neuraltus Pharmaceuticals, Inc. at 17 sites in United States.
Trial summary
This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS. 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
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 136 participants
- Where
- 17 sitesUnited States
- Study dates
- Feb 2011to Sep 2012
- 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
- Onset of symptoms less than 3 years prior to study entry.
- Forced Vital Capacity (FVC) at least 70% of that predicted for age and height.
- Stable dose 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
- Unstable medical condition(s) other than ALS.
- Life expectancy of less than 6 months.
- Require life-sustaining interventions for the 6 months following randomization.
- Have a tracheotomy or be using ventilatory assistance [including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)].
- Active pulmonary disease.
- Immune modulator therapy within 12 weeks of study entry or participation in studies of other agents within the last 4 weeks prior to the randomization.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
United States17 sites
The Emory Clinic
Atlanta, Georgia, 30322
Providence Saint Peter Hospital
Centralia, Washington, 98531
Massachusetts General Hospital
Charlestown, Massachusetts, 02129
Carolinas Medical Center
Charlotte, North Carolina, 28207
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University, Dept of Neurology
Durham, North Carolina, 27705
Methodist Hospital Research Institute, Methodist Neurologic Institute
Houston, Texas, 77030
UC, Irvine
Irvine, California, 92868-4281
Mayo Clinic, Jacksonville
Jacksonville, Florida, 32224
University of Kansas Medical Center, Landon Center on Aging
Kansas City, Kansas, 66160
University of Kentucky, Department of Neurology
Lexington, Kentucky, 40536-0284
UCLA
Los Angeles, California, 90095
Columbia University
New York, New York, 10032
Providence ALS Center
Portland, Oregon, 97213
California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center
San Francisco, California, 94115
Mayo Clinic, Scottsdale
Scottsdale, Arizona, 85259
SUNY Upstate Medical University, Syracuse
Syracuse, New York, 13210
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.
Read the detailed description
This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- NP001, Placebo
- Keywords
- ALS
- 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.
NP001Drug
Low dose of NP001
NP001Drug
High dose of NP001
PlaceboDrug
normal saline
| Study group | Role | What they receive |
|---|---|---|
| Low dose NP001 | Experimental | Low drug dose Receives: Drug: NP001 |
| High dose NP001 | Experimental | High drug dose Receives: Drug: NP001 |
| Placebo | Placebo comparator | normal saline Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the effects of NP001 on measures of clinical function
Measured: over 9 months
Secondary outcomes (2)
Safety and tolerability in ALS
Measured: duration of study
Pulmonary function and biomarkers
Measured: over 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2011 (estimated)
- Study startFeb 2011
- Primary completionSep 2012
- Study completionSep 2012
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
- 136 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert G. Miller, MD, Principal investigator, Forbes Norris ALS Treatment and Research, California Pacific Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01281631
- Sponsor's protocol ID
- NP001-10-002
- Status last verified
- Nov 2012
- First submitted
- 17 Jan 2011
- Last updated
- 22 Nov 2012
- Results posted
- No
- Data monitoring committee
- Yes
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 22 Nov 2012. 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)
- ClinicalTrials.gov ID
- NCT01281631
- Acronym / sponsor ID
- NP001-10-002
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 136 participants (actual)
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2011
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 24 Jan 2011 (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 is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.
Detailed description
This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low dose NP001 | Experimental | Drug: NP001Low drug dose |
| High dose NP001 | Experimental | Drug: NP001High drug dose |
| Placebo | Placebo comparator | Drug: Placebonormal saline |
Who may qualify
- Ages
- 21 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Onset of symptoms less than 3 years prior to study entry.
- Forced Vital Capacity (FVC) at least 70% of that predicted for age and height.
- Stable dose 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.
Key exclusion criteria (6 listed)
- Unstable medical condition(s) other than ALS.
- Life expectancy of less than 6 months.
- Require life-sustaining interventions for the 6 months following randomization.
- Have a tracheotomy or be using ventilatory assistance [including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)].
- Active pulmonary disease.
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 |
|---|---|---|
| NP001Low dose of NP001 | Drug | Low dose NP001 |
| NP001High dose of NP001 | Drug | High dose NP001 |
| Placebonormal saline | Drug | Placebo |
Primary outcome
To evaluate the effects of NP001 on measures of clinical function
Time frame: over 9 months
2 secondary outcomes
Safety and tolerability in ALS
Time frame: duration of study
Pulmonary function and biomarkers
Time frame: over 9 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 22 Nov 2012.
- 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.
Robert G. Miller, MD principal investigator
Forbes Norris ALS Treatment and Research, California Pacific Medical Center
17 sites in 1 country: 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 17 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 4 inclusion and 6 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 22 Nov 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 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. 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?
To evaluate the effects of NP001 on measures of clinical function (time frame: over 9 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 NCT01281631, 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 22 Nov 2012.