Amyotrophic Lateral SclerosisPhase 1NCT01091142
Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing NP001 in people with Amyotrophic Lateral Sclerosis, run by Neuraltus Pharmaceuticals, Inc. at 3 sites in United States.
Trial summary
Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS. Secondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS. Read more about this study
- Who is it for?
- Ages 21 to 75 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
- 56 participants
- Where
- 3 sitesUnited States
- Study dates
- Jul 2010to Sep 2010
- Phase
- Phase 1
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males and females, age 21 years - 75 years
- Subjects must be in generally sound health as appropriate for their ALS diagnosis.
- Subjects must have a clinical diagnosis of laboratory-supported probable or definite ALS, according to modified El Escorial criteria.
- Subjects receiving riluzole must be on a stable dose for at least 30 days prior to enrollment.
- Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test prior to dosing with study medication.
- Subjects must understand the study and be willing to adhere to protocol requirements as evidenced by provision of written informed consent.
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- Subject must be willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).
- Subjects must be willing to have an intravenous infusion.
- Subjects must have suitable veins for IV access as determined by examination.
Who cannot join
- Subjects should not require nor are expected to require life sustaining interventions for the next six months or longer. (e.g. invasive ventilation).
- Subjects must not have:
- presence of a tracheotomy or invasive ventilation. Nocturnal non-invasive ventilation system (e.g. C-PAP) is allowed.
- a diagnosis of neurologic disease known to mimic the muscle atrophy or weakness seen in ALS including MS, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies or neuromuscular diseases, foramen magnum or brainstem tumor, or toxic conditions.
- an active pulmonary disease under treatment including uncontrolled asthma, chronic obstructive pulmonary disease (pneumonia, bronchitis, etc.), pulmonary fibrosis, pulmonary infection in the last 2 months, or history of aspiration that may expose the subject to increased risk by participating in this trial as determined by the Investigator.
- a history of unstable medical illness in the 3 months prior to screening including any emergent hospitalizations.
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- renal disease based on screening estimated creatinine clearance (eCcr) < 50 mL/minute (Cockcroft Gault estimate using ideal body weight) where:
- evidence of elevated alanine aminotransferase greater than 3 times the upper limit of normal.
- evidence of anemia, thrombocytopenia, or neutropenia (screening hematocrit <33%, platelet count < lower limit of normal for the site laboratory, or neutrophil count less than 1,500/mm3).
- any condition that requires periodic red blood cell transfusions, erythropoietin or any blood dyscrasias undergoing active treatment in the past year.
- clinical laboratory parameters that are clinically significant in the opinion of the Investigator.
- systolic blood pressure in excess of 160 mmHg nor less than 100 mmHG or a diastolic blood pressure above 98 mmHg.
- a history of G6PD deficiency (Glucose-6-phosphate dehydrogenase deficiency) determined by subject report.
- a current history of hepatitis or HIV determined by subject report.
- Subjects must not be using systemic immunosuppressants including steroids and chemotherapeutic agents. Inhaled steroids, eye drops and local topical use are permitted with concurrence of Medical Monitor.
- Subjects must not have a hematologic disorder such as autoimmune anemia, or hemolytic anemia of any type including paroxysmal nocturnal hemoglobinuria or myoglobinuria.
- Subjects must not have a history of unexplained jaundice determined by subject report.
- Subjects must not have received IV Immunoglobulin (IG) within 30 days of the planned initial dose of study drug.
- Subjects must not be participating in another drug study or have participated in a drug study within the last 30 days prior to enrollment. Observational trials with no intervention are acceptable provided permission for the other study Sponsor is obtained in writing.
- Subjects must not have any other condition which in the Investigator's opinion would put the subject at risk by participating in this study.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Kansas Medical Center - Landon Center on Aging
Kansas City, Kansas, 66160
Department of Neurology; University of Kentucky Medical Center
Lexington, Kentucky, 40536
California Pacific Medical Center
San Francisco, California, 94115
About this study
Official title: Single-Ascending-Dose Safety and Tolerability Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS.
Secondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS.
Read the detailed description
This study is a double-blind, placebo-controlled single ascending dose safety and tolerability study. Approximately 32-56 subjects with clinical diagnosis of ALS according to modified El Escorial criteria are planned to receive a single dose of study drug, NP001.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- NP001, Placebo
- 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
Cohort 1: 0.3 mg/kg NP001(6:2 active:placebo) Cohort 2: 1.1 mg/kg NP001(6:2 active:placebo) Cohort 3: 2.1 mg/kg NP001(6:2 active:placebo) Cohort 4: 4.3 mg/kg NP001(6:2 active:placebo)
PlaceboDrug
Cohort 1: placebo Cohort 2: placebo Cohort 3: placebo Cohort 4: placebo
| Study group | Role | What they receive |
|---|---|---|
| NP001 | Experimental | Not described Receives: Drug: NP001 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of NP001 compared to placebo in subjects with ALS
Measured: 6 mo.
Secondary outcomes (1)
To explore the effects of NP001 on biomarkers potentially relevant to ALS
Measured: 6 mo.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2010 (estimated)
- Study startJul 2010
- Primary completionSep 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Robert G. Miller, MD, Principal investigator, Forbes Norris ALS Treatment and Research, California Pacific Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01091142
- Sponsor's protocol ID
- NP001 Single Dose - ALS
- Status last verified
- Oct 2010
- First submitted
- 19 Mar 2010
- Last updated
- 6 Oct 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-Ascending-Dose Safety and Tolerability Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT01091142
- Acronym / sponsor ID
- NP001 Single Dose - ALS
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Neuraltus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2010
- Primary completion
- Sep 2010 (actual)
- First posted
- 23 Mar 2010 (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.
Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS.
Secondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS.
Detailed description
This study is a double-blind, placebo-controlled single ascending dose safety and tolerability study. Approximately 32-56 subjects with clinical diagnosis of ALS according to modified El Escorial criteria are planned to receive a single dose of study drug, NP001.
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: NP001 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 21 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females, age 21 years - 75 years
- Subjects must be in generally sound health as appropriate for their ALS diagnosis.
- Subjects must have a clinical diagnosis of laboratory-supported probable or definite ALS, according to modified El Escorial criteria.
- Subjects receiving riluzole must be on a stable dose for at least 30 days prior to enrollment.
- Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test prior to dosing with study medication.
Key exclusion criteria (20 listed)
- Subjects should not require nor are expected to require life sustaining interventions for the next six months or longer. (e.g. invasive ventilation).
- Subjects must not have:
- Subjects must not be using systemic immunosuppressants including steroids and chemotherapeutic agents. Inhaled steroids, eye drops and local topical use are permitted with concurrence of Medical Monitor.
- Subjects must not have a hematologic disorder such as autoimmune anemia, or hemolytic anemia of any type including paroxysmal nocturnal hemoglobinuria or myoglobinuria.
- Subjects must not have a history of unexplained jaundice determined by subject report.
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 |
|---|---|---|
| NP001Cohort 1: 0.3 mg/kg NP001(6:2 active:placebo) Cohort 2: 1.1 mg/kg NP001(6:2 active:placebo) Cohort 3: 2.1 mg/kg NP001(6:2 active:placebo) Cohort 4: 4.3 mg/kg NP001(6:2 active:placebo) | Drug | NP001 |
| PlaceboCohort 1: placebo Cohort 2: placebo Cohort 3: placebo Cohort 4: placebo | Drug | Placebo |
Primary outcome
Safety and tolerability of NP001 compared to placebo in subjects with ALS
Time frame: 6 mo.
1 secondary outcome
To explore the effects of NP001 on biomarkers potentially relevant to ALS
Time frame: 6 mo.
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 6 Oct 2010.
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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
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NP001 in people with Amyotrophic Lateral Sclerosis, run by Neuraltus Pharmaceuticals, Inc. at 3 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 20 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 6 Oct 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Safety and tolerability of NP001 compared to placebo in subjects with ALS (time frame: 6 mo.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Oct 2010.