Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01091142
• Date of registration: 19/03/2010
• Prospective Registration: Yes
• Primary sponsor: Neuraltus Pharmaceuticals, Inc.
• Public title: Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: Single-Ascending-Dose Safety and Tolerability Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: July 2010
• Target sample size: 56
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01091142
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Robert G. Miller, MD
• Affiliation: Forbes Norris ALS Treatment and Research, California Pacific Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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.

- 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.

Exclusion Criteria:

- 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.

- 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.



Age minimum: 21 Years
Age maximum: 75 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo

Drug: NP001

Primary Outcome(s)

Safety and tolerability of NP001 compared to placebo in subjects with ALS [Time Frame: 6 mo.]

Secondary Outcome(s)

To explore the effects of NP001 on biomarkers potentially relevant to ALS [Time Frame: 6 mo.]

Secondary ID(s)

NP001 Single Dose - ALS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors