Quick Info
Status
Ongoing, But Not Recruiting
Estimated Enrollment
50
Phase
I
Treatment Type
Orally available
Trial Type
Open-Label
Primary Investigator
Dominic Rowe, MD
Contact Information
Locations
Macquarie University, Sydenham, NSW 2109
Calvary Health Care Bethlehem, Caulfield, 3162
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥ 70%
Months Since Onset
Number of months since first symptoms of ALS.
24 months or less
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
no
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
no
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Unknown
Update Notes
Recruitment status updated
7/1/2019
No significant updates
2/25/2019
Location added
3/9/2017
Location update
11/2/2016
Recruitment status updated
11/2/2016
Description updated
10/4/2016
No significant updates
8/24/2016
New trial added
8/19/2016

Other Information

Purpose
Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study
Eligibility
All genders. 18 to 75 year olds. No healthy volunteers
Details
Multicenter, open-label, phase 1 study of Cu(II)ATSM administered orally to patients wit amyotrophic lateral sclerosis/motor neuron disease. The study will be conducted in three phases. In the first two phases, dose cohorts of six patients each will participate in a single dose pharmacokinetic study followed by a 28-day repeated daily dose study to establish the recommended phase 2 dose (RP2D). The first dose cohort will be treated at 3 mg/day; planned dose escalations are 6, 12, 24, and 48 mg/day, subject to observed safety assessments. In the third phase of the study, participants will be treated at the RP2D to confirm tolerability and assess preliminary evidence of efficacy. In both the dose escalation and expansion cohorts, once the first 28 days of treatment and assessments are completed, at the discretion of the investigator a patient may continue to receive Cu(II)ATSM treatment for a maximum of six 28-day treatment cycles.
Collaborator(s)
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News Articles and Summaries
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ALS Forum
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Trial Protocol as Published on Clinicaltrials.gov
NCT02870634 (First Published: 8/10/2016)