Clinical Trial: Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1 Single and Multiple Dose Escalation and Pharmacokinetic Study of Cu(II)ATSM Administered Orally to Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease

Brief Summary: Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study

Detailed Summary:

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.


Sponsor: Collaborative Medicinal Development Pty Limited

Current Primary Outcome: recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities [ Time Frame: 24 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: 24 months ]
  • Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score [ Time Frame: 24 months ]
  • Treatment-related change in respiratory function by seated forced vital capacity (FCV) [ Time Frame: 24 months ]
  • Treatment-related change in quality of life by ALSSQOL-R score [ Time Frame: 24 months ]
  • Treatment-related change in disease severity by transcranial magnetic stimulation (TMS) response [ Time Frame: 24 months ]
  • Peak Cu(II)ATSM plasma concentration following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing [ Time Frame: 12 months ]
  • Area under the Cu(II)ATSM plasma concentration versus time curve (AUC) following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing [ Time Frame: 12 months ]
  • Treatment-related change in respiratory function by sniff nasal pressure (SNP) test [ Time Frame: 24 months ]


Original Secondary Outcome: Same as current

Information By: Collaborative Medicinal Development Pty Limited

Dates:
Date Received: August 10, 2016
Date Started: October 2016
Date Completion: April 2019
Last Updated: March 6, 2017
Last Verified: March 2017