NCT02870634

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Collaborative Medicinal Development Pty Limited · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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.

Conditions

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Interventions

Type	Name	Description
DRUG	Cu(II)ATSM	copper-containing synthetic small molecule

Timeline

• Start date: 2016-11-16
• Primary completion: 2019-10-30
• Completion: 2020-01-30
• First posted: 2016-08-17
• Last updated: 2020-03-17

Locations

2 sites across 1 country: Australia