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Amyotrophic Lateral SclerosisPhase 1NCT02870634

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

A Phase 1 study testing Cu(II)ATSM in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Collaborative Medicinal Development Pty Limited at 2 sites in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
Cu(II)ATSM
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
2 sitesAustralia
Study dates
16 Nov 2016to 30 Jan 2020
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 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Signed informed consent prior to initiation of any study-specific procedures;
  • Familial or sporadic ALS/MND defined as clinically possible, probable, or definite by Awaji-shima Consensus Recommendations;
  • First ALS/MND symptoms occurred no more than 2 years prior to screening visit;
  • Seated FVC ≥ 70% and SNP ≥ 50% of predicted value;
  • Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to screening visit (participants are not allowed to start taking riluzole during the study);
  • Age between 18 and 75 years at time of informed consent;
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  • Patient has a competent caregiver who can and will be responsible for administration of study drug;
  • Adequate bone marrow reserve, renal and liver function:
  • absolute neutrophil count ≥ 1500/µL
  • lymphocyte count < 48%
  • platelet count ≥ 150,000/µL
  • hemoglobin ≥ 11 g/dL
  • creatinine clearance ≥ 60 mL/min (Cockroft \& Gault formula)
  • ALT and/or AST ≤ 2 x ULN
  • total bilirubin ≤ 1.5 x ULN
  • serum albumin ≥ 2.8 g/dL
  • Women and men with partners of childbearing potential must take effective contraception while on study and women of childbearing potential must have a negative pregnancy test and be non-lactating at screening

Who cannot join

  • Inability to swallow oral medications or presence of GI disorder deemed to jeopardize intestinal absorption of Cu(II)ATSM
  • Dependence of mechanical ventilation (non-invasive or invasive) for any part of day or night
  • Exposure to any other investigational agent within 3 months or two investigational agents within 6 months prior to screening visit
  • Active GI disease (except gastrointestingal reflux disease) within 30 days of screening visit
  • Known immune compromising illness or treatment
  • Presence of any of the following clinical conditions
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  • drug abuse or alcoholism
  • unstable cardiac, pulmonary, renal, hepatic, endocrine or hematologic disorder
  • active infectious disease
  • AIDS or AIDS-related complex
  • current malignancy
  • unstable psychiatric illness, defined as psychosis or untreated major depression within 90 days of screening visit
  • neuromuscular disease other than ALS/MND
  • Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
  • Use of anticoagulants at therapeutic doses within 7 days prior to screening visit
  • Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6

Trial locations and contacts

2 sites in 1 country.

Australia2 sites

  • Calvary Health Care Bethlehem

    Caulfield, Victoria, 3162

  • Macquarie University

    Sydenham, New South Wales, 2109

Other trials in Australia

About this study

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

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

Read the 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.

Being tested
Cu(II)ATSM
Medical subject headings
Amyotrophic Lateral Sclerosis, Motor Neuron Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cu(II)ATSMDrug

    Also called diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)

    copper-containing synthetic small molecule

Study groups in NCT02870634
Study groupRoleWhat they receive
Cu(II)ATSMExperimentalCu(II)ATSM capsules, administered orally once daily
Receives: Drug: Cu(II)ATSM

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities

    Measured: 24 months

Secondary outcomes (8)

  1. Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)

    Measured: 24 months

  2. Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score

    Measured: 24 months

  3. Treatment-related change in respiratory function by seated forced vital capacity (FCV)

    Measured: 24 months

  4. Treatment-related change in quality of life by ALSSQOL-R score

    Measured: 24 months

  5. Treatment-related change in disease severity by transcranial magnetic stimulation (TMS) response

    Measured: 24 months

  6. 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

    Measured: 12 months

  7. 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

    Measured: 12 months

  8. Treatment-related change in respiratory function by sniff nasal pressure (SNP) test

    Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Aug 2016 (estimated)
  2. Study start16 Nov 2016
  3. Primary completion30 Oct 2019
  4. Study completion30 Jan 2020

Resources

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02870634
Sponsor's protocol ID
CMD-2016-001
Status last verified
Mar 2020
First submitted
10 Aug 2016
Last updated
17 Mar 2020
Results posted
No
Data monitoring committee
Yes
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cu(II)ATSM in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Collaborative Medicinal Development Pty Limited at 2 sites in Australia.

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 17 inclusion and 16 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 17 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Australia. 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. 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?

recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities (time frame: 24 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02870634.

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 17 Mar 2020.

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