Safety and Tolerability of Antiretroviral (Triumeq) in Patients With Amyotrophic Lateral Sclerosis (ALS).

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02868580
• Date of registration: 16/06/2016
• Prospective Registration: Yes
• Primary sponsor: Neuroscience Trials Australia
• Public title: Safety and Tolerability of Antiretroviral (Triumeq) in Patients With Amyotrophic Lateral Sclerosis (ALS). Lighthouse
• Scientific title: Phase 2a Open Label Study, Safety and Tolerability of Combination Antiretroviral Therapy (Triumeq) in Participants With Amyotrophic Lateral Sclerosis (ALS) - The Lighthouse Project.
• Date of first enrolment: October 2016
• Target sample size: 43
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02868580
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia

Contacts

• Name: Julian Gold, MD
• Affiliation: The Albion Centre

Key inclusion & exclusion criteria

Inclusion Criteria:

Subjects must meet all of the following inclusion criteria to be eligible to participate in
this study:

- Age 18-75 years at the time of the screening visit

- Able to provide informed consent and comply with study procedures

- Sporadic ALS diagnosed as probable, laboratory-supported probable or definite
according to the World Federation of Neurology El Escorial revised criteria as
determined by a neurologist with neuromuscular sub-specialty training

- Diagnosis <24 months from date of enrolment

- (Forced) Vital capacity at least 60% of predicted value for gender, height and age at
the screening visit

- Must be on a stable dose of riluzole for at least 30 days prior to the screening
visit.

- Subject has established care with a neurologist at one of the four specialized ALS
clinics involved in the study and will maintain this clinical care throughout the
study.

- Subjects can participate in clinical registries, but will be excluded to this protocol
if they are participating in a clinical trial involving additional or investigative
treatment exposure.

Exclusion Criteria:

A participant will be excluded if he or she has any of the following:

- Dependence on mechanical ventilation at the time of screening

- Gastrostomy at the time of screening

- Absence of Upper Motor Neuron Signs

- Participation in any other investigational drug trial or using investigational drug
(within 12 weeks prior to screening)

- Known hypersensitivity to dolutegravir, abacavir or lamivudine, or to any of the
excipients

- Presence of the HLA-B*5701 allele at screening

- Presence of a monogenic cause of ALS (e.g. known mutation in SOD1, expansion in
c9orf72 etc.)

- History of positive test or positive result at screening for HIV

- Subjects positive for Hepatitis B at screening (+HBsAg), or anticipated need for
Hepatitis C virus (HCV) therapy during the study*;

- Women must not be able to become pregnant (post menopausal for >1 year, surgically
sterile, adequate contraception) or breastfeed for the duration of the study. Women of
childbearing potential must have a negative pregnancy test at screening and be
non-lactating

- Other interventional clinical trial

- Subject is taking medication contraindicated with Triumeq. Dofetilide (or pilsicainide
[available in Japan]) is prohibited as DTG may inhibit its renal tubular secretion
resulting in increased dofetilide concentrations and potential for toxicity.

- Presence of any of the following clinical conditions at the time of screening:

Drug or alcohol abuse Unstable medical disease (such as unstable angina or chronic
obstructive pulmonary disease), or active infectious disease (such as Hepatitis B or C or
tuberculosis), or current malignancy Unstable psychiatric illness defined as psychosis or
untreated major depression within 90 days of the screening visit. This exclusion criteria
is based on a prior psychiatric diagnosis that is unstable as determined by the subject's
treating Psychiatrist Dementia as previously diagnosed by a medical practitioner

• Safety Laboratory Criteria at the screening visit: Alanine aminotransferase (ALT) >5
times the upper limit of normal (ULN), OR ALT >3xULN Total bilirubin, lactate,
triglycerides, amylase, or lipase greater than 2.0 times the upper limit of normal Subject
has creatinine clearance of <50 mL/min via Cockroft-Gault method Subjects with moderate to
severe hepatic impairment (Class B or greater) as determined by Child-Pugh classification;
Absolute neutrophil count of < 1 x 109/L Platelet concentration of < 100 x 109/L
Haemoglobin < 100g/L



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Triumeq

Primary Outcome(s)

Number of participants with treatment related adverse events as defined CTCAE V4.0. [Time Frame: 1 year]

Secondary Outcome(s)

Number of participants with abnormal forced vital capacity (FVC) test results as measured by hand-held spirometry [Time Frame: One year]

Number of participants with abnormal quantitative hand muscle testing as measured by dynanometry. [Time Frame: One year]

ALS Functional Rating Scale-Revised (ALSFRS-R) scoring [Time Frame: 1 year]

Number of Participants With Abnormal Laboratory Values for Neurophysiological Index (NI) Related to Treatment. [Time Frame: 1 year]

Number of participants with abnormal Sniff Nasal Inspiratory Pressure (SNIP) Test results [Time Frame: one year]

Number of participants with abnormal scores on the Columbia Suicide Severity Score C-SSRS). [Time Frame: One year]

Secondary ID(s)

CUR-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Westmead Hosptial

Calvary Health Care Bethlehem

Macquarie University, Australia

The University of Sydney - Brain and Mind Centre