Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00868166
• Date of registration: 23/03/2009
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS MITOTARGET
• Scientific title: Phase II/III, Multicenter, Randomized, Parallel Group, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) Patients Treated With Riluzole
• Date of first enrolment: April 30, 2009
• Target sample size: 512
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00868166
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Belgium, France, Germany, Netherlands, Spain, United Kingdom

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients with sporadic or familial Amyotrophic Lateral Sclerosis

- Patients with a clinical diagnosis of laboratory-supported probable, probable, or
definite ALS according to the modified El Escorial criteria8.

- Have signed an Informed Consent to participate to the trial before any study related
procedure has taken place.

- Be of age >18 (exclusive) and < 80 years (inclusive).

- If a female, not lactating, has a negative pregnancy test and agrees to use an
effective method of birth control.

- Onset of ALS Symptoms (weakness) for more than 6 months (inclusive) and less than 36
months(inclusive).

- Slow vital capacity (SVC), measured three times, one of the measure being >/= 70% of
that predicted.

- Treated with riluzole at the stable dose of 50 mg bid for at least 30 days before
enrolment.

Exclusion Criteria:

- Tracheostomy, invasive ventilation, or non invasive positive pressure ventilation
(NIPPV).

- Gastrostomy.

- Evidence of major psychiatric disorder or clinically evident dementia.

- Diagnosis of a neurodegenerative disease in addition to ALS.

- Have a current medication that could interfere with TRO19622 pharmacokinetics:
tamoxifene.

- Have current medications that could interfere with TRO19622 absorption such as
ezetimibe, bile salts chelators (cholesteramine), fibrates, phytosterols, niacin
(vitamin B3),fish oils. Have a current medication of lipid lowering agents other than
statins.

- Known hypersensitivity to any component of the study drug.

- Patients with known intolerance or contra-indication to riluzole.

- Have a recent history (within the previous 6 months) or current evidence of alcohol or
drug abuse.

- Have concurrent unstable disease involving any system eg, carcinoma other than basal
cell carcinoma, any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs
of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of
Coronary Artery Disease, or any other condition that in the opinion of the
Investigator would make the patient unsuitable for study participation.

. In Germany: Have any cardiac dysrhythmia, myocardial infarction, clinical or ECG
signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms
of Coronary Artery Disease or any cardiovascular illness known or identified at the
screening or inclusion visits, or have concurrent unstable disease involving any
system eg, carcinoma other than basal cell carcinoma or any other condition that in
the opinion of the Investigator would make the patient unsuitable for study
participation.

- Having a baseline QTc (Bazett) > 450 msec for males and > 470 msec for females.

- Patients with known hepatitis B/C or HIV positive serology.

- Be pregnant female or lactating.

- Have renal impairment defined as blood creatinine > 1:5 X upper limit of normal.

- Have hepatic impairment and/or liver enzymes (ALAT or ASAT) > 3 X ULN.

- Hemostasis disorders or current treatment with oral anticoagulants.

- Be possibly dependent on the Investigator or the Sponsor (eg, including, but not
limited to, affiliated employee).

- Participated in any other investigational drug or therapy study with a non approved
medication, within the previous 3 months.

- Patients without Social Security Insurance (France).



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Placebo Comparator

Drug: Riluzole

Drug: Olesoxime

Primary Outcome(s)

Overall Survival Rate at 18 Months [Time Frame: From the date of randomization until the date of death or last follow-up censored at 18 months (548 days)]

Secondary Outcome(s)

Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months [Time Frame: Month 18 (548 days)]

Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups [Time Frame: Inclusion, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18]

Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) [Time Frame: Inclusion, Month 1, Month 2, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18]

Slow Vital Capacity (SVC) Percent Predicted [Time Frame: Baseline, Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18]

The Single-Item Mc Gill Quality of Life Scale [Time Frame: Inclusion, Month 1, Month 3, Month 6, Month 9, Month 12, Month 15 and Month 18]

Percentage of Participants With a Global ALS FRS-R Score of <30 or Death [Time Frame: Month 18 (548 days)]

Percentage of Participants With Failure Over 18 Months [Time Frame: From randomization to the time of the first event to consider at 18 months (548 days)]

Secondary ID(s)

WN29853

EudraCT Number:2008-007320-25

TRO19622 CL E Q 1015-1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Commission

Results

• Results available: Yes
• Date Posted: 15/02/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00868166