Phase 3
Completed N=512
Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS
Source: ClinicalTrials.gov NCT00868166 ↗Enrolled (actual)
512
Serious AEs
26.0%
Results posted
Feb 2019
Primary outcomePrimary: Overall Survival Rate at 18 Months — 67.5; 69.4 percentage of partcipants — p=0.71
◆ Published Evidence
Highly cited
136citations · ~19 / year
A ferroptosis-based panel of prognostic biomarkers for Amyotrophic Lateral Sclerosis.
Summary
The purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month survival rate.
Linked Publications (2)
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A ferroptosis-based panel of prognostic biomarkers for Amyotrophic Lateral Sclerosis.
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Neurofilament light and heterogeneity of disease progression in amyotrophic lateral sclerosis: development and validation of a prediction model to improve interventional trials.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival Rate at 18 Months |
67.5; 69.4 | 0.71 |
| SECONDARY Percentage of Participants With Failure Over 18 Months |
67.1; 65.5 | 0.83 |
| SECONDARY Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R) |
39.1; 38.2; 38.2; 37.2; 37.6; 36.7 | — |
| SECONDARY Percentage of Participants With a Global ALS FRS-R Score of <30 or Death |
28.2; 24.9 | 0.21 |
| SECONDARY Slow Vital Capacity (SVC) Percent Predicted |
93.1; 93.1; 89.7; 89.7; 84.8; 85.7 | — |
| SECONDARY Percentage of Participants With SVC Percent Predicted <70% or Had Died Over 18 Months |
28.9; 31.9 | 0.56 |
| SECONDARY Global Score of Manual Muscle Testing (MMT) of 34 Muscle Groups |
128; 126; 121; 120; 117; 114 | — |
| SECONDARY The Single-Item Mc Gill Quality of Life Scale |
6.51; 6.47; 6.17; 6.27; 5.83; 5.75 | — |
Eligibility 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 /= 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).
Data sourced from ClinicalTrials.gov (NCT00868166) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.