Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis (FAIR-ALS II)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03293069|
Recruitment Status : Recruiting
First Posted : September 26, 2017
Last Update Posted : May 21, 2019
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Deferiprone Drug: Placebo Oral Tablet||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||240 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis: Multicentre, Parallel-group, Placebo-controlled, Randomized Clinical Trial of Deferiprone|
|Actual Study Start Date :||January 30, 2019|
|Estimated Primary Completion Date :||March 2022|
|Estimated Study Completion Date :||April 2022|
Half of participants will receive twice-daily oral deferiprone taken over 12 months.
One 600 mg delayed-release tablets of deferiprone twice a day, for at 30 mg/kg/day
Other Name: DFP
Placebo Comparator: Placebo
Half of participants will receive the placebo Twice-daily oral placebo taken over 12 months
Drug: Placebo Oral Tablet
the placebo twice daily morning and evening.
- CAFS score (Combined Assessment of Function and Survival) [ Time Frame: at 12 months ]CAFS score based on changes in amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) total scores and time to death from baseline (randomization visit) to 12 months
- Changes in ALS Functional Rating Scale-Revised (ALSFRS-R) total score [ Time Frame: Baseline, at 12 months ]
- All-cause and respiratory insufficiency mortality [ Time Frame: at 12 months ]Time to death for all cause or respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation for ≥ 23 h per day for 14 consecutive days) from baseline until 12 month
- Changes in muscle strength [ Time Frame: Baseline, at 12 months ]Muscle strength measurements are determined by the overall mega-score for handheld dynamometry and manual muscular testing with a validated medical device provided
- Change in the slow vital capacity [ Time Frame: Baseline, at 12 months ]The slow vital capacity is measure by the maximum amount of air that can be exhaled following a deep breath. Reflecting the Respiratory insufficiency.
- Changes in body weight [ Time Frame: Baseline, at 12 months ]
- Change in Quality of life [ Time Frame: Baseline, at 12 months ]Quality of life assessed using the five-item form of the ALS assessment questionnaire (ALSAQ-5) from baseline to 12 months
- DSMIV criteria [ Time Frame: at 12 months ]Dementia (yes/no)
- Fronto-Temporal Dementia (FTD) criteria [ Time Frame: at 12 months ]Using the revised guidelines for the diagnosis of behavioral variant frontotemporal dementia based on recent literature and collective experience by Rascovsky K et al 2011; Lamarre AK et al 2013
- Change in Montreal Cognitive Assessment (MoCA) [ Time Frame: Baseline, at 12 months ]MoCA evaluated of mild cognitive dysfunction.
- Change in Edinburgh Cognitive and Behavioural Amyotrophic Lateral Sclerosis Screen (ECAS) [ Time Frame: Baseline, at 12 months ]
ECAS determine cognitive and behavioral changes of patients suffering from Amyotrophic Lateral Sclerosis.
With ECAS, ALS-specific (fluency, executive functions and social cognition, language) and ALS-nonspecific (memory, visuospatial functions) functions can be analyzed to enable the distinction from other diseases with cognitive and behavioral impairments.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03293069
|Contact: David Devos, MD,PhD||3 22.214.171.124. ext +firstname.lastname@example.org|
|Hôpital Roger Salengro, CHU||Recruiting|
|Lille, France, 59000|
|Contact: DAVID DEVOS +33 320446243|
|Contact: PAULINE GUYON +33 320446711 email@example.com|
|Principal Investigator: David Devos, MD,PhD|
|Principal Investigator:||David Devos, MD,PhD||University Hospital, Lille|