Study Purpose:
Prospective, international, randomised, double-blind, placebo controlled, multicentre, parallel group study. Patients will be randomised in a 2:1 allocation ratio to receive either IFB-088 + riluzole 100 mg or placebo + riluzole 100 mg. This clinical trial is an exploratory study, designed to show a signal of efficacy of IFB-088 through ALSFRS-R, MITOS and King's College. Respiratory function will be followed through SVC. Biomarkers and quality of life will also be evaluated throughout the study.Patients will be treated over a 6-month period. After a screening/consent visit, patients will undergo clinic visits at randomisation (V0), at 2 weeks (V1), and at months 1 (V2), 3 (V3) and 6 (V4). One week after V0, the patient will undergo urine analysis (dipstick) and blood sampling for measurement of creatinine. At the V2 visit, in addition to other assessments, patients will undergo blood sampling for PK measurements and urine sampling for crystalluria examination. Blood and urine chemistry, as well as physical examination and vital signs assessment to assess safety will be performed at each visit for safety purpose and crystalluria examination will be repeated at the follow-up visit, performed one month ± one week after V4.
Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis , ALS
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
IFB-088 50mg/day, Placebo, Riluzole 100mg/day
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Shahram Attarian, Pr, Assistance Publique Hôpitaux de Marseille (APHM) Hospital La Timone Adultes, France, Giuseppe Lauria, Pr, IRCCS Carlo Besta Institute of Milan, Italy
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Anne VISBECQ, MD / email hidden; JavaScript is required / 33 6 30 63 20 20
Study Sponsor:
InFlectis BioScience
Estimated Enrollment:
50
Estimated Study Start Date:
11 / 01 / 2022
Estimated Study Completion Date:
01 / 01 / 2025
Posting Last Modified Date:
11 / 25 / 2022
Date Study Added to neals.org:
08 / 19 / 2022
Minimum Age:
18 Years
Maximum Age:
N/A
Inclusion Criteria:1. Diagnosis of probable or definite ALS according to the revised El Escorial criteria [29], with bulbar onset of disease, familial or sporadic form,
2. Onset of symptoms ≤ 18 months prior to screening, as reported by the patient,
3. Adult males or females, aged at least 18 years old,
4. SVC > 60% of predicted value for age and sex,
5. ALSFRS-R score ≥ 36, with score 3 or 4 for item 3 (swallowing),
6. Treatment with riluzole 100 mg/day, at stable dose since at least one month and well tolerated,
7. Male or female patient of childbearing potential10 who agrees to use highly effective mechanical contraception methods (sexual abstinence, intrauterine device, bilateral tubal occlusion, vasectomised partner) throughout the study, and for 3 months after the end of the treatment,
8. Patient who read, understood and signed the ICF,
9. Patient who is willing to adhere to the study visit schedule and is capable to understand and comply with protocol requirements.
Exclusion Criteria:
1. Known other significant neurological disease(s),
2. Serious illness(es) or medical condition(s) (e.g. unstable cardiac disease, cancer, hematologic disease, hepatitis or liver failure, renal failure) that is not stabilised or that could require hospitalisation and may jeopardise the participation in the study,
3. Abnormal renal function at screening defined as estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2,
4. Abnormal liver function at screening defined as total bilirubin levels >1.5 ULN, and/or AST and/or ALT >3 ULN,
5. Neutropenia (ANC <1.5 x 109/L) at screening,
6. Other causes of neuromuscular weakness,
7. Non progressive or very rapidly progressing ALS (ALSFRS-R decline from disease onset to randomisation ≤ 0.1 / month or ≥ 1.2 / month)11,
8. Non-invasive ventilation,
9. Tracheotomy,
10. Weight loss ≥ 10% compared to weight at symptoms onset as declared by the patient or BMI <18 kg/m2 at screening,
11. Dementia or other severe active psychiatric illness, including suicidal ideation assessed using the Columbia-Suicide Severity Rating Scale (C-SSRS),
12. Patient with a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function,
13. Patient treated by edaravone for ALS,
14. Patient using unauthorised concomitant treatments, namely moderate or strong inhibitors or inducers of CYP1A2, strong inhibitors or inducers of CYP2D6 or 2C19 and strong inhibitors of OCT2, as listed in Section 6.2. Combined oral contraceptives containing ethinylestradiol are forbidden concomitant medications,
15. Smoker of > 10 cigarettes per day (e-cigarettes and nicotine patches are permitted),
16. Known hypersensitivity to any of the ingredients or excipients of the IMPs,
17. Pregnant, lactating women,
18. Patient who participated in another trial of investigational drug(s) within 30 days prior to randomisation, or 5 half-lives of the previous investigational product, whichever is longer,
19. Patient who has forfeited their freedom by administrative or legal award, or who is under guardianship or under limited judicial protection.
CHU d'Angers | Not yet recruiting
Julien CASSEREAU, Dr / 33 2 41 35 38 31 / email hidden; JavaScript is required
Angers 49100
France
CHRU Hôpital de la Cavale Blanche | Recruiting
Steeve GENESTET, Dr / 33 2 98 34 73 09 / email hidden; JavaScript is required
Brest 29200
France
Hôpital Neurologique Pierre Wertheimer | Not yet recruiting
Emilien BERNARD, Dr / 33 4 72 35 72 18 / email hidden; JavaScript is required
Bron 69677
France
CHU de Clermont-Ferrand, Hôpital Gabriel Montpied | Recruiting
Nathalie GUY, Dr / 33 4 73 75 20 43 / email hidden; JavaScript is required
Clermont-Ferrand 63003
France
Hôpital Roger Salengro - Centre SLA | Not yet recruiting
Veronique DANEL-BRUNAUD, Dr / 33 3 20 44 67 52 / email hidden; JavaScript is required
LILLE cedex 59037
France
CHU Dupuytren | Not yet recruiting
Philippe COURATIER, Pr / 33 5 55 05 65 59 / email hidden; JavaScript is required
Limoges 87042
France
APHM Hôpital La Timone Adultes SCE Maladies Neuromusculaires / SLA | Recruiting
Shahram ATTARIAN, Pr / 33 4 91 38 65 79 / email hidden; JavaScript is required
Marseille Cedex 05 13385
France
Hôpital Central | Recruiting
Sophie PITTION- VOUYOVITCH, Dr / 33 3 83 85 16 88 / email hidden; JavaScript is required
NANCY Cedex 54035
France
CHU de Nantes - Hôpital Laennec | Recruiting
Claire BOUTOLEAU BRETONNIERE, Dr / 33 2 40 16 52 81 / email hidden; JavaScript is required
Nantes 44093
France
CHU de Nice Pasteur 2-zone C | Recruiting
Marie-Hélène SORIANI, Dr / 33 4 92 03 55 04 / email hidden; JavaScript is required
NICE Cedex 1 06001
France
Hôpital Pitié-Salpêtrière | Not yet recruiting
François SALACHAS, Dr / 33 1 42 16 24 72 / email hidden; JavaScript is required
Paris 75013
France
CHU de Toulouse - Hôpital Pierre-Paul Riquet | Recruiting
Pascal CINTAS, Dr / 33 5 61 77 94 40 / email hidden; JavaScript is required
Toulouse Cedex 9 31059
France
CHU Bretonneau | Recruiting
Philippe CORCIA, Pr / 33 2 47 47 37 24 / email hidden; JavaScript is required
Tours Cedex 1 37044
France
Ospedale Civile Sant'Agostino Estense | Not yet recruiting
Jessica Mandrioli, Dr / email hidden; JavaScript is required
Baggiovara 41126
Italy
Clinica Neurologica Amaducci (tertiary motor neuron Centre) | Not yet recruiting
Isabella Simone, Dr / email hidden; JavaScript is required
Bari 70124
Italy
Centro Clinico NeMO per le Malattie Neuromuscolari | Not yet recruiting
Massimiliano Filosto, Pr / email hidden; JavaScript is required
Gussago 25064
Italy
IRCSS Istituto Neurologico Carlo Besta | Not yet recruiting
Giuseppe Lauria, Pr / email hidden; JavaScript is required
Milano 20133
Italy
AOU Università degli Studi della Campania "Luigi Vanvitelli" | Not yet recruiting
Francesca Trojsi, Dr / email hidden; JavaScript is required
Napoli 80138
Italy
Sant'Andrea Hospital Unit of Neuromuscular Disorders | Not yet recruiting
Giovanni Antonini, Pr / email hidden; JavaScript is required
Roma 00189
Italy
Centro Regionale Esperto per la Sclerosi Laterale Amiotrofica (C.R.E.S.L.A.) | Not yet recruiting
Adriano Chiò, Pr / email hidden; JavaScript is required
Torino 10126
Italy