Clinical trial NCT03488524: Open Label Extension Study of AMX0035 in Patients With ALS
Official title: Open Label Extension Study of AMX0035 in Patients With ALS
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
ALS
Lead sponsor: Amylyx Pharmaceuticals Inc.
Sponsor class: INDUSTRY
Collaborators: name: Massachusetts General Hospital Neurology Clinical Research Institute
First posted: 2018-04-05
Enrollment: 95
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This an open-label extension study to CENTAUR (AMX-3500)
Primary purpose: TREATMENT

Brief summary: This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the Centaur study.

Detailed description: The Centaur Open Label Extension Study (CENTAUR-OLE) is designed to provide longer term access to AMX0035 for patients with ALS who participated in the CENTAUR study. The study will assess longer term safety and therapeutic potential of AMX0035.

Eligibility criteria: Inclusion Criteria:

1. Completion of all visits in the randomized, double blind AMX0035 study. Subjects that receive tracheostomy or PAV during the course of the main study will still be followed as ITT until the week 24 visit before enrollment in the OLE.
2. Must enroll in the OLE within 28 days of the Week 24 visit of the main study.
3. Signed informed consent to enter the open label extension phase.

Exclusion Criteria:

1. Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.
2. Exposure to or anticipated requirement for any disallowed medication listed below.
3. Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.
4. Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study
5. Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.

Primary outcomes:
measure: Quantity of adverse events and serious adverse events observed in the study
timeFrame: 30 months
Secondary outcomes:
measure: Hospitalizations
timeFrame: 30 months
measure: Rate of Progression on the Amyotrophic Lateral Sclerosis Rating Scale Revised (ALSFRS-R)
timeFrame: 30 months
measure: Rate of Progression on ATLIS Strength Measurement
timeFrame: 30 months
measure: Rate of Progression on Slow Vital Capacity
timeFrame: 30 months
measure: Gastric Tube Frequency
timeFrame: 30 months
measure: Permanent Invasive Ventilation
timeFrame: 30 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Worcester
country: United States
facility: University of Massachusetts Memorial Medical Center
state: Massachusetts
Study officials:
References:
citation: Paganoni S, Hendrix S, Dickson SP, Knowlton N, Berry JD, Elliott MA, Maiser S, Karam C, Caress JB, Owegi MA, Quick A, Wymer J, Goutman SA, Heitzman D, Heiman-Patterson TD, Jackson C, Quinn C, Rothstein JD, Kasarskis EJ, Katz J, Jenkins L, Ladha SS, Miller TM, Scelsa SN, Vu TH, Fournier C, Johnson KM, Swenson A, Goyal N, Pattee GL, Babu S, Chase M, Dagostino D, Hall M, Kittle G, Eydinov M, Ostrow J, Pothier L, Randall R, Shefner JM, Sherman AV, Tustison E, Vigneswaran P, Yu H, Cohen J, Klee J, Tanzi R, Gilbert W, Yeramian P, Cudkowicz M. Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial. J Neurol Neurosurg Psychiatry. 2022 May 16;93(8):871-5. doi: 10.1136/jnnp-2022-329024. Online ahead of print.