A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)
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|ClinicalTrials.gov Identifier: NCT04768972|
Recruitment Status : Recruiting
First Posted : February 24, 2021
Last Update Posted : April 9, 2021
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: ION363 Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||64 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS)|
|Estimated Study Start Date :||April 2021|
|Estimated Primary Completion Date :||March 2024|
|Estimated Study Completion Date :||March 2024|
ION363 will be administered by lumbar intrathecal (IT) bolus injection beginning with 1 dose every 4 weeks followed by 1 dose every 8 weeks over a 29-week double-blind treatment period and every 8 weeks for 73 weeks in the open-label extension treatment period.
ION363 will be administered by IT bolus injection.
Placebo Comparator: Placebo
Placebo will be administered by lumbar IT bolus injection beginning with 1 dose every 4 weeks followed by 1 dose every 8 weeks a 29-week double-blind treatment period.
Placebo will be administered by IT bolus injection.
- 1. Change from Day 1 through Study Day 253 in Part 1 in functional impairment [ Time Frame: Baseline, Day 253 in Part 1 ]Functional impairment to be measured by joint rank analysis of the combined assessment of: In-clinic Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Total Score, time of rescue or discontinuation from Part 1 and entering Part 2 due to a deterioration in function, and Ventilation Assistance-free survival (VAFS)." ALSFRS-R measures functional disease severity. The scale measures four functional domains, bulbar function, gross motor skills, fine motor skills, and respiratory. The assessment will contain 12 questions scored from 0 (no function) to 4 (full function), with a total possible score of 48, which will indicate the highest level of function. ALSFRS-R will be a part of the combined assessment of joint rank analysis to assess efficacy in Part 1.
- Change From Baseline in Amyotrophic Lateral Sclerosis Specific Quality of Life - Revised (ALSSQOL-R) Score to Day 253 in Part 1 [ Time Frame: Baseline, Day 253 in Part 1 ]The ALSSQOL-R is a disease-specific 50-item assessment that measures the quality of life (QoL). Each item will be rated by the participant on a scale of 0 to 10, with 0 being the least desirable situation and 10 being the most desirable.
- Survival [ Time Frame: Day 253 in Part 1 ]
- Change From Baseline in In-clinic Slow Vital Capacity (SVC) to Day 253 in Part 1 [ Time Frame: Baseline, Day 253 in Part 1 ]
- Change From Baseline in Handheld Dynamometry (HHD) to Day 253 in Part 1 [ Time Frame: Baseline, Day 253 in Part 1 ]
- Change From Baseline in Neurofilament Light (NfL) Concentration in Cerebrospinal Fluid (CSF) to Day 253 [ Time Frame: Baseline, Day 253 in Part 1 ]
- Change From Baseline in Fused in Sarcoma (FUS) Protein Concentration in CSF to Day 253 [ Time Frame: Baseline, Day 253 in Part 1 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04768972
|Contact: Ionis Pharmaceuticalsfirstname.lastname@example.org|
|United States, New York|
|Columbia University Medical Center||Recruiting|
|New York, New York, United States, 10032|
|Contact: Sonya Aziz-Zaman 646-799-2175 email@example.com|