A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
|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: NCT04374136|
Recruitment Status : Recruiting
First Posted : May 5, 2020
Last Update Posted : July 27, 2020
|Condition or disease||Intervention/treatment||Phase|
|Frontotemporal Dementia||Drug: AL001 Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||180 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene|
|Actual Study Start Date :||July 23, 2020|
|Estimated Primary Completion Date :||October 30, 2023|
|Estimated Study Completion Date :||December 30, 2023|
AL001 every 4 weeks
Administered via intravenous (IV) infusion
Placebo Comparator: Placebo
Placebo every 4 weeks
Administered via intravenous (IV) infusion
- Evaluation of efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB [ Time Frame: Through study completion, on average up to 48 or 96 weeks ]The Clinical Dementia Rating Dementia Staging Instrument PLUS National Alzheimer's Disease Coordinating Center frontotemporal lobar degeneration Behavior & Language Domains Sum of Boxes (CDR® plus NACC FTLD-SB) is administered by a healthcare professional and based on individual ratings of the eight domains: memory, orientation, judgment and problem solving, community affairs, home and hobbies, personal care, language and behavior. Impairment is scored on a scale in which none = 0, questionable = 0.5, mild = 1, moderate = 2 and severe = 3. The 8 individual domain ratings, or "box scores", were added together to give the CDR® plus NACC FTLD-SB which ranges from 0-24. Higher score indicates severe impairment.
- Change in Clinical Global Impression-Severity (CGI-S) Score [ Time Frame: Baseline to 48 weeks ]The CGI-S is used by a clinician to rate the severity of a participant's disease relative to the clinician's past experience with patients who have the same disease using an ordinal scale ranging from 1=normal, not at all ill; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6=severely ill; and 7=among the most extremely ill patients. Higher scores indicate worsening.
- Change in Clinical Global Impression-Improvement (CGI-I) Score [ Time Frame: Baseline to 48 weeks ]The CGI-I is used by a clinician to rate how much a participant's disease has improved or worsened relative to baseline using an ordinal scale ranging from 1=very much improved; 2=much improved; 3=minimally improved; 4=no change from baseline; 5=minimally worse; 6= much worse; and 7=very much worse. Higher scores indicate worsening.
- Change in Frontotemporal Dementia Rating Scale (FRS) Score [ Time Frame: Baseline to 48 weeks ]The FRS is a 30 item scale with item responses of "All the time", "Sometimes", "Never", or "Not applicable" for each item. The total percentage score will be calculated as the number of items with response of "Never" divided by the number items that do not have response of "Not applicable". The percentage score will be converted to a logit score ranging from 5.39 to -6.66 as well as a categorized severity score of Very Mild, Mild, Moderate, Severe, Very Severe, or Profound.
- Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score [ Time Frame: Baseline to 48 weeks ]RBANS is 20 to 25 minute battery developed for cognitive assessment, detection, and characterization of dementia. RBANS includes 12 subtests that measure following 5 indices: (1)Attention Index, composed of Digit Span and Coding; (2)Language Index, consisting of Picture Naming and Semantic Fluency subtests; (3)Visuospatial/Construction Index, made up of Figure Copy and Line Orientation subtests; (4)Immediate Memory Index, composed of List Learning and Story Memory subtests, and (5)Delayed Memory Index, consisting of List Recall, List Recognition, Story Recall, and Figure Recall subtests. Completion of RBANS yields 5 index scores based on participant performance on various subtests, as well as a composite Total Index score for battery. Total index scores range from 40 to 160, and are normalized to a mean of 100 and standard deviation (SD) of 15. Higher scores indicate less impairment.
- Pharmacodynamic Biomarkers [ Time Frame: Baseline to 48 weeks ]Change in magnetic resonance imaging and blood-based biomarkers and optional CSF biomarkers (neurofilament light chain and progranulin)
- Evaluation of safety and tolerability of AL001: Incidence of adverse events [ Time Frame: Baseline to 48 weeks ]Incidence of adverse events
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): NCT04374136
|Contact: Study Leademail@example.com|
|Principal Investigator:||Peter Ljubenkov, MD||University of California, San Francisco|