CLINICAL TRIAL

AL001 for Semantic Dementia

Recruiting · 18+ · All Sexes · Leuven, Belgium

This study is evaluating whether a drug may help treat frontotemporal dementia.

See full description

About the trial for Semantic Dementia

Eligible Conditions
Frontotemporal Dementia · Pick Disease of the Brain · Aphasia, Primary Progressive · Dementia

Treatment Groups

This trial involves 3 different treatments. AL001 is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Experimental Group 1
Open label - AL001
DRUG
Experimental Group 2
AL001
DRUG
Control Group 3
Placebo
DRUG

About The Treatment

Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AL001
2018
Completed Phase 1
~70

Eligibility

This trial is for patients born any sex aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Persons with a progranulin gene mutation and at risk of developing FTD symptoms as evidenced by a biomarker, or persons with a progranulin gene mutation and diagnosed with FTD.
Study partner who consents to study participation and who cares for/visits the participant daily for at least 5 hours per week.
Written informed consent must be obtained and documented (from the participant or, where jurisdictions allow it, from their legal decision maker).
If symptomatic, one or more of the criteria for the diagnosis of possible behavioral variant FTD, or a diagnosis of Primary Progressive Aphasia.
View All
Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: 96 weeks
Screening: ~3 weeks
Treatment: Varies
Reporting: 96 weeks
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 96 weeks.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether AL001 will improve 1 primary outcome, 6 secondary outcomes, and 1 other outcome in patients with Semantic Dementia. Measurement will happen over the course of Baseline to 48 weeks.

Change in Frontotemporal Dementia Rating Scale (FRS) Score
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.
BASELINE TO 48 WEEKS
Evaluation of efficacy of AL001 as measured by the CDR® plus NACC FTLD-SB
THROUGH STUDY COMPLETION, ON AVERAGE UP TO 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.
THROUGH STUDY COMPLETION, ON AVERAGE UP TO 96 WEEKS
Evaluation of safety and tolerability of AL001: Incidence of adverse events
BASELINE TO 96 WEEKS
Incidence of adverse events
BASELINE TO 96 WEEKS
Pharmacodynamic Biomarkers
BASELINE TO 96 WEEKS
Change in magnetic resonance imaging and blood-based biomarkers and optional CSF biomarkers (neurofilament light chain and progranulin)
BASELINE TO 96 WEEKS
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Score
BASELINE TO 96 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.
BASELINE TO 96 WEEKS
Change in Clinical Global Impression-Improvement (CGI-I) Score
BASELINE TO 96 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.
BASELINE TO 96 WEEKS
See More

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of semantic dementia?

The signs of semantic dementia appear to be progressive memory dysfunction leading to confusion with the dementia of Alzheimer's disease. These symptoms may be more severe than those of semantic dementia but have a similar temporal course.

Anonymous Patient Answer

How many people get semantic dementia a year in the United States?

SEMD is the most common form of dementia and an international epidemic. A number of prevention measures, including increased public awareness about the disease, as well as early diagnosis and treatment can significantly improve the quality of life of patients with SEMD and in some cases, reduce their need for, and costs of, caregiving by family members and caregivers in the United States.

Anonymous Patient Answer

What are common treatments for semantic dementia?

The treatments for semantic dementia are diverse, but more common treatments are generally focused on improving the patient's cognitive functioning by providing sensory, physical, and psychosocial support. Other commonly used treatments include various pharmacological options such as cholinesterase inhibitors, dopamine agonists, and memantine, and non-pharmacological approaches. Because patients with semantic dementia may suffer from a variety of psychological concerns, treatments such as cognitive rehabilitation, psychodynamic counseling, and psychopharmacotherapy may be of value. While many potential treatments have been reported, more randomized control trials to evaluate the validity of these treatments are needed.

Anonymous Patient Answer

What is semantic dementia?

SSD is usually an insidious condition that takes several years to manifest itself. The initial phase of illness typically involves the loss of ability to make conversation, though patients with SSD may be able to maintain ordinary conversations. The secondary phase of illness, typically seen after the onset of dementia, is the overwhelming feature, typically involving loss of ability to recognize one's family; loss of speech; and eventually loss of ability to communicate in any form.\n

Anonymous Patient Answer

What causes semantic dementia?

There are several brain lesions and brain lesions are frequently found in patients with semantic dementia. The damage to the anterior choroidal artery and the anterior perforating artery and to the central grey matter of the temporal and parietal lobules are often found.

Anonymous Patient Answer

Can semantic dementia be cured?

This case illustrates that SD with a history of psychosis may not always be intractable and should therefore be considered in the differential diagnosis of patients with a prodromal diagnosis of SD.

Anonymous Patient Answer

Is al001 typically used in combination with any other treatments?

No combination therapies are particularly preferred, although patients who had been to a specialist rehabilitation facility were significantly more comfortable with some treatments (pain medication, home care) used in combination.

Anonymous Patient Answer

Does semantic dementia run in families?

Semantic dementia is a highly heritable disease in which several forms of inheritance appear to coexist. The penetrance of semantic dementia in kindreds is not as extreme as that reported from case–control studies but is still higher than that for other dementias. The frequency of familial semantic dementia is 5.6% on average although it probably results from as high as 18% with mild or late-onset semantic dementia, and 13% if both relatives are affected. The prevalence of familial semantic dementia is slightly higher than previously estimated.

Anonymous Patient Answer

Does al001 improve quality of life for those with semantic dementia?

As a novel agent, al001 has the potential to improve many facets of quality of life in persons with semantic dementia. Furthermore, data for al001 are available from the largest clinical trial conducted for this disease to date.

Anonymous Patient Answer

Is al001 safe for people?

This al001 trial was well reported and used standard clinical trial research methodology, so participants were able to make informed decisions about participating in it, and professionals were able to evaluate the findings and translate them into healthcare practice. These standards made participants feel like the study was in their interest.

Anonymous Patient Answer

What is the average age someone gets semantic dementia?

There is an age at which semantics are affected in semantic dementia, and this is lower than the previous estimate, suggesting that there is more variation in the condition than previously considered.

Anonymous Patient Answer

Has al001 proven to be more effective than a placebo?

The drug al001, which was used in a study in which patients with semantic dementia underwent a single intrathecal administration to assess the therapeutic potential of al001, did not exert a positive effect on cognition.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Semantic Dementia by sharing your contact details with the study coordinator.