Current Clinical Trials

Alzheimer's disease (AD)

 

GSK Progress AD      > We are recruiting!

Official title
A phase 2, parallel group, randomized, double-blind, placebo-controlled, 3-arm, multicenter treatment study to evaluate the efficacy and safety of GSK4527226 [AL101] intravenous infusion compared with placebo in patients with early Alzheimer’s disease (AD)

Purpose of the study
To assess the efficacy and safety of GSK4527226 in participants with early AD (including Mild Cognitive Impairment (MCI) and mild dementia due to AD). GSK4527226 is an antibody which blocks sortilin and increases PGRN levels which may reduce the rate of neuronal loss and clinical decline.

Participants
Patients with a diagnosis of early AD, with a MMSE score of 21-29, who are between 50 and 85 years of age. Participants must be in good general health, other than AD.

What is involved

12 week screening period: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups and enter the treatment period.

76 weeks (1.5 years) of treatment: monthly IV administrations of drug or placebo

12 week follow-up safety period: continued assessments and safety monitoring

Throughout all stages of the trial, participants will visit us regularly in Queen Square for a number of assessments including medical and health checks, neurological examinations, brain scans, blood and urine tests, memory and thinking questionnaires, and optional lumbar punctures.

ImmunoBrain (IBC-01-01)      > We are recruiting!

Official title

A first in human study (Phase I) to evaluate the safety, tolerability and pharmacokinetics of IBC-Ab002 in persons with early Alzheimer’s disease (AD).

Purpose of the study

To investigate the safety (side effects) of a new drug, IBC-Ab002, in patients with early AD. IBC-Ab002 is an antibody which may help suppress age-related immune system decline by blocking certain immune system pathways that cause age-related impairment, slowing progression of AD.

Participants

Patients with a diagnosis of early AD, with a MMSE score of 20-28, who are between 50 and 80 years of age. Participants must be in good general health, other than AD.

What is involved?

This study will be carried out in 2 parts; Part A and Part B. In Part A, a single dose of study medication is given. Part B will begin approximately 3 months later, and 3 doses of study drug will be given 3 months apart. The drug or placebo is administered via IV infusion. Total participation in this study will include approx. 23 visits to the study site over a period of just over a year. Participants will visit us for drug/placebo administration, and also for assessments including medical and neurological examinations, memory and thinking questionnaires, blood and urine tests, brain scans (MRI and PET), and lumbar puncture.

Alnylam-EOAD (ALN-APP-001)      > recruitment paused

Official title

A phase 1 study to evaluate the safety and tolerability of ALN-APP in patients with early onset Alzheimer’s disease (AD)

Purpose of the study

To evaluate the safety (side effects) of a drug called ALN- APP. This drug may have the potential to slow disease progression by reducing production of amyloid protein, which builds up in the brains of people with AD

Participants

Patients with a diagnosis of early onset AD (onset <65 years), with a MMSE score of 21+, who are over the age of 18. Participants must be in good general health, other than AD.

What is involved?

In Part A of the study, participants will make a total of approx. 18 visits over a period of just over a year. Each visit will take approx. 3 to 6 hours depending on the procedures completed at each visit. An overnight stay may also be required. If a participant passes screening, a single dose of ALN-APP or placebo is administered, and they are closely monitored for several months afterwards. Assessments include medical and neurological examinations, memory and thinking questionnaires, blood and urine tests, brain scans (MRI and PET), and a lumbar puncture.

Part B will be a multi-dose open-label period including patients previously enrolled in Part A, as well as new participants. This will involve multiple administrations of ALN-APP (all patients will receive the active drug and there will be no placebo). The estimated duration of Part B for each participant is up to 2 years, including a 12 month dosing period (7 separate visits) and 6-12 month follow-up period (at least 2-3 visits).

Brain Shuttle (Trontinemab)      > recruitment paused

Official title
A phase 1b/2a, randomized, double-blind, placebo- controlled, multiple-ascending dose, parallel-group study to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RO7126209 following intravenous infusion in patients with prodromal or mild to moderate Alzheimer’s disease (AD)

Purpose of the study
To assess the safety and side effects of Trontinemab (RO7126209), an anti-amyloid antibody with enhanced blood-brain barrier crossing, in patients with prodromal or mild to moderate AD.

Participants
Patients with a diagnosis of Mild Cognitive Impairment (MCI) due to AD, or mild to moderate AD, with a MMSE score of 18-28, who are between 50 and 85 years of age. Participants must be in good general health, other than AD.

What is involved

Screening: consent and eligibility assessments. If passed, participants will be randomised into drug or placebo groups and enter the treatment period.

Placebo-controlled treatment: Intravenous (IV) administration of gradually increasing doses of drug (or placebo) with close safety monitoring at each dose escalation, followed by continued treatment at the determined optimum dosing regimens (or placebo). There will be a treatment period of 28 weeks, followed by a period of safety monitoring, then another treatment period of 24 weeks followed by further safety monitoring.

Open-label extension: Eligible participants may be offered continued treatment with the active drug (no placebo group) and safety monitoring for approx. 2 years.

Throughout all stages of the trial, participants will visit us regularly in Queen Square for assessments including medical and health checks, neurological examinations, brain scans, blood and urine tests, memory and thinking questionnaires, and lumbar punctures.

 

Upcoming trials

We have a number of studies currently in set-up which are due to open to recruitment in the coming weeks and months. 

• A phase 1b anti-tau gene silencing trial looking at the effects of drug on tau production and clearance in Alzheimer’s disease (AD) and Dominantly Inherited Alzheimer’s Disease (DIAD)
• A phase 1 trial investigating the safety and side effects of lowering tau protein levels in early symptomatic AD
• A phase 3 trial evaluating safety and efficacy of an anti-amyloid antibody treatment in early symptomatic AD

 

Ongoing trials      (closed to recruitment)

• INVOKE (AL002-2): A phase 2 study evaluating safety and efficacy of an antibody targeting the immune system in early Alzheimer’s disease (AD)
• DESPIAD: A phase 2b trial investigating the safety and efficacy of miridesap, a drug which reduces a protein called Serum Amyloid P Component, in mild AD
• Biogen CELIA (BIIB080): A phase 2 study assessing safety and efficacy of anti-tau drug BIIB080 in patients with Mild Cognitive Impairment (MCI) and mild AD. Biogen are investigating if reducing tau production will slow down the progression of cognitive dysfunction.

Dominantly Inherited Alzheimer’s Disease (DIAD), or familial Alzheimer’s disease (fAD)

Upcoming trials

We have studies currently in set-up which are due to open to recruitment in the coming weeks and months. 

• A phase 1b anti-tau gene silencing trial looking at the effects of drug on tau production and clearance in Alzheimer’s disease (AD) and Dominantly Inherited Alzheimer’s Disease (DIAD)
• An open label extension study evaluating impact of continued amyloid removal on disease progression in DIAD

Ongoing trials      (closed to recruitment)

• DIAN-TU-001 (E2814 Secondary Prevention Tau NexGen): A phase 2/3 trial evaluating biomarker, cognitive, and clinical endpoints following open-label lecanemab administration alongside another potential disease-modifying therapy, E2814 in patients with Dominantly Inherited Alzheimer’s Disease (DIAD).  

 

Frontotemporal dementia (FTD)

INFRONT-2 (AL001-2)      > recruitment closed

Official title
A Phase 2 Open-Label Study to Evaluate the Safety and Tolerability of AL001 in Heterozygous Carriers of Granulin or C9orf72 Mutations Causative of Frontotemporal Dementia

Purpose of the study
To see how safe and well-tolerated the experimental drug AL001 is when given intravenously to participants with a mutation in the gene for Granulin (GRN) or C9orf72, which causes frontotemporal dementia (FTD). Mutations such as GRN and C9orf72 cause accumulation of a protein called TDP-43 in the brain, leading to disruption of brain activity, and neurodegeneration. AL001 prevents the breakdown of another protein, Progranulin, which has been found to reduce TDP-43 in non-clinical studies. Therapeutics targeted at reducing TDP-43 may slow the progression of disease in FTD.

Participants
This study will enrol symptomatic and asymptomatic participants with a GRN mutation who completed the AL001-1 study, as well as symptomatic patients with a GRN or C9orf72 mutation who have not had AL001 before.

What is involved
Participants will receive up to 13 doses of AL001 over a 48 week period. Study procedures will include blood sampling, adverse event monitoring, ECG, blood pressure, physical and neurological examinations, lumbar puncture, questionnaires, and MRI. 

INFRONT-3 (Alector, AL001-3)      > recruitment closed

Official title
A Phase 3, Randomized, 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

Purpose of the study
Alector, Inc. is studying AL001 as a new experimental drug for frontotemporal dementia (FTD) caused by mutations in the progranulin gene. These mutations reduce progranulin levels in the body and may lead to symptoms of FTD. The purpose of the phase 3 study is to learn whether increasing progranulin levels with treatment with AL001 will delay onset of symptoms or slow disease progression, when compared to a placebo (a solution that contains no active AL001 drug).

Participants
Open to participants who have been diagnosed with FTD and have a progranulin gene mutation OR have a progranulin gene mutation and are at risk of developing FTD symptoms as evidenced by a biomarker.

What is involved
AL001 or placebo will be administered every 4 weeks by an intravenous (IV) infusion. Assessments will include regular medical examinations, blood tests, brain imaging (MRI), and completion of questionnaires. All participants will be in the study about 2 years and will need to visit the study site at least once a month during this time. Participants who complete the study and who meet the criteria will be eligible to continue to the optional Open Label Extension, during which all participants will receive AL001.