Study on the Safety and Efficacy of Tominersen for Patients with Prodromal and Early Manifest Huntington’s Disease

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What is this study about?

This clinical study focuses on examining the safety and effectiveness of a medication called tominersen for people in the early stages of Huntington’s disease, a condition that affects the brain and can lead to movement, thinking, and psychiatric problems. The study includes individuals who are either in the very early phases (prodromal) or have just started showing symptoms (early manifest) of Huntington’s disease.

The purpose of the study is to assess how safely and effectively tominersen, administered as a solution for injection, works in comparison with a placebo. The study is designed to understand if tominersen can help reduce certain signs related to Huntington’s disease that are measured by changes in biomarker levels and overall patient ability to perform daily activities. Participants will undergo evaluations using clinical tests and procedures such as MRI to track changes over time.

Tominersen belongs to a group known as antisense oligonucleotides, which are designed to target and reduce specific proteins in the body, like the mutant huntingtin protein in this case. Throughout the trial, researchers will measure various aspects, including safety indicators and biomarker levels, to determine if tominersen provides any benefits for individuals with early signs of Huntington’s disease.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying the presence of the Huntington’s disease gene expansion mutation and other health criteria.

The assessment ensures that the participant meets the age, weight, and health requirements specified for the study.

2 randomization and treatment assignment

Participants are randomly assigned to receive either the active medication, tominersen, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

The medication is administered as a solution for injection directly into the intrathecal space, which is the area surrounding the spinal cord.

3 treatment administration

The treatment involves regular injections of tominersen or placebo. The frequency and dosage are determined by the study protocol and are designed to evaluate the safety and efficacy of the treatment.

Participants will receive these injections over a period of 16 months, with specific intervals as outlined in the study schedule.

4 monitoring and assessments

Throughout the study, participants undergo regular monitoring to assess the safety and effects of the treatment. This includes clinical laboratory tests, magnetic resonance imaging (MRI), and evaluations of cerebrospinal fluid (CSF) protein levels.

Additional assessments include changes in vital signs, electrocardiogram (ECG) parameters, and cognitive function tests.

5 final evaluation

At the end of the 16-month treatment period, a final evaluation is conducted to assess the overall impact of the treatment on the participant’s condition.

This includes a comprehensive review of all collected data, including any changes in the participant’s functional capacity and any adverse events experienced during the study.

Who Can Join the Study?

You must have a specific change in your genes related to Huntington’s disease. This is confirmed by a CAP score between 400 and 500.
You should either be in the early stages of Huntington’s disease, known as prodromal HD, or in the early noticeable stages, known as early manifest HD. This is determined by specific scales that measure your independence and ability to function.
Your kidney function should be at a certain level, measured by something called the estimated glomerular filtration rate, which should be at least 60 mL/min/1.73 m² in at least one of two tests.
Your total body weight should be more than 40 kg, and your body mass index (BMI) should be between 18 and 32.
You need to be between 25 and 50 years old when you agree to participate in the study.
You must have a study companion, someone who can support you during the study.

Who Cannot Join the Study?

Patients with any other serious medical condition that might interfere with the study.
Patients who have had a recent surgery or are planning to have surgery during the study period.
Patients who are currently participating in another clinical trial.
Patients who have a history of drug or alcohol abuse.
Patients who are pregnant or breastfeeding.
Patients who have a known allergy to the study medication or its ingredients.
Patients who have a significant mental health disorder that could affect their ability to participate.
Patients who are unable to comply with the study procedures or follow-up visits.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Universitaet Innsbruck	Innsbruck	Austria
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Centre Hospitalier Universitaire De Lille	Lille	France
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Oncopole Claudius Regaud	Toulouse	France

Other Sites

Site Name	City	Country
Universitaetsklinikum Aachen AöR	Aachen	Germany
Hospital De Santa Maria E.P.E.	Lisbon	Portugal
Katholisches Klinikum Bochum gGmbH	Bochum	Germany
Copernicus Podmiot Leczniczy Sp. z o.o.	Gdansk	Poland
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.	Bonn	Germany
Krakowska Akademia Neurologii Sp. z o.o.	Cracow	Poland
CNS Saude Lda.	Torres Vedras	Portugal
Universitaetsklinikum Erlangen AöR	Erlangen	Germany
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Hospital Universitario De Cruces	Barakaldo	Spain
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Rigshospitalet	Copenhagen	Denmark
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Hopital Beaujon	Clichy	France
Area De Salud De Burgos Y Soria	Burgos	Spain
Khr Iuhnhgtmzlqqvytfhbo Tteppyxmwow (wiykj	Taufkirchen/Vils	Germany
Wcgwibwj Ilyludyk Mwfdqwiy Likknquvn	Warsaw	Poland
Anooicnzxr Pyxudedm Hzxmyjdc Da Misfxkpia	Marseille	France
Hxvmwrlc Da Lx Szirc Cfhh I Sate Pzo	Barcelona	Spain
Cigjfn Hywirsikdul Rzitagmd Dtucqbprustoyq	Angers	France
Hgukjplh Upgvpbknbkqix Dw Baqraka	Badajoz	Spain
Hpdeygcb Unbbcwugkhhpyh Sxuvivcapa &exxizx Hlpnwmf da Hvzjsuxahdz	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	03.03.2023
Denmark	Not recruiting	03.03.2023
France	Not recruiting	03.03.2023
Germany	Not recruiting	03.03.2023
Italy	Not recruiting	03.03.2023
Poland	Not recruiting	03.03.2023
Portugal	Not recruiting	03.03.2023
Spain	Not recruiting	03.03.2023

Trial locations

Investigated drugs:

Tominersen

Tominersen is a medication being studied for its potential to treat individuals with prodromal and early manifest Huntington’s Disease. The trial aims to assess the safety of tominersen by monitoring adverse events and changes in clinical laboratory results, such as cerebrospinal fluid white blood cell and protein levels. Additionally, the study evaluates the medication’s effect on mutant huntingtin protein levels in the cerebrospinal fluid and its efficacy in improving symptoms and functional capacity in patients over a period of 16 months.

Investigated diseases:

Huntington's disease

Huntington’s Disease – Huntington’s Disease is a genetic disorder that affects the brain, leading to the progressive breakdown of nerve cells. It typically begins with subtle changes in mood, cognition, and movement, which gradually worsen over time. As the disease progresses, individuals may experience involuntary movements, difficulty with coordination, and cognitive decline. Early stages might include irritability, depression, and small involuntary movements. Over time, these symptoms become more pronounced, affecting daily activities and quality of life. The disease is caused by a genetic mutation and is inherited in an autosomal dominant pattern.

Trial ID:

2023-503928-10-00

Protocol code:

BN42489

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Efficacy of Tominersen for Patients with Prodromal and Early Manifest Huntington’s Disease

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