Study on the Safety and Effects of PTC518 for Patients with Huntington’s Disease

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What is this study about?

This clinical trial is focused on studying Huntington’s Disease, a genetic disorder that causes the progressive breakdown of nerve cells in the brain. The study is testing a new treatment called PTC518, which is taken as a tablet. The purpose of the study is to evaluate the safety of PTC518 and its effects on reducing a specific protein in the blood associated with Huntington’s Disease. Participants in the study will receive either the PTC518 tablet or a placebo, which looks like the PTC518 tablet but does not contain the active medication.

The study will be conducted over a period of 12 months. During this time, participants will take the medication orally and will have regular check-ups to monitor their health and the effects of the treatment. These check-ups will include various assessments, such as blood tests and other evaluations, to track changes in the levels of the protein related to Huntington’s Disease. The study aims to gather information on how well PTC518 works and its safety profile.

By participating in this study, researchers hope to gain valuable insights into the potential benefits of PTC518 for individuals with Huntington’s Disease. The ultimate goal is to find a treatment that can help manage the symptoms and progression of this condition. The study is designed to ensure the safety and well-being of all participants throughout the research process.

1 joining the study

Upon joining the study, participation is confirmed through informed consent. This involves understanding the study’s purpose and agreeing to follow the study’s requirements.

Eligibility is based on specific criteria, including age, genetic confirmation of Huntington’s disease, and certain health scores.

2 randomization and initial assessment

Participants are randomly assigned to receive either the study medication, PTC518, or a placebo. This ensures that the study results are unbiased.

Initial assessments are conducted to establish baseline health data. This includes blood tests and other evaluations to measure health status before starting the medication.

3 medication administration

Participants take the assigned medication orally in tablet form. The dosage and frequency are determined by the study protocol.

The study medication, PTC518, is administered in varying doses to evaluate its safety and effectiveness.

4 ongoing monitoring and assessments

Regular monitoring is conducted to track health changes and any side effects. This includes laboratory tests, vital signs checks, and other health assessments.

Participants undergo periodic evaluations to measure changes in specific health markers, such as blood protein levels related to Huntington’s disease.

5 final assessments and study completion

At the end of the study period, final assessments are conducted to evaluate the overall effects of the medication.

The study aims to determine changes in health markers from the beginning to the end of the study, including safety and efficacy outcomes.

Who Can Join the Study?

Must be a male or female who can walk and is aged 25 years or older.
Must be willing and able to give consent and follow all study rules.
Must have a confirmed diagnosis of Huntington’s Disease with a specific genetic marker called CAG repeat length between 40 and 50. This can be checked through a lab test.
Must have a UHDRS IS score of 100. This is a score from a test that measures certain abilities in people with Huntington’s Disease.
Must have a UHDRS TFC score of 13. This is another score from a test that measures daily living skills in people with Huntington’s Disease.
Must have a score between 0.18 and 4.93 on a special index called the HD prognostic index. This index helps predict the progression of Huntington’s Disease.
Women who can have children must agree to use very effective birth control methods while taking the study medication and for 6 months after stopping it. This includes women who are not yet menopausal or have not had surgery to prevent pregnancy.
Men who are sexually active and can father children must agree to use a condom during sex while taking the study medication and for 6 months after stopping it. This is to prevent passing the drug through semen.
For another group in the study, must have a UHDRS TFC score of 11 or 12, or a score of 13 with a UHDRS IS score of less than 100.

Who Cannot Join the Study?

Individuals who do not have a confirmed diagnosis of Huntington’s Disease cannot participate. Huntington’s Disease is a genetic disorder that affects the brain.
Participants must be within a specific age range, typically adults, to be eligible.
Both males and females are eligible, but certain health conditions or factors may exclude them.
Individuals who are considered part of a vulnerable population, such as those unable to give informed consent, may be excluded.
Participants must not have any other medical conditions or be taking medications that could interfere with the study.
Pregnant or breastfeeding women are usually excluded to avoid any potential risks to the baby.
Individuals with a history of severe allergic reactions to any of the study medications or similar drugs may be excluded.
Participants must not have participated in another clinical trial recently, as this could affect the study results.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Medizinische Universitaet Innsbruck	Innsbruck	Austria

Other Sites

Site Name	City	Country
Casa Sollievo Della Sofferenza	San Giovanni Rotondo	Italy
Katholisches Klinikum Bochum gGmbH	Bochum	Germany
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Hospital Universitario De Cruces	Barakaldo	Spain
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH	Ulm	Germany
Area De Salud De Burgos Y Soria	Burgos	Spain
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
George-Huntington-Institut GmbH	Munster	Germany
Aufgeblove Phebdknt Hwxkzoqi Do Mzcxniejy	Marseille	France
Hbpuumip Ds Lg Slper Csvo I Szpi Pof	Barcelona	Spain
Ldbdb Uspbrtxwzoty Mcgxcbr Ckxjxsj (ygfls	Leiden	The Netherlands
Cqmmkr Hkuhgsjmdjw Rcjkeppu Dojaayjeegfncg	Angers	France

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	20.04.2022
France	Not recruiting	20.04.2022
Germany	Not recruiting	20.04.2022
Italy	Not recruiting	20.04.2022
Spain	Not recruiting	20.04.2022
The Netherlands	Not recruiting	20.04.2022

Trial locations

Investigated drugs:

Ptc518

PTC518 is a medication being studied for its potential to treat Huntington’s disease. The trial aims to assess the safety of this medication and its ability to lower the levels of a specific protein called total huntingtin (tHTT) in the blood. This protein is associated with the progression of Huntington’s disease, and reducing its levels may help manage the condition.

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 in middle adulthood and is characterized by movement disorders, cognitive decline, and psychiatric symptoms. As the disease progresses, individuals may experience involuntary movements, difficulty with coordination, and changes in mood and behavior. Cognitive abilities, such as thinking and reasoning, gradually deteriorate, impacting daily functioning. The disease is caused by a mutation in the HTT gene, which leads to the production of an abnormal form of the huntingtin protein. Over time, the accumulation of this protein damages specific areas of the brain, leading to the symptoms observed in affected individuals.

Trial ID:

2023-509835-26-00

Protocol code:

PTC518-CNS-002-HD

NCT ID:

NCT05358717

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effects of PTC518 for Patients with Huntington’s Disease

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