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Home » Find Clinical Trials » A Phase 1b/2a first-in-human, multicentre, randomized, double-blind, placebo-controlled, multiple ascending dose followed by an open-label extension of S233107 in participants with spinocerebellar ataxia type 3

A Phase 1b/2a first-in-human, multicentre, randomized, double-blind, placebo-controlled, multiple ascending dose followed by an open-label extension of S233107 in participants with spinocerebellar ataxia type 3

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Servier Protocol Code: S233107-284

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How to participate in this study
If you think you are eligible for this study (see
eligibility criteria
below), you can identify the location closest to you and contact them directly. If you can’t find a location close to you, please contact Institut de Recherches Internationales Servier (I.R.I.S.)
Name: Institut de Recherches Internationales Servier, Département des études cliniques
Phone number: +33 1 55 72 60 00
The study has 8 locations
More centres in the US and Japan will be added soon.

Study description

This study is needed to find a specific treatment for a type of degenerative disease called spinocerebellar ataxia type 3 (SCA3). Symptoms of ataxia are unsteady walking, balance, and speech problems.

SCA3 is caused by changes in a gene called ATXN3. These changes lead to a faulty protein called Ataxin-3, which disrupts how brain cells work.

The study drug, S233107, is designed to reduce the amount of the faulty version of the Ataxin-3 protein made by the body. Researchers believe that S233107 may help to treat patients with SCA3. This study is important because it could lead to a treatment that prevents the disease from worsening in patients with SCA3.

Official title: A Phase 1b/2a first-in-human, multicentre, randomized, double-blind, placebo-controlled study of multiple ascending dose (Partl) followed by an open-label extension (Part 2) to assess the safety, tolerability, and pharmacokinetics of intrathecally administered 5233107 in participants with spinocerebellar ataxia type 3
Conditions
Spinocerebellar Ataxia Type 3 SCA3
Interventions / Treatments
  • S233107
Other study id numbers
  • S233107-284

Eligibility Criteria

Eligible age for the study

18 years to 64 years (Adult)

Sex

Male/Female

Accepts Healthy Volunteers

No

To take part, participants have to:

  • Have a confirmed diagnosis of SCA3 through genetic test.
  • Be an adult aged 18 to 65 years.
  • Be able to walk on their own without any support.

Participants cannot take part if they have other types of brain disorders, major reduced kidney function, or a history or presence of significant suicide risk.

How is the study designed?

Allocation
Randomized
Interventional study model
Parallel
Participant Group / Arm
Experimental: S233107 Part 1

Small groups of participants will receive different doses of S233107. The first group receives the lowest dose, then each new group receives a higher dose.

Intervention / Treatment
Drug: S233107
Participant Group / Arm
Placebo Comparator: Placebo Part 1

Small groups of participants will receive placebo. A placebo looks like S233107 but does not contain any real medicine.

Intervention / Treatment
Drug: Placebo
Participant Group / Arm
Experimental: S233107 Part 2

Participants who complete Part 1 will receive S233107 until the end of the study. Participants will continue to have regular check-ups for any unwanted medical events.

Intervention / Treatment
Drug: S233107

Keywords

Provided by Servier
Spinocerebellar Ataxia Type 3 SCA3