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Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

The REVEAL Pediatric Study is an early-stage clinical trial investigating a new gene therapy called TSHA-102 for young girls with Rett Syndrome, aged between 5 and 8 years old. This study is being conducted in multiple medical centres and is 'open-label', meaning both the doctors and the families will know which treatment is being given. Researchers want to understand if TSHA-102 is safe, how well children tolerate it, and if it shows any signs of improving their condition. They will look at two different dose levels of the treatment. The study will follow children for up to six years to monitor the long-term effects. This research could help bring new treatment options for girls living with Rett Syndrome.

Auf einen Blick

Status
Aktiv, aber nicht rekrutierend
Phase
PHASE1, PHASE2
Sponsor
Taysha Gene Therapies, Inc.
Einschreibeziel
6
Starten
12 Dec 2023
Geschätzte Fertigstellung
02 Nov 2031
Rett Syndrome

Worum geht es in dieser Studie?

This study, called the REVEAL Pediatric Study, is focusing on a new type of medicine called a gene therapy, known as TSHA-102. Rett Syndrome is a serious genetic condition that affects the brain and mostly impacts girls. It's caused by a faulty MECP2 gene, which is important for brain development. Gene therapy aims to correct this problem by delivering a healthy version of the gene.

The main goal of this particular study is to find out if TSHA-102 is safe for girls with Rett Syndrome and how well their bodies cope with the treatment. Researchers will also be looking for any signs that the treatment might be helping with the symptoms of Rett Syndrome. Because this is an early-stage study, it's about gathering important information on safety and initial effectiveness, rather than being a full test of whether the medicine works perfectly.

The study is designed to involve girls aged 5 to 8 years old. They will explore two different amounts, or 'dose levels', of the TSHA-102 treatment. By studying these different doses, the researchers hope to learn which amount might be best. The children taking part will be looked after and monitored for quite a long time – up to six years – to understand the full picture of how the treatment affects them over time. This long-term follow-up is very important for understanding any potential lasting effects.

Wichtige Erkenntnisse

  • This study is for girls aged 5-8 with a specific diagnosis of Rett Syndrome.
  • It tests a new gene therapy called TSHA-102, given via a spinal injection.
  • The main goals are to check the treatment's safety and early effectiveness.
  • Participation involves regular medical checks and assessments for up to six years.
  • There's no guarantee of benefit, and potential risks will be discussed fully.

Wer könnte berechtigt sein?

To be considered for this study, a girl must have a clear diagnosis of a specific type of Rett Syndrome (called 'classical' or 'typical') caused by a confirmed fault in the MECP2 gene. She needs to be between 5 and 8 years old when she's first included in the study. All her routine vaccinations must be up to date, and she must have had her last vaccination at least 42 days before starting the study treatment. Her parent or guardian must also agree to allow her to receive blood transfusions if needed during the study for safety reasons.

There are also some reasons why a girl might not be able to join. For example, if she has another brain condition or genetic illness that causes different or ongoing problems, or if she had significant developmental delays very early in her life (before 6 months old). Girls with a different type of Rett Syndrome ('atypical') or those whose MECP2 gene fault doesn't cause Rett Syndrome are also not eligible. Additionally, if a girl needs help breathing with a machine, has certain serious infections like hepatitis B or C, or has other medical conditions that would make the study treatment or necessary procedures unsafe for her, she wouldn't be able to participate.

Könnte diese Studie für Sie geeignet sein?

Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.

  1. Is your daughter a girl aged between 5 and 8 years old?
  2. Does she have a confirmed diagnosis of classical/typical Rett Syndrome, with a known MECP2 gene fault?
  3. Are her routine vaccinations all up to date?
  4. Does she NOT have other serious underlying health conditions or brain injuries that cause problems?
  5. Does she NOT require breathing support from a machine?
Beantworten Sie jede Frage, um Ihr Ergebnis zu sehen.

Was beinhaltet die Teilnahme?

If a child takes part in this study, they will receive the investigational gene therapy, TSHA-102. This treatment is given directly into the fluid surrounding the brain and spinal cord through a procedure called a lumbar puncture. Before and after receiving the treatment, the child will have various medical appointments and tests. These will include physical examinations, blood tests, and assessments to see how the treatment might be affecting their symptoms and overall health. The study involves regular check-ups and monitoring over a long period, up to six years. Over this time, healthcare professionals will carefully watch for any side effects and will assess any changes in the child's development and abilities. The specific schedule of visits and tests will be explained in detail by the study team.

Potenzielle Risiken und Vorteile

Participating in a study like this means you're helping researchers learn more about new treatments. A potential but unproven benefit is that TSHA-102 might help improve your child's Rett Syndrome symptoms or slow down its progression. However, because this is an early-stage study, there's no guarantee of direct benefit for your child. Like all medical treatments and procedures, there are potential risks, including side effects from the gene therapy itself, risks associated with the way it's given (spinal injection), and risks from other medications used in the study. These risks will be fully explained by the study team. You always have the right to withdraw your child from the study for any reason, at any time, without it affecting their ongoing medical care.

Standorte (5)

  • Rush University Medical Center & Children's Hospital
    Verifizierte Postleitzahl
    Chicago, Vereinigte Staaten
  • Gillette Children's Specialty Healthcare
    Verifizierte Postleitzahl
    Saint Paul, United States
  • Washington University, St. Louis
    Verifizierte Postleitzahl
    St. Louis, Vereinigte Staaten
  • CHU Ste-Justine
    Verifizierte Postleitzahl
    Montreal, Kanada
  • Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust
    Verifizierte Postleitzahl
    London, Vereinigtes Königreich

Häufige Fragen

Was ist Gentherapie?

Gene therapy is a new type of medicine that aims to correct problems caused by faulty genes. In this study, it's designed to provide a healthy copy of the MECP2 gene.

What does 'Phase 1/2' mean?

Phase 1 and 2 are early stages of clinical trials. Phase 1 focuses on safety, and Phase 2 looks at both safety and if the treatment shows signs of working. This study combines both stages.

Will my child get the actual treatment or a dummy treatment?

In this specific study, it's an 'open-label' design, meaning all participants will receive the active investigational treatment, TSHA-102, at one of the two doses being tested.

Wie lange wird die Studie dauern?

Children who join this study will be monitored for up to six years to understand the long-term effects of the treatment. There will be regular check-ups during this time.

Can we stop participating if we change our minds?

Yes, you can withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

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