NCT04885114Visszavonva1. fázis
Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease
A Phase 1B, Open-label, Randomized, Controlled, Multicenter, Dose Escalation Study of the Safety, Tolerability, and Biological Effects of VY-HTT01 Administered Via Intraparenchymal Infusion of the Putamen and Thalamus in Adults With Huntington's Disease
Forrás: ClinicalTrials.gov · Utoljára frissítve: 2021. aug. 16.
Megadott ok: We have discontinued our 1st generation HD program with the VYTAL Study \& have initiated a 2nd generation program using a novel, proprietary AAV capsid that may enable intravenous administration \& achieve widespread distribution to affected tissue.
Rövid összefoglaló (regiszter, angolul)
This is the first clinical study of VY-HTT01, a gene therapy for early-stage Huntington's Disease (HD) patients. The primary goal of this trial is to evaluate the safety and tolerability of VY-HTT01. This study is a first in human study, Phase 1b, open-label, randomized, multicenter, dose escalation study with a delayed treatment control arm.
- Állapot (regiszter)
- Visszavonva
- Fázis (regiszter)
- 1. fázis
- Vizsgálattípus
- Beavatkozással járó
- Résztvevők száma
- 0 (tényleges)
- Első benyújtás
- 2021. márc. 2.
- Kezdés
- 2021. júl. 30.
- Elsődleges befejezés
- 2024. dec. 30.
- Fő szponzor
- Voyager Therapeutics
- Betegségek
- Huntington-kór
- NCT-szám
- NCT04885114
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Korábbi kezelés
- Started or changed dose of a concomitant CNS medication within 30 days.
- Had prior neurosurgical procedures that could complicate the study procedures.
- Have used any investigational therapies within 30 days prior to Screening, oligonucleotide therapies within 9 months prior to Baseline, or any prior gene therapy.
- Male or female with reproductive capacity and is unwilling to use highly effective contraception for 12 months after surgery.
Más betegség
- Have any significant structural or degenerative neurologic disease other than HD.
- Have any chronic disability, significant systemic illness and/or, unstable medical condition, or clinical findings noted.
- Have primary or secondary immune-compromise due to infections or medical conditions or chronic therapies.
- Have contraindications to lumbar puncture or increased risks of bleeding upon surgery.
- Have contraindications to MRI such as claustrophobia, embedded metal in the body, or known allergy or intolerance to contrast agents.
Kiket keres a vizsgálat
- Must be at least 18 years old.
- Have CAGn repeat >39.
- Have diagnostic confidence score of 4 based on motor, cognitive, or behavioral symptoms.
- Have a TFC score of 13 to 11.
- Have stable dosing of neurological and psychiatric medications.
- Capable of giving informed consent.
- Able to comply with all procedures and study visits.
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