Erken Belirti Gösteren Huntington Hastalığı Olan Yetişkinlerde AB-1001'in Striatuma Uygulanmasını Değerlendiren Çalışma

Sade dilde özet

Bu çalışma, AB-1001 adlı bir gen tedavisinin, beynin striatum bölgesine uygulanarak erken belirti gösteren Huntington hastalığı (Huntington's disease) olan yetişkinlerde güvenliliğini ve etkisini inceliyor. Çalışmaya, huntingtin geninde patolojik CAG genişlemesi (≥40) genetik olarak doğrulanmış, 18-65 yaş arası hastalar alınmaktadır. Çalışma Faz 1/Faz 2 aşamasında yürütülmekte; şu an katılımcı almamakta ancak devam etmektedir (active, not recruiting).

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Devam ediyor, katılım kapalı

Sponsor

AskBio France, SAS, a subsidiary of AskBio Inc.

Müdahale

AB-1001 Gene Therapy

Başlangıç

12.10.2022

Tahmini bitiş

01.04.2028

Ülkeler

France

İlgili genler

Merkezler

• Paris, France

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria:

* Male or Female subjects between ages 18 and 65 years (both inclusive) at time of consenting, able to provide Informed Consent and able to understand and comply with all study procedures.
* Documented genetic confirmation of pathological CAG expansion in the huntingtin gene ≥40.
* Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression.
* Striatal MRI volumes per hemisphere: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 (per side) on Screening MRI.
* All HD concomitant medications stable for at least 30 days prior to screening at the investigator's discretion.

Key Exclusion Criteria:

* Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, would impact subject's safety and compliance with the study procedures.
* Metastatic neoplasms within the five years prior to screening.
* Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator's clinical judgment.
* Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization.
* History of prior suicide attempt or imminent risk of self-harm based on investigator's judgment or with a "yes" answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS).
* Patients with history of confirmed stroke, known intracranial neoplasms, vascular malformations, or intracranial hemorrhage.
* Subjects not deemed suitable for the surgical procedure as per the Neurosurgeon's judgment.
* Any history of gene therapy, cell transplantation or any other experimental brain surgery.
* Any RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides within 6 months prior to screening.
* Subjects unable to tolerate or unwilling to undergo multiple lumbar punctures.
* Participation in any clinical trial of an approved or non-approved investigational drug or intervention within 12 weeks or 5 half-lives whichever is longer prior to treatment.