SOD1 Gen Mutasyonu Olan Amyotrofik Lateral Skleroz Hastalarında RAG-17'nin Güvenliği ve Tolere Edilebilirliği

Sade dilde özet

Bu çalışma, SOD1 gen mutasyonu olan amyotrofik lateral skleroz (amyotrophic lateral sclerosis, ALS) hastalarında RAG-17 adlı tedavinin güvenliğini ve tolere edilebilirliğini incelemektedir. Çalışmaya, SOD1 gen mutasyonu doğrulanmış, 18-75 yaş arası ve akciğer kapasitesi belirli düzeyin üzerinde olan hastalar dahil edilmiştir. Erken Faz 1 aşamasında yürütülmüş ve tamamlanmıştır.

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

Deney bilgileri

Faz

Erken Faz 1 (Early Phase 1)

Durum

Tamamlandı

Sponsor

Beijing Tiantan Hospital

Müdahale

RAG-17

Başlangıç

24.05.2023

Tahmini bitiş

11.07.2024

Ülkeler

China

İlgili genler

Merkezler

• Beijing, China

Uygunluk kriterleri

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

Inclusion Criteria:

* Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle;
* 18 years old ≤ age ≤ 75 years old, males or females;
* ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported);
* Forced vital capacity ≥ 50% of predicted vital capacity during the screening period;
* Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology;
* The patient or patient's legal representative clearly understands and voluntarily participates in the study and signs the informed consent form;
* Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs.

Exclusion Criteria:

* Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation;
* Patients who have previously received or are currently receiving Tofersen treatment;
* HIV test positive or history of positive tests;
* Positive hepatitis C virus antibody or history of positive tests;
* Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody);
* Have used other investigational drugs within 1 month or within 5 drug half-lives;
* Diseases and deformities of the lumbar spine;
* Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis;
* Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent;
* With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value≥2.0 times the upper limit of normal value or AST value≥2.0 times the upper limit of normal value; severe renal insufficiency refers to CRE≥1.5 times the upper limit of normal value or eGFR\<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4);
* Permanently dependent on ventilator-assisted ventilation;
* History of alcohol and drug abuse;
* Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant;
* Patients participating in other clinical trials or using other biological agents, drugs or devices under investigation;
* Patients who have received any vaccinations within 28 days;
* Contraindications to MRI (eg, claustrophobia);
* Unable to be cooperative and complete the follow-up due to other reasons.