Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Plain-language summary

This study examined the safety and tolerability of the treatment RAG-17 in patients with amyotrophic lateral sclerosis (ALS) who have a SOD1 gene mutation. It included patients aged 18 to 75 with a confirmed SOD1 gene mutation and lung capacity above a set level. It was conducted in Early Phase 1 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Early Phase 1

Status

Completed

Sponsor

Beijing Tiantan Hospital

Intervention

RAG-17

Start date

5/24/2023

Estimated completion

7/11/2024

Countries

China

Related genes

Locations

• Beijing, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.