Phase I/II Study of Gene Therapy for Sporadic ALS

JRCT ID: jRCT2033210686

Registered date:22/03/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Sporadic amyotrophic lateral sclerosis (Sporadic ALS)
• Date of first enrollment: 15/07/2022
• Target sample size: 6
• Study type: Interventional
• Intervention(s): AAV.GTX-ADAR2 is designed to be injected into the medullary cavity of the Sporadic ALS patient.

Outcome(s)

• Primary Outcome: To examine the tolerability, safety, and efficacy in patients with Sporadic ALS, after medullary cavity administrations of AAV.GTX-ADAR2.

Key inclusion & exclusion criteria

• Age minimum: >= 40age old
• Age maximum: < 79age old
• Gender: Both

Include criteria

-Patients aged over 40 years and less than 79 years at the time of obtaining informed consent.
-Patients diagnosed with Clinically Definite ALS, Clinically Probable ALS, or Clinically Probable-Supported ALS according to the revised El Escorial Airlie House diagnostic criteria.
-No relatives diagnosed with ALS (sporadic ALS).
-Revised ALS Functional Rating Scale (ALSFRS-R) at the start of the preliminary observation period Subjects with score of 37 or more and no score of 2 or less in any of the items. However, patients scored 4 for "12. Respiratory failure".
-Patients within 2 years after onset of ALS at the time of informed consent.
-Patients whose total ALSFRS-R score decreased by at least 2 points during the preliminary observation period (12 weeks).

Exclude criteria

-Patients undergoing tracheostomy.
-Patients who have had non-invasive ventilator support devices associated with ALS.
-Patients with a % FVC (forced vital capacity) of 60% or less.
-Patients with any item on the Modified Norris Scale (MNS) Symptom Scale that scores less than 2 at the start of the preliminary observation period.
-Patients with chronic obstructive pulmonary disease (COPD).
-Patients with hepatic dysfunction who meet any of the following criteria within 3 days before administration of the investigational product.
1.AST or ALT: not less than 3 times above the upper limit of normal.
2.Total bilirubin in blood: 1.5 times or more from the upper limit of normal.
-Patients who have newly started taking riluzole 2 weeks before the start of the preliminary observation period or those who have changed or discontinued the dosage and administration.
-Patients taking peran panel hydrate, ropinirole hydrochloride, bosutinib hydrate, or edaravone within 4 weeks before the start of the preliminary observation period.
-Patients with clinically suspected cognitive impairment.
-Patients who cannot undergo MRI. (e.g., patients with metal in their bodies, patients with tattoos, patients with claustrophobia, etc.)
Patients with respiratory disease, cardiovascular disease, liver disease or renal disease grade 3 or higher according to CTCAE ver. 5.0.
Patients with malignancies.
Patients with a history of hypersensitivity or complications to the ingredients of this product or those used in the manufacturing process of this product.
-Patients with uncontrolled diabetes mellitus.
-Patients with the following backgrounds that make lumbar puncture difficult.
1.Intracranial hypertension.
2.Degenerative diseases of the lumbar spine.
3.Bleeding tendency.
-Patients with or suspected of having meningitis, ventriculitis, lumbar skin infection, bacteremia, or sepsis.

Related Information

• Primary Sponsor: Morita Mitsuya
• Secondary Sponsor: Gene Therapy Research Institution Co., Ltd.