Clinical trial NCT06493279: Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
Official title: A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis Als
Lead sponsor: RJK Biopharma Ltd
Sponsor class: INDUSTRY
Collaborators: name: Peking University Third Hospital
First posted: 2024-07-09
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).


Eligibility criteria: Main Inclusion Criteria:

1. Female or male subjects who are ≥ 18 years of age at screening;
2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
3. The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive);
4. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be ≥3;
5. The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
6. Body mass index (BMI) greater than 18 kg/m2 at screening;

Main Exclusion Criteria:

1. Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;
2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
3. Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;

Primary outcomes:
measure: incidence of adverse events (AEs)/serious adverse events (SAEs)
timeFrame: Each visit within 5 years after administration
measure: Dose limiting toxicities (DLT)
timeFrame: 28 days after administration
Secondary outcomes:
measure: Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score
timeFrame: 28 days, 56 days, 84 days, 6 months, 12months, 18 months after administration
Other outcomes:
measure: change of concentration of neurofilament light chain (NFL) in blood and CSF
timeFrame: 84 days, 6 months, 12months, 18 months after administration
Locations:
city: Beijing
country: China
facility: Peking University Third Hospital
Study officials:
affiliation: Peking University Third Hospital
name: Dongsheng Fan, PhD
role: PRINCIPAL_INVESTIGATOR
References: