This is a Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation
The study is a phase 1, randomized, double-blind, placebo controlled study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in patients with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) gene mutation. The dose levels will be evaluated sequentially across separate cohorts using a rules-based design, wherein participants will receive RAG-17 or placebo at a ratio of 3:1.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
32
Beijing Tiantan Hospital
Beijing, China
RECRUITINGWest China Hospital of Sichuan University
Chengdu, China
RECRUITINGThe Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, China
RECRUITINGAdverse Events(AEs)
Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)
Time frame: Before treatment and within 57 days after treatment
Plasma Pharmacokinetic (PK) Parameter: AUC0-last
Area Under the Plasma Concentration-Time Curve from Time 0 to the Last Measurable Non-zero Concentration
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: AUC0-inf
Area Under the Plasma Concentration-Time Curve from Time 0 Extrapolated to Infinity
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Cmax
Peak Plasma Concentration
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Tmax
Time to reach Cmax. If the maximum value occurs at more than one time point, Tmax is defined as the first time point with this value
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: λz
Elimination Rate Constant
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: T½
Apparent Terminal Elimination Half-life of Study Drug
Time frame: Before treatment and within 48 hours after treatment
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Plasma Pharmacokinetic (PK) Parameter: CL/F
Apparent Clearance
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Vz/F
Apparent Volume of Distribution
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: MRT
Mean Residence Time
Time frame: Before treatment and within 48 hours after treatment
CSF Pharmacokinetic (PK) Parameter: Concentration
Concentration in Cerebrospinal Fluid(CSF)
Time frame: Before treatment and within 29 days after treatment
CSF Pharmacokinetic (PK) Parameter: T½
Half-life in Cerebrospinal Fluid(CSF)
Time frame: Before treatment and within 29 days after treatment