Clinical trial NCT06556394: A 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
Official title: A 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Ractigen Therapeutics.
Sponsor class: OTHER
First posted: 2024-08-16
Enrollment: 32
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
2. ≥ 18 years of age at the time of informed consent.
3. Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
4. Documented SOD1 mutation.
5. Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
6. If taking riluzole or edaravone, subject must be on a stable dose or ≥30 days prior to Day 1 and expected to remain at that dose until the final study visit.

Exclusion Criteria:

1. Documented p.F21C SOD1 mutation.
2. Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.
3. Current enrollment in any other interventional study.
4. History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
5. Pregnant or currently breastfeeding.

Primary outcomes:
measure: Adverse Events(AEs)
timeFrame: Before treatment and within 57 days after treatment
Secondary outcomes:
measure: Plasma Pharmacokinetic (PK) Parameter: AUC0-last
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: AUC0-inf
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: Cmax
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: Tmax
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: λz
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: T½
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: CL/F
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: Vz/F
timeFrame: Before treatment and within 48 hours after treatment
measure: Plasma Pharmacokinetic (PK) Parameter: MRT
timeFrame: Before treatment and within 48 hours after treatment
measure: CSF Pharmacokinetic (PK) Parameter: Concentration
timeFrame: Before treatment and within 29 days after treatment
measure: CSF Pharmacokinetic (PK) Parameter: T½
timeFrame: Before treatment and within 29 days after treatment
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Tiantan Hospital
city: Chengdu
country: China
facility: West China Hospital of Sichuan University
city: Hangzhou
country: China
facility: The Second Affiliated Hospital Zhejiang University School of Medicine
Study officials:
References: