Amyotrophic Lateral SclerosisPhase 1NCT06556394
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
A Phase 1 study testing RAG-17 in people with Amyotrophic Lateral Sclerosis, run by Ractigen Therapeutics. at 3 sites in China.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- RAG-17
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 3 sitesChina
- Study dates
- 24 Dec 2024to Apr 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
- ≥ 18 years of age at the time of informed consent.
- Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
- Documented SOD1 mutation.
- Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
- 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.
Who cannot join
- Documented p.F21C SOD1 mutation.
- 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.
- Current enrollment in any other interventional study.
- History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
- Pregnant or currently breastfeeding.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites, 3 recruiting
- Recruiting
Beijing Tiantan Hospital
Beijing
Yilong Wang, PhD&MD, yilong528@gmail.com
- Recruiting
West China Hospital of Sichuan University
Chengdu
Huifang Shang, PhD&MD, hfshang2002@163.com
- Recruiting
The Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou
Zhiying Wu, PhD&MD, zhiyingwu@zju.edu.cn
About this study
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
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
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- RAG-17, Placebo
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAG-17Drug
RAG-17 is a therapeutic small interfering RNA (siRNA).
PlaceboDrug
Placebo will be administered via intrathecal injection
| Study group | Role | What they receive |
|---|---|---|
| RAG-17 | Experimental | RAG-17 is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation Receives: Drug: RAG-17 |
| Placebo | Placebo comparator | Placebo is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events(AEs)
Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)
Measured: Before treatment and within 57 days after treatment
Secondary outcomes (11)
Plasma Pharmacokinetic (PK) Parameter: AUC0-last
Area Under the Plasma Concentration-Time Curve from Time 0 to the Last Measurable Non-zero Concentration
Measured: 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
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Cmax
Peak Plasma Concentration
Measured: 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
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: λz
Elimination Rate Constant
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: T½
Apparent Terminal Elimination Half-life of Study Drug
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: CL/F
Apparent Clearance
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Vz/F
Apparent Volume of Distribution
Measured: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: MRT
Mean Residence Time
Measured: Before treatment and within 48 hours after treatment
CSF Pharmacokinetic (PK) Parameter: Concentration
Concentration in Cerebrospinal Fluid(CSF)
Measured: Before treatment and within 29 days after treatment
CSF Pharmacokinetic (PK) Parameter: T½
Half-life in Cerebrospinal Fluid(CSF)
Measured: Before treatment and within 29 days after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2024
- Study start24 Dec 2024
- Primary completionFeb 2026 (estimated)
- Study completionApr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ractigen Therapeutics.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06556394
- Sponsor's protocol ID
- RGN17-001
- Status last verified
- Jan 2025
- First submitted
- 1 Aug 2024
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06556394
- Acronym / sponsor ID
- RGN17-001
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Ractigen Therapeutics.University, hospital or other
- Study start
- 24 Dec 2024 (actual)
- Primary completion
- Feb 2026 (estimated)
- Study completion
- Apr 2026 (estimated)
- First posted
- 16 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RAG-17 | Experimental | Drug: RAG-17RAG-17 is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo is administered by intrathecal injection to subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.
- ≥ 18 years of age at the time of informed consent.
- Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.
- Documented SOD1 mutation.
- Forced vital capacity (FVC) ≥50% of predicted value as adjusted for sex, age, and height (measured seated).
Key exclusion criteria (5 listed)
- Documented p.F21C SOD1 mutation.
- 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.
- Current enrollment in any other interventional study.
- History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.
- Pregnant or currently breastfeeding.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RAG-17RAG-17 is a therapeutic small interfering RNA (siRNA). | Drug | RAG-17 |
| PlaceboPlacebo will be administered via intrathecal injection | Drug | Placebo |
Primary outcome
Adverse Events(AEs)
Time frame: Before treatment and within 57 days after treatment
Assesment of Safety and Tolerability: Incidence and severity of treatment-emergent Adverse Events(AEs) and Serious Adverse Events(SAEs)
11 secondary outcomes
Plasma Pharmacokinetic (PK) Parameter: AUC0-last
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: AUC0-inf
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Cmax
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Tmax
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: λz
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: T½
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: CL/F
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: Vz/F
Time frame: Before treatment and within 48 hours after treatment
Plasma Pharmacokinetic (PK) Parameter: MRT
Time frame: Before treatment and within 48 hours after treatment
CSF Pharmacokinetic (PK) Parameter: Concentration
Time frame: Before treatment and within 29 days after treatment
CSF Pharmacokinetic (PK) Parameter: T½
Time frame: Before treatment and within 29 days after treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RAG-17 in people with Amyotrophic Lateral Sclerosis, run by Ractigen Therapeutics. at 3 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Long-Cheng Li (+86 18051622388, lilc@ractigen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events(AEs) (time frame: Before treatment and within 57 days after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.