RAG-17 in Amyotrophic Lateral Sclerosis: NCT05903690 Clinical Landscape Report 2026

15 October 2026
9 min read

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This Clinical Landscape Report was built with PatSnap Life Sciences MCP workflows. Clinical Trials MCP supplies protocol and result records, Drug & Asset MCP adds asset context, and Company & Deal Intelligence MCP resolves sponsor background. Use the same structured MCP building blocks in your research workflow.

Data snapshot: 15 October 2026. This strategic research report is not medical, regulatory or investment advice. Trial status and dates can change.

Early Phase 1

Clinical phase

Completed

Recruitment status

not reported

Planned enrollment

2024-07-11

Primary-completion proxy

Executive view

NCT05903690 evaluates RAG-17 in Amyotrophic Lateral Sclerosis. The disclosed sponsor is Beijing Tiantan Hospital, the design is Interventional, and the geographic footprint is China. The first listed primary endpoint is Adverse events (AE) and serious adverse events (SAE), assessed over Within 240 days after treatment.

The key landscape question is whether this protocol can generate a clinically interpretable signal relative to nearby programs. Phase alone is not a measure of evidence quality. Endpoint relevance, comparator choice, masking, enrollment feasibility, patient selection, country coverage and follow-up must be considered together.

PatSnap MCP Servers make this assessment reproducible by keeping protocol facts, result evidence, asset attributes and sponsor identity in separate structured calls.

How the MCP evidence stack was assembled

Clinical Trials MCP clinical_trial_fetch retrieved the design, outcomes, phase, status, enrollment, sponsor, countries and timing for NCT05903690 and selected peers. clinical_trial_result_fetch supplied detailed result records from the wider Amyotrophic Lateral Sclerosis landscape. Drug & Asset MCP drug_fetch was queried for RAG-17, while Company & Deal Intelligence MCP organization_fetch was queried for Beijing Tiantan Hospital.

This separation reduces a common diligence error: treating a registry label, a company description or a result excerpt as if each represented the complete evidence package. Explore the source workflow at the PatSnap MCP marketplace.

Trial landscape table

TrialAsset / interventionPhase / statusSponsorGeographyPrimary endpointReadout proxy
NCT05903690RAG-17Early Phase 1 / CompletedBeijing Tiantan HospitalChinaAdverse events (AE) and serious adverse events (SAE)
Within 240 days after treatment
2024-07-11
NCT06665165AMX-0114Phase 1 / RecruitingAmylyx Pharmaceuticals, Inc.Canada, United StatesEvaluate the safety and tolerability of AMX0114 in adult participants living with ALS
Day 1 - Day 145 (End of Study)
2027-02-01
NCT06658977Abacavir sulfate/Dolutegravir sodium/LamivudinePhase 3 / TerminatedMacquarie UniversityAustraliaIncidence of Treatment-Emergent Adverse Events [Safety and Tolerability]).
Duration of the study plus 7 days
2025-06-03
NCT06656702PsilocybinEarly Phase 1 / RecruitingThe Johns Hopkins UniversityUnited StatesFeasibility of psilocybin treatment as assessed by number of participants recruited, retained and adhere to treatment
6 months
2027-01-30
NCT06643481VHB937Phase 2 / Status not reportedNovartis PharmaceuticalsUnited States, United Kingdom, Switzerland, Spain, Canada, Sweden, South Korea, Netherlands, Belgium, Ireland, China, Poland, Denmark, Italy, France, Australia, GermanyThe composite of PAV-free survival and change in ALSFRS-R. Analysis method: Combined Assessment of Function and Surviva…
Baseline to DB Week 40
2026-06-23

The table aligns endpoints, sponsors, phases, geographies and readout proxies. It is descriptive, not a head-to-head efficacy comparison. Differences in population, baseline risk, intervention schedule and follow-up can dominate apparent cross-trial differences.

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Protocol design and endpoint interpretation

NCT05903690 is a Early Phase 1, completed study with not reported planned participants. Allocation is N/A, masking is None (Open Label), and the intervention model is Single Group Assignment.

The primary endpoint is “Adverse events (AE) and serious adverse events (SAE)” over “Within 240 days after treatment.” The retrieved endpoint description is: The incidence of adverse events (AE) and serious adverse events (SAE) within 240 days after the first injection of RAG-17, and number of participants with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) V5.0.

Interpretation should test whether the endpoint captures a clinically meaningful change, whether its time horizon matches the proposed biology, and whether treatment discontinuation or missing data can bias the estimate. The planned enrollment of not reported should be assessed against expected effect size, event frequency, multiplicity, subgroup plans and attrition.

No named control drug was returned in the protocol field set. A placebo comparator can strengthen internal efficacy assessment, while an active comparator may better test clinical differentiation. Single-arm and open-label programs require greater weight on objective outcomes, independent assessment and external benchmarks.

Indexed readouts in the surrounding landscape

0 recent result records were selected as contextual evidence for Amyotrophic Lateral Sclerosis. These records do not establish direct evidence for NCT05903690 unless the registration number matches.

Indexed evidence status

No directly comparable result record was retrieved for this disease query. This is a monitoring gap, not evidence of absence.

Result fields should be reconciled with the source record before quantitative comparison. Population definitions, analysis sets, dose cohorts, estimands, confidence intervals, rescue therapy and follow-up can materially change the meaning of a numerical endpoint. Clinical Trials MCP supports repeatable refreshes as result records change.

Asset and sponsor context

RAG-17 is indexed as siRNA with SOD1 biology and a global stage of Phase 2. The asset profile lists Ractigen Therapeutics as an originator or developer.

Beijing Tiantan Hospital is indexed in an unreported country with the website http://www.bjtth.org. Operates hospitals The record lists 17 development-stage drug assets.

For execution diligence, monitor sponsor ownership, collaborator additions, site expansion, protocol amendments and enrollment revisions. A change in ownership or geography can alter operational confidence as well as the commercial meaning of a future readout.

Development white space

  1. Endpoint white space. Determine whether a more patient-relevant outcome, longer durability window or blinded central assessment would resolve uncertainty left by the current endpoint.
  2. Population white space. Test biomarker-defined, treatment-line or risk-stratified subgroups where effect size and unmet need could be clearer.
  3. Comparator white space. Identify whether the study can support differentiation against the current standard of care rather than only activity against baseline or placebo.
  4. Geographic white space. Assess whether the disclosed footprint supports recruitment, regulatory transferability and commercial generalizability.
  5. Sequencing white space. Clarify whether RAG-17 is intended for monotherapy, combination, maintenance, rescue or an earlier treatment line.

White space should be framed as an unanswered development question, not merely an unoccupied mechanism label. A credible program closes a measurable clinical uncertainty with a design that can be executed and interpreted.

Strategic implications and next readouts

For sponsors, the endpoint hierarchy, safety window, enrollment pace and protocol amendment history should all support the same target product profile. Advancement criteria should be set before the readout and tied to clinical effect, uncertainty, tolerability and operational feasibility.

For business-development teams, differentiation may come from a sharper population, stronger comparator, more durable benefit, simpler delivery or clearer sequencing role. For investors, the central risk is evidence quality relative to time and capital, not the phase label in isolation.

Track recruitment status, enrollment changes, primary-completion timing, endpoint revisions, new result records, sponsor ownership and collaborator changes. Re-run the PatSnap MCP workflow when a surrogate becomes a clinical outcome, a single-country study expands, the comparator changes or a new result materially shifts the competitive benchmark.

Source trail and bottom line

Anchor trial: NCT05903690
Protocol source: https://clinicaltrials.gov/study/NCT05903690
MCP sources: Clinical Trials MCP (clinical_trial_fetch and clinical_trial_result_fetch); Drug & Asset MCP (drug_fetch); Company & Deal Intelligence MCP (organization_fetch).
Data snapshot: 15 October 2026.

RAG-17 in Amyotrophic Lateral Sclerosis is best understood through the interaction of protocol design, surrounding readouts, asset context and sponsor execution. The current record makes Adverse events (AE) and serious adverse events (SAE) and 2024-07-11 the leading decision points.

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