Ledipasvir/Sofosbuvir in Beta-Thalassemia: NCT04353986 Clinical Landscape Report 2026
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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: 28 September 2026. This strategic research report is not medical, regulatory or investment advice. Trial status and dates can change.
Phase 3
Clinical phase
Unknown status
Recruitment status
24
Planned enrollment
2023-04-01
Primary-completion proxy
Executive view
NCT04353986 evaluates Ledipasvir/Sofosbuvir in Beta-Thalassemia. The disclosed sponsor is Ain Shams University, the design is Interventional, and the geographic footprint is Egypt. The first listed primary endpoint is Predictive Pharmacokinetic Model, assessed over 10 days.
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 NCT04353986 and selected peers. clinical_trial_result_fetch supplied detailed result records from the wider Beta-Thalassemia landscape. Drug & Asset MCP drug_fetch was queried for Ledipasvir/Sofosbuvir, while Company & Deal Intelligence MCP organization_fetch was queried for Ain Shams University.
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
| Trial | Asset / intervention | Phase / status | Sponsor | Geography | Primary endpoint | Readout proxy |
|---|---|---|---|---|---|---|
| NCT04353986 | Ledipasvir/Sofosbuvir | Phase 3 / Unknown status | Ain Shams University | Egypt | Predictive Pharmacokinetic Model 10 days | 2023-04-01 |
| NCT04474626 | Isoquercetin | Phase 2 / Withdrawn | Beth Israel Deaconess Medical Center, Inc. | Geography not reported | Change in sP Selectin levels with isoquercetin baseline to 28 Days | 2022-12-31 |
| NCT04432623 | Benserazide | Phase 1/2 / Enrolling by invitation | PHOENICIA BIOSCIENCES, INC. | Canada, United States | Safety and Tolerability 12 to 24 weeks | 2026-02-28 |
| NCT04411082 | Tovinontrine | Phase 2 / Terminated | Cardurion Pharmaceuticals, Inc. | Lebanon, Greece, Netherlands, Turkey, Morocco, Denmark, United Kingdom, Italy, Malaysia, Israel, United States, France, Tunisia | IMR-687 Safety and Tolerability Baseline to Week 40 | 2022-03-11 |
| NCT04364269 | Vamifeport hydrochloride | Phase 2 / Completed | Vifor (International) AG | Lebanon, Greece, Italy, Israel, Thailand | Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From baseline to Week 16 | 2021-10-11 |
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
NCT04353986 is a Phase 3, unknown status study with 24 planned participants. Allocation is Non-Randomized, masking is None (Open Label), and the intervention model is Parallel Assignment.
The primary endpoint is “Predictive Pharmacokinetic Model” over “10 days.” The retrieved endpoint description is: serial blood samples will be withdrawn to measure the drug level develop a Predictive Pharmacokinetic Model for sofosbuvir, ledipasvir and GS 331007.
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 24 should be assessed against expected effect size, event frequency, multiplicity, subgroup plans and attrition.
The protocol identifies Ledipasvir/Sofosbuvir as control therapy. 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
5 recent result records were selected as contextual evidence for Beta-Thalassemia. These records do not establish direct evidence for NCT04353986 unless the registration number matches.
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
Phase 2; n=56; Number of Participants With Engraftment = 53 Participants Source: https://clinicaltrials.gov/ct2/show/results/NCT01050855
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia
Phase 1/2; n=9; Adverse Event: decreased lymphocyte counts = One patient had decreased lymphocyte counts attributed to reni-cel Source: https://pubmed.ncbi.nlm.nih.gov/41931048/
REDEFINING CURATIVE HORIZONS IN THALASSEMIA MAJOR: 100% SURVIVAL FOLLOWING ALTERNATIVE DONOR HSCT IN A RESOURCE- LIMITED SETTING
Not Applicable; n=33; EFS = 50.0 % ; EFS = 100.0 % Source: https://www.ebmt.org/sites/default/files/2026-03/AM26_Abstracts%20Book_final_v.pdf
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
Ledipasvir/Sofosbuvir is indexed as Small molecule drug with NS5A x NS5B polymerase biology and a global stage of Approved. The asset profile lists Gilead Sciences, Inc. as an originator or developer.
Ain Shams University is indexed in Egypt with the website http://www.asu.edu.eg. Ain Shams University provides study in the field of astronomy, engineering and medicine. The record lists 18 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
- 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.
- Population white space. Test biomarker-defined, treatment-line or risk-stratified subgroups where effect size and unmet need could be clearer.
- Comparator white space. Identify whether the study can support differentiation against the current standard of care rather than only activity against baseline or placebo.
- Geographic white space. Assess whether the disclosed footprint supports recruitment, regulatory transferability and commercial generalizability.
- Sequencing white space. Clarify whether Ledipasvir/Sofosbuvir 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: NCT04353986
Protocol source: https://clinicaltrials.gov/study/NCT04353986
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: 28 September 2026.
Ledipasvir/Sofosbuvir in Beta-Thalassemia is best understood through the interaction of protocol design, surrounding readouts, asset context and sponsor execution. The current record makes Predictive Pharmacokinetic Model and 2023-04-01 the leading decision points.
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