Trial · NCT04059406
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
NCT04059406, Phase 2, Terminated, Ionis Pharmaceuticals, Inc.; results first posted 2025-02-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx). Trials, record NCT04059406. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04059406
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
- NCT number
- NCT04059406
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 29 participants, actual
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Conditions
- Beta Thalassemia Intermedia
- Interventions
- sapablursen
- First posted
- 2019-08-16
- Last updated
- 2025-02-18
- Other registries' IDs
- eudract:2019-003505-96
- Results first posted
- 2025-02-18
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27 | Baseline and Week 27 | Blood hemoglobin |
| Secondary | Percentage of Participants With a ≥1.5 g/dL Increase From Baseline in Hb at Week 53 | Week 53 | Blood hemoglobin |
| Secondary | Percentage of Participants With a ≥1.0 Milligrams of Iron Per Grams of Dry Weight of Liver (mg Fe/g) Decrease From Baseline in Liver Iron Concentration (LIC) at Week 53 | Week 53 | Liver iron content |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27 | Baseline and Week 27 | Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of sapablursen and who had at least 1 Hb assessment collected after Day 1. Here, the overall number of participants analyzed signifies the number of participants available for analysis for this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With a ≥1.5 g/dL Increase From Baseline in Hb at Week 53 | Week 53 | FAS included all randomized participants who received at least 1 dose of sapablursen and who had at least 1 Hb assessment collected after Day 1. Here, the overall number of participants analyzed signifies the number of participants available for analysis for this outcome measure. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With a ≥1.0 Milligrams of Iron Per Grams of Dry Weight of Liver (mg Fe/g) Decrease From Baseline in Liver Iron Concentration (LIC) at Week 53 | Week 53 | FAS included all randomized participants who received at least 1 dose of sapablursen and who had at least 1 Hb assessment collected after Day 1. Here, the overall number of participants analyzed signifies the number of participants available for analysis for this outcome measure. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27 | Cohort A: Sapablursen | 0 | 95% Confidence Interval 0.0 to 45.9 | percentage of participants | |
| Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27 | Cohort B: Sapablursen | 0 | 95% Confidence Interval 0.0 to 45.9 | percentage of participants | |
| Percentage of Participants With a ≥1.0 Grams Per Deciliter (g/dL) Increase From Baseline in Hemoglobin (Hb) at Week 27 | Cohort C: Sapablursen | 6.7 | 95% Confidence Interval 0.2 to 31.9 | percentage of participants | |
| Percentage of Participants With a ≥1.5 g/dL Increase From Baseline in Hb at Week 53 | Cohort A: Sapablursen | 0 | 95% Confidence Interval 0.0 to 45.9 | percentage of participants | |
| Percentage of Participants With a ≥1.5 g/dL Increase From Baseline in Hb at Week 53 | Cohort B: Sapablursen | 0 | 95% Confidence Interval 0.0 to 45.9 | percentage of participants | |
| Percentage of Participants With a ≥1.5 g/dL Increase From Baseline in Hb at Week 53 | Cohort C: Sapablursen | 0 | 95% Confidence Interval 0.0 to 23.2 | percentage of participants | |
| Percentage of Participants With a ≥1.0 Milligrams of Iron Per Grams of Dry Weight of Liver (mg Fe/g) Decrease From Baseline in Liver Iron Concentration (LIC) at Week 53 | Cohort A: Sapablursen | 33.3 | 95% Confidence Interval 4.3 to 77.7 | percentage of participants | |
| Percentage of Participants With a ≥1.0 Milligrams of Iron Per Grams of Dry Weight of Liver (mg Fe/g) Decrease From Baseline in Liver Iron Concentration (LIC) at Week 53 | Cohort B: Sapablursen | 50.0 | 95% Confidence Interval 11.8 to 88.2 | percentage of participants | |
| Percentage of Participants With a ≥1.0 Milligrams of Iron Per Grams of Dry Weight of Liver (mg Fe/g) Decrease From Baseline in Liver Iron Concentration (LIC) at Week 53 | Cohort C: Sapablursen | 28.6 | 95% Confidence Interval 8.4 to 58.1 | percentage of participants |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort A: Sapablursen | 6 | |
| Overall Study | STARTED | Cohort B: Sapablursen | 6 | |
| Overall Study | STARTED | Cohort C: Sapablursen | 17 | |
| Overall Study | COMPLETED | Cohort A: Sapablursen | 1 | |
| Overall Study | COMPLETED | Cohort B: Sapablursen | 1 | |
| Overall Study | COMPLETED | Cohort C: Sapablursen | 2 | |
| Overall Study | NOT COMPLETED | Cohort A: Sapablursen | 5 | |
| Overall Study | NOT COMPLETED | Cohort B: Sapablursen | 5 | |
| Overall Study | NOT COMPLETED | Cohort C: Sapablursen | 15 |
Source ClinicalTrials.gov
Adverse events276
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Asthenia | General disorders | Cohort A: Sapablursen | 0 | 6 | |
| other | Asthenia | General disorders | Cohort B: Sapablursen | 1 | 6 | |
| other | Asthenia | General disorders | Cohort C: Sapablursen | 0 | 17 | |
| other | Injection Site Erythema | General disorders | Cohort A: Sapablursen | 0 | 6 | |
| other | Injection Site Erythema | General disorders | Cohort B: Sapablursen | 1 | 6 | |
| other | Injection Site Erythema | General disorders | Cohort C: Sapablursen | 0 | 17 | |
| other | Injection Site Pain | General disorders | Cohort A: Sapablursen | 1 | 6 | |
| other | Injection Site Pain | General disorders | Cohort B: Sapablursen | 0 | 6 | |
| other | Injection Site Pain | General disorders | Cohort C: Sapablursen | 0 | 17 | |
| other | Injection Site Pruritus | General disorders | Cohort A: Sapablursen | 0 | 6 |
1 to 10 of 276Source ClinicalTrials.gov
See all 276 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort A: Sapablursen | 0 | 6 |
| Total, all-cause mortality | Cohort B: Sapablursen | 0 | 6 |
| Total, all-cause mortality | Cohort C: Sapablursen | 0 | 17 |
| Total, other adverse events | Cohort A: Sapablursen | 6 | 6 |
| Total, other adverse events | Cohort B: Sapablursen | 6 | 6 |
| Total, other adverse events | Cohort C: Sapablursen | 14 | 17 |
| Total, serious adverse events | Cohort A: Sapablursen | 0 | 6 |
| Total, serious adverse events | Cohort B: Sapablursen | 2 | 6 |
| Total, serious adverse events | Cohort C: Sapablursen | 1 | 17 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 42241700 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04059406
GET https://conductscience.org/api/trials/v1/NCT04059406/events?page=2