Trial · NCT04340362
Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
NCT04340362, Phase 2, Completed, Vertex Pharmaceuticals Incorporated; results first posted 2023-07-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis. Trials, record NCT04340362. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04340362
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
- NCT number
- NCT04340362
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 16 participants, actual
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Glomerulosclerosis, Focal Segmental
- Interventions
- VX-147
- First posted
- 2020-04-09
- Last updated
- 2025-09-24
- Other registries' IDs
- eudract:2020-000185-42
- Results first posted
- 2023-07-10
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in UPCR | From Baseline up to Week 13 | |
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 17 | |
| Secondary | Maximum Observed Concentration (Cmax) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5 | |
| Secondary | Observed Pre-dose Concentration (Ctrough) of VX-147 | Pre-dose on Day 8, 15, Week 3, 5, 9 and 13 | |
| Secondary | Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5 |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in UPCR | From Baseline up to Week 13 | The Full Analysis Set (FAS) included all participants who received at least 1 dose of study drug in the treatment period. Here Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome and the Number Analyzed signifies participants who were evaluable for the specified Cohorts. | percent change | GEOMETRIC_MEAN |
| SECONDARY | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 17 | Safety set included all participants who received at least 1 dose of study drug in the treatment period. Here, the Number Analyzed signifies participants who were evaluable for the specified cohorts. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Maximum Observed Concentration (Cmax) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here Number Analyzed signifies participants who were evaluable at specified timepoint. | micrograms per milliliter (mcg/ml) | MEAN |
| SECONDARY | Observed Pre-dose Concentration (Ctrough) of VX-147 | Pre-dose on Day 8, 15, Week 3, 5, 9 and 13 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here Number Analyzed signifies participants who were evaluable at specified timepoint. | mcg/ml | MEAN |
| SECONDARY | Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 | Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5 | The PK set included all those who received at least 1 dose of study drug. Though participants in this study were divided into two cohorts based on their UPCR range, PK data was collected and presented based on dose levels as all participants received 15 mg qd for 2 weeks and 45 mg qd for 11 weeks. Therefore, presenting data based on cohorts as per UPCR range was not relevant for purpose of PK endpoints. Here Number Analyzed signifies participants who were evaluable at specified timepoint. | hour*mcg/mL (h*mcg/mL) | MEAN |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in UPCR | VX-147 | Cohort 1 | -47.7 | 95% Confidence Interval -70.1 to -8.5 | percent change |
| Percent Change From Baseline in UPCR | VX-147 | Cohort 2 | -47.5 | 95% Confidence Interval -63.4 to -24.6 | percent change |
| Percent Change From Baseline in UPCR | VX-147 | Total | -47.6 | 95% Confidence Interval -60.0 to -31.3 | percent change |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | VX-147 | Cohort 1: Participants With TEAEs | 3 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | VX-147 | Cohort 1: Participants With SAEs | 0 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | VX-147 | Cohort 2: Participants With TEAEs | 12 | Participants | |
| Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | VX-147 | Cohort 2: Participants With SAEs | 1 | Participants | |
| Maximum Observed Concentration (Cmax) of VX-147 | VX-147 | 15 mg qd: Day 1 | 0.168 | Standard Deviation 0.0630 | micrograms per milliliter (mcg/ml) |
| Maximum Observed Concentration (Cmax) of VX-147 | VX-147 | 45 mg qd: Week 5 | 0.846 | Standard Deviation 0.303 | micrograms per milliliter (mcg/ml) |
| Observed Pre-dose Concentration (Ctrough) of VX-147 | VX-147 | 15 mg qd: Day 8 | 0.153 | Standard Deviation 0.0984 | mcg/ml |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | VX-147 | 16 | |
| Overall Study | Cohort 1 | VX-147 | 3 | |
| Overall Study | Cohort 2 | VX-147 | 13 | |
| Overall Study | COMPLETED | VX-147 | 15 | |
| Overall Study | NOT COMPLETED | VX-147 | 1 |
Source ClinicalTrials.gov
Adverse events117
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 | Palpitations | Cardiac disorders | VX-147 : Cohort 1 | 0 | 3 | |
| other | Palpitations | Cardiac disorders | VX-147: Cohort 2 | 1 | 13 | |
| other | Palpitations | Cardiac disorders | VX-147 Total | 1 | 16 | |
| other | Abdominal distension | Gastrointestinal disorders | VX-147 : Cohort 1 | 0 | 3 | |
| other | Abdominal distension | Gastrointestinal disorders | VX-147: Cohort 2 | 1 | 13 | |
| other | Abdominal distension | Gastrointestinal disorders | VX-147 Total | 1 | 16 | |
| other | Abdominal pain | Gastrointestinal disorders | VX-147 : Cohort 1 | 1 | 3 | |
| other | Abdominal pain | Gastrointestinal disorders | VX-147: Cohort 2 | 0 | 13 | |
| other | Abdominal pain | Gastrointestinal disorders | VX-147 Total | 1 | 16 | |
| other | Abdominal pain lower | Gastrointestinal disorders | VX-147 : Cohort 1 | 0 | 3 |
1 to 10 of 117Source ClinicalTrials.gov
See all 117 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | VX-147 : Cohort 1 | 0 | 3 |
| Total, all-cause mortality | VX-147: Cohort 2 | 0 | 13 |
| Total, all-cause mortality | VX-147 Total | 0 | 16 |
| Total, other adverse events | VX-147 : Cohort 1 | 3 | 3 |
| Total, other adverse events | VX-147: Cohort 2 | 12 | 13 |
| Total, other adverse events | VX-147 Total | 15 | 16 |
| Total, serious adverse events | VX-147 : Cohort 1 | 0 | 3 |
| Total, serious adverse events | VX-147: Cohort 2 | 1 | 13 |
| Total, serious adverse events | VX-147 Total | 1 | 16 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
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/NCT04340362
GET https://conductscience.org/api/trials/v1/NCT04340362/events?page=2