Trial · NCT04659863
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
NCT04659863, Phase 3, Completed, Novartis Pharmaceuticals; results first posted 2025-05-31
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia. Trials, record NCT04659863. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04659863
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
- NCT number
- NCT04659863
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 13 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Familial Hypercholesterolemia - Homozygous
- Interventions
- Inclisiran, Placebo
- First posted
- 2020-12-09
- Last updated
- 2026-01-13
- Other registries' IDs
- eudract:2020-002755-38
- Results first posted
- 2025-05-31
Source ClinicalTrials.gov
Registered outcomes22
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) | Baseline and Day 330 | Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1) |
| Secondary | Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1) | Baseline, after Day 90 up to Day 330 | Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330 |
| Secondary | Percent Change in LDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Percentage change in LDL-C from baseline to each assessment time up to Day 720. |
| Secondary | Absolute Change in LDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Absolute change in LDL-C from baseline to each assessment time up to Day 720. |
| Secondary | Percent Change in Apo B From Baseline up to Day 720 | Baseline, up to Day 720 | Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720. |
| Secondary | Absolute Change in Apo B From Baseline up to Day 720 | Baseline, up to Day 720 | Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720. |
| Secondary | Percent Change in Lp(a) From Baseline up to Day 720 | Baseline, up to Day 720 | Percentage change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720. |
| Secondary | Absolute Change in Lp(a) From Baseline up to Day 720 | Baseline, up to Day 720 | Absolute change in lipoprotein (a) \[Lp(a)\] from baseline to each assessment time up to Day 720. |
| Secondary | Percent Change in Non-HDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720. |
| Secondary | Absolute Change in Non-HDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720. |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 registered outcomesOutcomes22
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) | Baseline and Day 330 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization. | percent change in LDL-C | MEAN |
| SECONDARY | Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1) | Baseline, after Day 90 up to Day 330 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | Time-adjusted percent change in LDL-C | MEAN |
| SECONDARY | Percent Change in LDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | Percent change in LDL-C | MEAN |
| SECONDARY | Absolute Change in LDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | mg/dL | MEAN |
| SECONDARY | Percent Change in Apo B From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | Percent change in Apo B | MEAN |
| SECONDARY | Absolute Change in Apo B From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | mg/dL | MEAN |
| SECONDARY | Percent Change in Lp(a) From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | Percent change in Lp(a) | MEAN |
| SECONDARY | Absolute Change in Lp(a) From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | nmol/L | MEAN |
| SECONDARY | Percent Change in Non-HDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | Percent change in non-HDL-C | MEAN |
| SECONDARY | Absolute Change in Non-HDL-C From Baseline up to Day 720 | Baseline, up to Day 720 | Full Analysis Set (FAS) comprised all participants to whom study treatment had been assigned by randomization | mg/dL | MEAN |
1 to 10 of 22Source ClinicalTrials.gov
See all 22 outcomesResults by group180
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) | Part 1- Inclisiran | -21.6 | Standard Deviation 13.36 | percent change in LDL-C | |
| Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) | Part 1 - Placebo | 11.7 | Standard Deviation 30.52 | percent change in LDL-C | |
| Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1) | Part 1- Inclisiran | -21.0 | Standard Deviation 15.11 | Time-adjusted percent change in LDL-C | |
| Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1) | Part 1 - Placebo | 13.0 | Standard Deviation 41.88 | Time-adjusted percent change in LDL-C | |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1- Inclisiran | Day 90 | -26.1 | Standard Deviation 13.53 | Percent change in LDL-C |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1 - Placebo | Day 90 | 6.8 | Standard Deviation 16.83 | Percent change in LDL-C |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1- Inclisiran | Day 150 | -24.1 | Standard Deviation 14.65 | Percent change in LDL-C |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1 - Placebo | Day 150 | 17.0 | Standard Deviation 41.22 | Percent change in LDL-C |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1- Inclisiran | Day 270 | -17.3 | Standard Deviation 21.64 | Percent change in LDL-C |
| Percent Change in LDL-C From Baseline up to Day 720 | Part 1 - Placebo | Day 270 | 10.2 | Standard Deviation 54.00 | Percent change in LDL-C |
1 to 10 of 180Source ClinicalTrials.gov
See all 180 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 (Double-blind Period) | STARTED | Part 1- Inclisiran | 9 | |
| Part 1 (Double-blind Period) | STARTED | Part 1 - Placebo | 4 | |
| Part 1 (Double-blind Period) | STARTED | Part 2 - Inclisiran (Total) | 0 | |
| Part 1 (Double-blind Period) | COMPLETED | Part 1- Inclisiran | 9 | |
| Part 1 (Double-blind Period) | COMPLETED | Part 1 - Placebo | 4 | |
| Part 1 (Double-blind Period) | COMPLETED | Part 2 - Inclisiran (Total) | 0 | |
| Part 1 (Double-blind Period) | NOT COMPLETED | Part 1- Inclisiran | 0 | |
| Part 1 (Double-blind Period) | NOT COMPLETED | Part 1 - Placebo | 0 | |
| Part 1 (Double-blind Period) | NOT COMPLETED | Part 2 - Inclisiran (Total) | 0 | |
| Part 2 (Open-label Period) | STARTED | Part 1- Inclisiran | 0 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events84
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 | Abdominal pain upper | Gastrointestinal disorders | Part 1 - Inclisiran | 2 | 9 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Part 1 - Placebo | 0 | 4 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Part 2 - Inclisiran (Total) | 1 | 13 | |
| other | Toothache | Gastrointestinal disorders | Part 1 - Inclisiran | 1 | 9 | |
| other | Toothache | Gastrointestinal disorders | Part 1 - Placebo | 0 | 4 | |
| other | Toothache | Gastrointestinal disorders | Part 2 - Inclisiran (Total) | 0 | 13 | |
| other | Vomiting | Gastrointestinal disorders | Part 1 - Inclisiran | 1 | 9 | |
| other | Vomiting | Gastrointestinal disorders | Part 1 - Placebo | 0 | 4 | |
| other | Vomiting | Gastrointestinal disorders | Part 2 - Inclisiran (Total) | 0 | 13 | |
| other | Influenza like illness | General disorders | Part 1 - Inclisiran | 1 | 9 |
1 to 10 of 84Source ClinicalTrials.gov
See all 84 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Part 1 - Inclisiran | 0 | 9 |
| Total, all-cause mortality | Part 1 - Placebo | 0 | 4 |
| Total, all-cause mortality | Part 2 - Inclisiran (Total) | 0 | 13 |
| Total, other adverse events | Part 1 - Inclisiran | 7 | 9 |
| Total, other adverse events | Part 1 - Placebo | 1 | 4 |
| Total, other adverse events | Part 2 - Inclisiran (Total) | 8 | 13 |
| Total, serious adverse events | Part 1 - Inclisiran | 0 | 9 |
| Total, serious adverse events | Part 1 - Placebo | 0 | 4 |
| Total, serious adverse events | Part 2 - Inclisiran (Total) | 0 | 13 |
Source ClinicalTrials.gov
Papers3
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/NCT04659863
GET https://conductscience.org/api/trials/v1/NCT04659863/registered?page=2