Trial · NCT04681170
Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
NCT04681170, Phase 3, Completed, Amryt Pharma; results first posted 2025-08-27
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH). Trials, record NCT04681170. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04681170
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
- NCT number
- NCT04681170
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 46 participants, actual
- Lead sponsor
- Amryt Pharma
- Conditions
- Homozygous Familial Hypercholesterolaemia (HoFH)
- Interventions
- Lomitapide
- First posted
- 2020-12-23
- Last updated
- 2025-08-27
- Results first posted
- 2025-08-27
- Limitations and caveats
- Lp(a) analysis was analysed locally as central analysis was only available from January 2022.
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change in low density lipoprotein cholesterol (LDL C) at the maximum tolerated dose (MTD) |
| Secondary | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change of the following lipid parameters at the maximum tolerated dose (MTD): * Non-high-density lipoprotein cholesterol (Non-HDL-C) * Total cholesterol (TC) * Very-low-density lipoprotein cholesterol (VLDL-C) * Apolipoprotein B (Apo B) * TG (Triglycerides) |
| Secondary | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | Baseline through Week 24 | To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in LDL-C at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in Non-HDL-C at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in TC at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in VLDL-C at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in apo B at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in TG at the maximum tolerated dose (MTD) |
| Secondary | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Baseline through Week 104 | To evaluate the efficacy of lomitapide, as defined by the percent change in Lp(a) at the maximum tolerated dose (MTD) |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | Baseline through Week 24 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Baseline through Week 24 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Efficacy Endpoint: Percent Change From Baseline at Week 24 for Lp(a) | Baseline through Week 24 | Patients have been evaluated both, as individual age groups and all together. Note: Lp(a) assessment was inadvertently removed from the central laboratory lipid panel during the laboratory set up phase. However, Lp(a) has also been assessed locally, but in various units (mg/dL, g/L, and nmol/L). Sicne Lp(a) concentrations should not be converted from 'nmol/L' to 'mg/dL', or vice versa, and more subjects had values reported in nmol/L, results are presented for the nmol/l only. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for LDL-C | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for Non-HDL-C | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for TC | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for VLDL-C | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for Apo B | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for TG | Baseline through Week 104 | Patients from all age groups have been evaluated together. | Percent change from Baseline | MEAN |
| SECONDARY | Percent Change From Baseline at All Other Time Points Through Week 104 for Lipoprotein(a) (Lp(a)) | Baseline through Week 104 | Patients have been evaluated both, as individual age groups and all together. Note: Lp(a) assessment was inadvertently removed from the central laboratory lipid panel during the laboratory set up phase. However, Lp(a) has also been assessed locally, but in various units (mg/dL, g/L, and nmol/L). Sicne Lp(a) concentrations should not be converted from 'nmol/L' to 'mg/dL', or vice versa, and more subjects had values reported in nmol/L, results are presented for the nmol/l only. | Percent change from Baseline | MEAN |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group629
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | All Age Groups | -53.910 | Standard Deviation 25.8287 | Percent change from Baseline | |
| Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | Age 5-10 Years | -56.529 | Standard Deviation 26.5401 | Percent change from Baseline | |
| Efficacy Endpoint: Percent Change in Low-density Lipoprotein Cholesterol (LDL C) at Week 24 Compared to Baseline | Age 11-17 Years | -51.633 | Standard Deviation 25.5658 | Percent change from Baseline | |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | All Age Groups | Non-HDL-C | -54.185 | Standard Deviation 25.0256 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Age 5-10 Years | Non-HDL-C | -56.655 | Standard Deviation 25.9804 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Age 11-17 Years | Non-HDL-C | -52.038 | Standard Deviation 24.5423 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | All Age Groups | TC | -50.176 | Standard Deviation 24.5397 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Age 5-10 Years | TC | -52.883 | Standard Deviation 25.6260 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | Age 11-17 Years | TC | -47.822 | Standard Deviation 23.8756 | Percent change from Baseline |
| Efficacy Endpoint: Percent Change From Baseline at Week 24 for Various Lipid Parameters | All Age Groups | VLDL-C | -49.678 | Standard Deviation 31.6126 | Percent change from Baseline |
1 to 10 of 629Source ClinicalTrials.gov
See all 629 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Pre-run in | STARTED | Age 5-10 Years | 21 | |
| Pre-run in | STARTED | Age 11-17 Years | 25 | |
| Pre-run in | COMPLETED | Age 5-10 Years | 21 | |
| Pre-run in | COMPLETED | Age 11-17 Years | 25 | |
| Pre-run in | NOT COMPLETED | Age 5-10 Years | 0 | |
| Pre-run in | NOT COMPLETED | Age 11-17 Years | 0 | |
| Stratified Enrolment & run-in | STARTED | Age 5-10 Years | 21 | |
| Stratified Enrolment & run-in | STARTED | Age 11-17 Years | 25 | |
| Stratified Enrolment & run-in | COMPLETED | Age 5-10 Years | 20 | |
| Stratified Enrolment & run-in | COMPLETED | Age 11-17 Years | 23 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events178
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 | Alanine aminotransferase increased | Investigations | Age 5-10 Years | 9 | 20 | 11 |
| other | Alanine aminotransferase increased | Investigations | Age 11-17 Years | 8 | 23 | 17 |
| other | Aspartate aminotransferase increased | Investigations | Age 5-10 Years | 7 | 20 | 10 |
| other | Aspartate aminotransferase increased | Investigations | Age 11-17 Years | 8 | 23 | 18 |
| other | Blood creatine phosphokinase increased | Investigations | Age 5-10 Years | 4 | 20 | 4 |
| other | Blood creatine phosphokinase increased | Investigations | Age 11-17 Years | 2 | 23 | 2 |
| other | C-reactive protein increased | Investigations | Age 5-10 Years | 3 | 20 | 5 |
| other | C-reactive protein increased | Investigations | Age 11-17 Years | 3 | 23 | 3 |
| other | ECG signs of ventricular hypertrophy | Investigations | Age 5-10 Years | 2 | 20 | 2 |
| other | ECG signs of ventricular hypertrophy | Investigations | Age 11-17 Years | 3 | 23 | 3 |
1 to 10 of 178Source ClinicalTrials.gov
See all 178 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Age 5-10 Years | 1 | 20 |
| Total, all-cause mortality | Age 11-17 Years | 1 | 23 |
| Total, other adverse events | Age 5-10 Years | 19 | 20 |
| Total, other adverse events | Age 11-17 Years | 23 | 23 |
| Total, serious adverse events | Age 5-10 Years | 4 | 20 |
| Total, serious adverse events | Age 11-17 Years | 7 | 23 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39426393 | 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Age 5-10 Years vs Age 11-17 Years: deaths from any cause | Trial comparisons |
| Age 5-10 Years vs Age 11-17 Years: serious adverse events | Trial comparisons |
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/NCT04681170
GET https://conductscience.org/api/trials/v1/NCT04681170/results?page=2