Trial · NCT02855268
Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
NCT02855268, Phase 2, Terminated, Genzyme, a Sanofi Company; results first posted 2023-10-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Lademirsen (SAR339375) in Patients With Alport Syndrome. Trials, record NCT02855268. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02855268
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
- NCT number
- NCT02855268
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 43 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Alport Syndrome
- Interventions
- lademirsen (SAR339375), Placebo
- First posted
- 2016-08-04
- Last updated
- 2025-09-11
- Other registries' IDs
- eudract:2019-004394-10
- Results first posted
- 2023-10-23
- Limitations and caveats
- Due to early study termination, Stage 2/Cohort 2 was not conducted. In Stage 1 analysis of DB period was performed until 48 weeks. In Stage 1 OL period, only safety data was collected and assessed.
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB: from 1st dose of IMP upto 1st dose of IMP in OLE for participant who entered OLE (Week 48); up to 7 days post last dose for participant not continuing to OLE (Week 49); OLE:1st dose of IMP (at Week 48) in OLE upto 10 weeks post last dose (Week 106) | Adverse event (AE): any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. Serious adverse event (SAE) was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event. TEAEs: AEs with onset after the first dose of investigational medicinal product (IMP) or existing AEs that worsened during TEAE Period (for DB Period: from first IMP administration up to first administration in OLE period for participant who entered OLE period; and up to 7 days post last IMP administration for participant not continuing OLE period; for open-label: time from 1st IMP administration in open-label to last IMP administration+ 10 weeks). |
| Primary | DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48 | Baseline, Week 48 | Annualized change in eGFR was calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Creatinine Equation (for participants with age greater than 16 years) as: eGFR=142\*min(Scr/K, 1)α\*max(Scr/K, 1)\^-1.200\*0.9938\^Age\*1.012 \[if female\], where Scr = serum creatinine in milligrams per deciliter (mg/dL), K = 0.7 for females (F) and 0.9 for males (M), α = -0.241(F) and -0.302(M); age=years, calculated at time of creatinine measurement. eGFR measurements collected from baseline to Week 48 were the response variable and included fixed effects of treatment (lademirsen or placebo), screening eGFR stratification factor (less than \[\<\]60 versus greater than or equal to \[\>=\]60 milliliters per minute per 1.73 meters squared \[mL/min/1.73 m\^2\]), time, and treatment-by-time interaction. Least square (LS) mean and standard error (SE) estimated by linear mixed effect model. |
| Secondary | DB Period: Absolute Change From Baseline in eGFR Values at Week 24 and 48 | Baseline, Weeks 24 and 48 | eGFR was used to measure level of kidney function and determine the stage of kidney disease. eGFR was calculated using CKD-EPI Creatinine Equation as: eGFR =142\*min (Scr/K, 1) α\*max (Scr/K, 1)\^-1.200\*0.9938\^Age\*1.012 \[if female\], where Scr was serum creatinine in mg/dL, K is 0.7 for females and 0.9 for males, α was -0.241 for females and -0.302 for males. Unit of age was years, calculated to reflect the age at the time when creatinine was measured. |
| Secondary | DB Period: Percent Change From Baseline in eGFR Values at Week 24 and 48 | Baseline, Weeks 24 and 48 | eGFR was used to measure level of kidney function and determine the stage of kidney disease. eGFR was calculated using CKD-EPI Creatinine Equation as: eGFR =142\*min (Scr/K, 1) α\*max (Scr/K, 1)\^-1.200\*0.9938\^Age\*1.012 \[if female\], where Scr was serum creatinine in mg/dL, K is 0.7 for females and 0.9 for males, α was -0.241 for females and -0.302 for males. Unit of age was years, calculated to reflect the age at the time when creatinine was measured. |
| Secondary | DB Period: Number of Participants With a Reduction From Baseline in eGFR of <10%, <20%, <30%, or <40% at Weeks 24 and 48 | At Weeks 24 and 48 | Estimated glomerular filtration rate was used to measure level of kidney function and determine the stage of kidney disease. eGFR was calculated using CKD-EPI Creatinine Equation as: eGFR = 142\*min(Scr/K,1)α\*max(Scr/K,1)\^-1.200\*0.9938\^Age\*1.012 \[if female\], where Scr was serum creatinine in mg/dL, K is 0.7 for females and 0.9 for males, α was -0.241 for females and -0.302 for males. Number of participants with a reduction from baseline in eGFR value of \<10%, \<20%, \<30%, or \<40% at Weeks 24 and 48 were reported in this outcome measure. |
| Secondary | DB Period: Number of Participants Who Developed End Stage Renal Disease (ESRD) | From Baseline up to Week 48 | ESRD was defined as: eGFR \<=15 mL/min/1.73 m\^2; or initiation of hemodialysis; or receiving a renal transplantation during the double-blind treatment period. |
| Secondary | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities (PCSA): Hematological Parameters | From Baseline up to Week 48 | Criteria for PCSA included: Hemoglobin (Hb): \<= 115 grams per liter (g/L) (Male), \<= 95 g/L (Female); greater than or equal to (\>=) 185 g/L (18.5 g/dL) (Male), \>= 165 g/L (16.5g/dL) (Female); decrease from Baseline (DFB) = 20 g/L (2g/dL); Hematocrit: \<= 0.37 volume/volume (v/v) (Male); \<= 0.32 v/v (Female); \>= 0.55 v/v (Male); \>= 0.5 v/v (Female); Red Blood Cells (RBCs):\>=6 Tera/ liter (L) and Platelets: \<100 Giga/L; \>= 700 Giga/L. |
| Secondary | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters | From Baseline up to Week 48 | Criteria for potentially clinically significant abnormalities: Creatinine: \>=150 micromol/L (adults); \>=30% change from baseline, \>=100% change from baseline; Blood urea nitrogen: \>=17 millimoles per liter (mmol/L); Uric acid: \<120 micromol/L; \>408 micromol/L; Creatinine clearance: \<15 mL/min; \>=15 to \<30 mL/min; \>=30 to \<60 mL/min; \>=60 to \<90 mL/min; \>=90 mL/min; eGFR: \< 15 mL/min/1.73m\^2; \>=15 to \<30 mL/min/1.73m\^2; \>=30 to \<60 mL/min/1.73m\^2; \>=60 to \<90 mL/min/1.73m\^2; \>=90 mL/min. Participants might be counted more than once for specified categories. |
| Secondary | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | From Baseline up to Week 48 | Criteria for PCSA: Total bilirubin (TBILI): \>1.5 upper limit of normal (ULN); \>2 ULN; Alanine Aminotransferase (ALT): \>3 ULN; \>5 ULN; \>10 ULN; \>20 ULN; Aspartate aminotransferase (AST): \>3ULN; \>5 ULN; \>10 ULN; \>20 ULN; Alkaline phosphatase: \>1.5 ULN; ALT\>3 ULN and TBILI\>2 ULN and Direct Bilirubin\> 35% TBILI and TBILI\> 1.5 ULN. |
| Secondary | DB Period: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs | From Baseline up to Week 48 | Criteria for potentially clinically significant vital sign abnormalities: Systolic blood pressure (SBP):\<=95 mmHg and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg; SBP (Orthostatic): \<=-20mmHg; Diastolic blood pressure (DBP): \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg; DBP (Orthostatic): \<=10 mmHg; heart rate (HR): \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB\>=20 bpm and Weight: \>=5% DFB; \>=5% IFB. |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB: from 1st dose of IMP upto 1st dose of IMP in OLE for participant who entered OLE (Week 48); up to 7 days post last dose for participant not continuing to OLE (Week 49); OLE:1st dose of IMP (at Week 48) in OLE upto 10 weeks post last dose (Week 106) | Analysis was performed on safety population that included all participants who received at least one dose or partial of a dose of the IMP, analyzed according to the treatment actually received. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48 | Baseline, Week 48 | Analysis was performed on primary population that included all randomized participants who completed the double-blinded period (the first 48 weeks) or discontinued double-blinded period early. | mL/min/1.73 m^2/year | LEAST_SQUARES_MEAN |
| SECONDARY | DB Period: Absolute Change From Baseline in eGFR Values at Week 24 and 48 | Baseline, Weeks 24 and 48 | Analysis was performed on Intent-to-Treat (ITT) population that included all randomized participants analyzed according to the treatment group allocated by randomization. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. | mL/min/1.73 m^2 | LEAST_SQUARES_MEAN |
| SECONDARY | DB Period: Percent Change From Baseline in eGFR Values at Week 24 and 48 | Baseline, Weeks 24 and 48 | Analysis was performed on ITT population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure and 'number analyzed' = participants with available data for each specified category. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | DB Period: Number of Participants With a Reduction From Baseline in eGFR of <10%, <20%, <30%, or <40% at Weeks 24 and 48 | At Weeks 24 and 48 | Analysis was performed on ITT population. Here, 'number analyzed' = participants considered for the analysis for each specified category. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Period: Number of Participants Who Developed End Stage Renal Disease (ESRD) | From Baseline up to Week 48 | Analysis was performed on ITT population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities (PCSA): Hematological Parameters | From Baseline up to Week 48 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters | From Baseline up to Week 48 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | From Baseline up to Week 48 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | DB Period: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs | From Baseline up to Week 48 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group258
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB Period: Placebo | TEAE | 14 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB Period: Lademirsen | TEAE | 29 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | OLE Period: Placebo/Lademirsen | TEAE | 8 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | OLE Period: Lademirsen/Lademirsen | TEAE | 19 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB Period: Placebo | TESAE | 0 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | DB Period: Lademirsen | TESAE | 2 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | OLE Period: Placebo/Lademirsen | TESAE | 2 | Participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) | OLE Period: Lademirsen/Lademirsen | TESAE | 1 | Participants | |
| DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48 | DB Period: Placebo | -4.70 | Standard Error 2.69 | mL/min/1.73 m^2/year | |
| DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48 | DB Period: Lademirsen | -4.91 | Standard Error 1.86 | mL/min/1.73 m^2/year |
1 to 10 of 258Source ClinicalTrials.gov
See all 258 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| DB Period (up to 48 Weeks) | STARTED | Placebo/Lademirsen | 14 | |
| DB Period (up to 48 Weeks) | STARTED | Lademirsen/Lademirsen | 29 | |
| DB Period (up to 48 Weeks) | COMPLETED | Placebo/Lademirsen | 9 | |
| DB Period (up to 48 Weeks) | COMPLETED | Lademirsen/Lademirsen | 19 | |
| DB Period (up to 48 Weeks) | NOT COMPLETED | Placebo/Lademirsen | 5 | |
| DB Period (up to 48 Weeks) | NOT COMPLETED | Lademirsen/Lademirsen | 10 | |
| OLE Period (up to 96 Weeks) | STARTED | Placebo/Lademirsen | 9 | |
| OLE Period (up to 96 Weeks) | STARTED | Lademirsen/Lademirsen | 19 | |
| OLE Period (up to 96 Weeks) | COMPLETED | Placebo/Lademirsen | 1 | |
| OLE Period (up to 96 Weeks) | COMPLETED | Lademirsen/Lademirsen | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events488
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 | Covid-19 | Infections and infestations | DB Period: Placebo | 5 | 14 | 5 |
| other | Covid-19 | Infections and infestations | DB Period: Lademirsen | 5 | 29 | 5 |
| other | Covid-19 | Infections and infestations | OLE Period: Placebo/Lademirsen | 1 | 9 | 1 |
| other | Covid-19 | Infections and infestations | OLE Period: Lademirsen/Lademirsen | 2 | 19 | 2 |
| other | Folliculitis | Infections and infestations | DB Period: Placebo | 0 | 14 | 0 |
| other | Folliculitis | Infections and infestations | DB Period: Lademirsen | 0 | 29 | 0 |
| other | Folliculitis | Infections and infestations | OLE Period: Placebo/Lademirsen | 0 | 9 | 0 |
| other | Folliculitis | Infections and infestations | OLE Period: Lademirsen/Lademirsen | 1 | 19 | 1 |
| other | Gastroenteritis | Infections and infestations | DB Period: Placebo | 0 | 14 | 0 |
| other | Gastroenteritis | Infections and infestations | DB Period: Lademirsen | 1 | 29 | 1 |
1 to 10 of 488Source ClinicalTrials.gov
See all 488 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB Period: Placebo | 0 | 14 |
| Total, all-cause mortality | DB Period: Lademirsen | 0 | 29 |
| Total, all-cause mortality | OLE Period: Placebo/Lademirsen | 0 | 9 |
| Total, all-cause mortality | OLE Period: Lademirsen/Lademirsen | 0 | 19 |
| Total, other adverse events | DB Period: Placebo | 14 | 14 |
| Total, other adverse events | DB Period: Lademirsen | 29 | 29 |
| Total, other adverse events | OLE Period: Placebo/Lademirsen | 8 | 9 |
| Total, other adverse events | OLE Period: Lademirsen/Lademirsen | 19 | 19 |
| Total, serious adverse events | DB Period: Placebo | 0 | 14 |
| Total, serious adverse events | DB Period: Lademirsen | 2 | 29 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers2
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| DB Period: Lademirsen vs DB Period: Placebo: DB Period: Number of Participants Who Developed End Stage Renal Disease (ESRD) |
Source 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/NCT02855268
GET https://conductscience.org/api/trials/v1/NCT02855268/registered?page=2