Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02855268
Registration number
NCT02855268
Ethics application status
Date submitted
28/07/2016
Date registered
4/08/2016
Date last updated
23/10/2023
Titles & IDs
Public title
Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
Query!
Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, Pharmacodynamics, and Pharmacokinetics of Lademirsen (SAR339375) for Subcutaneous Injection Administered Every Week in Patients With Alport Syndrome
Query!
Secondary ID [1]
0
0
2019-004394-10
Query!
Secondary ID [2]
0
0
ACT16248
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
HERA
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Alport Syndrome
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Renal and Urogenital
0
0
0
0
Query!
Kidney disease
Query!
Other
0
0
0
0
Query!
Research that is not of generic health relevance and not applicable to specific health categories listed above
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - lademirsen (SAR339375)
Treatment: Drugs - Placebo
Experimental: Placebo/Lademirsen - Participants received subcutaneous (SC) doses of placebo (matched to lademirsen) every week (QW) during the 48 weeks of double blind (DB) treatment period. Participants who received placebo and completed DB treatment period entered in open-label extension (OLE) treatment period and received lademirsen at a dose of 110 milligrams (mg) QW for an additional 48 weeks (i.e., up to Week 96).
Experimental: Lademirsen/Lademirsen - Participants received SC doses of lademirsen 110 mg QW during the 48 weeks of DB treatment period. Participants who completed DB treatment period entered in OLE treatment period and continued the same lademirsen treatment in OLE period for an additional 48 weeks (i.e., up to Week 96).
Treatment: Drugs: lademirsen (SAR339375)
Pharmaceutical form: Solution for injection Route of administration: Subcutaneous injection
Treatment: Drugs: Placebo
Pharmaceutical form: Solution for injection Route of administration: Subcutaneous injection
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
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)
Query!
Primary outcome [2]
0
0
DB Period: Annualized Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 48
Query!
Assessment method [2]
0
0
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)a*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), a = -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.
Query!
Timepoint [2]
0
0
Baseline, Week 48
Query!
Secondary outcome [1]
0
0
DB Period: Absolute Change From Baseline in eGFR Values at Week 24 and 48
Query!
Assessment method [1]
0
0
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) a*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, a 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.
Query!
Timepoint [1]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [2]
0
0
DB Period: Percent Change From Baseline in eGFR Values at Week 24 and 48
Query!
Assessment method [2]
0
0
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) a*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, a 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.
Query!
Timepoint [2]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [3]
0
0
DB Period: Number of Participants With a Reduction From Baseline in eGFR of <10%, <20%, <30%, or <40% at Weeks 24 and 48
Query!
Assessment method [3]
0
0
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)a*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, a 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.
Query!
Timepoint [3]
0
0
At Weeks 24 and 48
Query!
Secondary outcome [4]
0
0
DB Period: Number of Participants Who Developed End Stage Renal Disease (ESRD)
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [5]
0
0
DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities (PCSA): Hematological Parameters
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [6]
0
0
DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters
Query!
Assessment method [6]
0
0
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.
Query!
Timepoint [6]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [7]
0
0
DB Period: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
Query!
Assessment method [7]
0
0
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.
Query!
Timepoint [7]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [8]
0
0
DB Period: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs
Query!
Assessment method [8]
0
0
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.
Query!
Timepoint [8]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [9]
0
0
DB Period: Number of Participants With Potentially Clinically Significant Abnormalities in 12-lead Electrocardiogram (ECG) Findings
Query!
Assessment method [9]
0
0
Criteria for potentially clinically significant ECG abnormalities: HR: <50 bpm; <50 bpm and DFB >=20 bpm; <40 bpm; <40 bpm and DFB >=20 bpm; <30 bpm; <30 bpm and DFB >=20 bpm; >90 bpm; >=90 bpm and IFB >=20 bpm; >100 bpm; >=100bpm and IFB >=20 bpm; >120 bpm; >=120 bpm and IFB >=20 bpm; PR Interval: >200 millisecond(ms); >200 ms and IFB >=25%; >220 ms; >220 ms and IFB >=25%; >240 ms; >240 ms and IFB>=25%; QRS Interval: >110 ms; >110 ms and IFB >=25%; >120 ms; >120 ms and IFB >=25%; QT Interval: >500 ms and QTc Fridericia (QTc F): >450 ms; >480 ms; >500 ms; IFB >30 and <=60 ms; IFB >60 ms.
Query!
Timepoint [9]
0
0
From Baseline up to Week 48
Query!
Secondary outcome [10]
0
0
DB Period: Pharmacokinetics (PK): Plasma Concentration of Lademirsen, Its Metabolite (RG0005) and SUM (Lademirsen+RG0005)
Query!
Assessment method [10]
0
0
Post-dose (4 hours) plasma concentration of lademirsen, its active major metabolite (RG0005), and SUM (lademirsen+RG0005) on Day 1, and at Weeks 24 and 48 are reported in the outcome measure. 4-hour SUM concentrations are calculated values (sum of measured lademirsen+RG0005).
Query!
Timepoint [10]
0
0
Post-dose (4 hours) on Day 1, Weeks 24 and 48
Query!
Secondary outcome [11]
0
0
DB Period: Pharmacokinetics: Trough Plasma Concentrations (Ctrough) of SUM (Lademirsen+RG0005)
Query!
Assessment method [11]
0
0
Ctrough was measured from the pre-dose (up to 4 hours before study drug administration) plasma samples collected at Weeks 4, 12, 24, 36 and 48. SUM concentrations (lademirsen+RG0005) are measured values (assay measures lademirsen+ RG0005).
Query!
Timepoint [11]
0
0
Pre-dose (up to 4 hours before study drug administration) on Weeks 4, 12, 24, 36 and 48
Query!
Secondary outcome [12]
0
0
DB Period: Number of Participants With Treatment-emergent Anti-drug Antibodies (ADAs) Response
Query!
Assessment method [12]
0
0
ADA responses were categorized as: treatment-induced, and treatment-boosted response. Participant whose ADA status was negative at baseline but positive (ADA titer value >=50) anytime post-baseline or missing at baseline was considered to have treatment-induced ADA. Participant whose ADA status was positive at baseline (pre-existing ADA) and the ADA titer level anytime post-baseline was significantly higher (>= twice the minimum required dilution) than that at baseline was considered to have treatment-boosted ADA.
Query!
Timepoint [12]
0
0
From first IMP administration (Day 1) up to first administration in OLE period for participant who entered OLE period (i.e., up to W48) & up to 7 days post last IMP administration for participant not continuing OLE period (i.e., up to W49)
Query!
Secondary outcome [13]
0
0
DB Period: Number of Participants With Treatment-emergent Adverse Events (TEAEs) Associated With Anti-drug Antibody (ADA) Responses
Query!
Assessment method [13]
0
0
TEAEs: AE developed/worsened/became serious during TEAE period (from first IMP administration in DB period to first administration in OLE period for those who entered OLE (i.e., up to W48), up to 7 days post last dose for those not continuing to OLE period (i.e., up to W49). TESAEs: any untoward medical occurrence that resulted in death, was life-threatening, required inpatient hospitalization/prolongation of hospitalization, resulted in persistent/significant disability, was a congenital anomaly/birth defect, or a medically important event. ADA response was categorized as treatment-induced (participant whose ADA status was positive [ADA titer value >=50] anytime post-baseline and was negative/missing at baseline), treatment-boosted (participant whose ADA status was positive at baseline & ADA titer level anytime post-baseline was significantly higher). In this outcome measure, number of participants with TEAEs as per ADA responses (positive or negative) were reported.
Query!
Timepoint [13]
0
0
From first IMP administration (Day 1) up to first administration in OLE period for participant who entered OLE period (i.e., up to W48) & up to 7 days post last IMP administration for participant not continuing OLE period (i.e., up to W49)
Query!
Secondary outcome [14]
0
0
DB Period: Change From Baseline in Circulating MicroRNA-21 at Weeks 24 and 48
Query!
Assessment method [14]
0
0
Circulating microRNA-21 were the supportive biomarkers assessed in study.
Query!
Timepoint [14]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [15]
0
0
DB Period: Change From Baseline in Blood Urea Nitrogen (BUN) Values at Weeks 24 and 48
Query!
Assessment method [15]
0
0
BUN was the supportive biomarker assessed during the study. Change from Baseline in BUN at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [15]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [16]
0
0
DB Period: Change From Baseline in Urine Protein/Creatinine Ratio at Weeks 24 and 48
Query!
Assessment method [16]
0
0
Urine protein and creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine protein to creatinine ratio at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [16]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [17]
0
0
DB Period: Change From Baseline in Urine Albumin/Creatinine Ratio at Weeks 24 and 48
Query!
Assessment method [17]
0
0
Urine albumin and creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine albumin to creatinine ratio at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [17]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [18]
0
0
DB Period: Change From Baseline in Urine Epidermal Growth Factor (EGF)/Creatinine Ratio at Weeks 24 and 48
Query!
Assessment method [18]
0
0
EGF and creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine EGF to creatinine ratio at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [18]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [19]
0
0
DB Period: Change From Baseline in Blood Creatinine Values at Weeks 24 and 48
Query!
Assessment method [19]
0
0
Creatinine was the supportive biomarker assessed during the study. Change from Baseline in blood creatinine values at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [19]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [20]
0
0
DB Period: Change From Baseline in Urine Creatinine Values at Weeks 24 and 48
Query!
Assessment method [20]
0
0
Creatinine was the supportive biomarker assessed during the study. Change from Baseline in urine creatinine values at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [20]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [21]
0
0
DB Period: Change From Baseline in Blood Cystatine C Values at Weeks 24 and 48
Query!
Assessment method [21]
0
0
Cystatine C was the supportive biomarker assessed during the study. Change from Baseline in blood cystatine C values at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [21]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [22]
0
0
DB Period: Change From Baseline in Urine Cystatin C/Creatinine Ratio at Weeks 24 and 48
Query!
Assessment method [22]
0
0
Cystatin C and Creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine cystatin C to creatinine ratio at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [22]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [23]
0
0
DB Period: Change From Baseline in Blood Transforming Growth Factor Beta 1 Values at Week 24 and 48
Query!
Assessment method [23]
0
0
Transforming growth factor beta 1 was the supportive biomarker assessed during the study. Change from Baseline in blood transforming growth factor beta 1 values at Week 24 and 48 was reported in this outcome measure.
Query!
Timepoint [23]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [24]
0
0
DB Period: Change From Baseline in Urine Transforming Growth Factor Beta 1/Creatinine Ratio at Week 24 and 48
Query!
Assessment method [24]
0
0
Transforming growth factor beta 1 and creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine transforming growth factor beta 1 to creatinine ratio at Week 24 and 48 was reported in this outcome measure.
Query!
Timepoint [24]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [25]
0
0
DB Period: Change From Baseline in Blood Lipocalin-2 Values at Weeks 24 and 48
Query!
Assessment method [25]
0
0
Blood Lipocalin-2 was the supportive biomarker assessed during the study. Change from Baseline in blood lipocalin-2 at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [25]
0
0
Baseline, Weeks 24 and 48
Query!
Secondary outcome [26]
0
0
DB Period: Change From Baseline in Urine Lipocalin-2/Creatinine Ratio at Weeks 24 and 48
Query!
Assessment method [26]
0
0
Lipocalin-2 and Creatinine were the supportive biomarker assessed during the study. Change from Baseline in urine lipocalin-2 to creatinine ratio at Weeks 24 and 48 was reported in this outcome measure.
Query!
Timepoint [26]
0
0
Baseline, Weeks 24 and 48
Query!
Eligibility
Key inclusion criteria
Inclusion criteria:
- Male or female.
- Confirmed diagnosis of Alport syndrome
1. Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
2. Genetic confirmation of Alport Syndrome in the participant or the family member,
OR
3. Kidney biopsy showing glomerular basement membrane abnormalities (e.g.,
significant thinning, thickening, irregularity or lucencies) consistent with
Alport Syndrome.
- Age 18-55 years old.
- eGFR > 35 ml/min/1.73m^2 and <90 mL/min/1.73m^2 (based on CKD-EPI) at screening.
- Renal Function Criteria (participants must have met at least one of the following
CRITERIA A, B or C):
- A) Decline in eGFR of >=4 mL/min/1.73 m^2/year (eGFR slope <= -4) based on a
linear regression slope analysis of >=4 eGFR measurements within 3 years prior to
the study and with a minimum of 2-year time span (the last, of the screening
measurement, and first eGFR measurements should be separated by at least 2
years). eGFR was calculated by using either the Chronic Kidney Disease
Epidemiology Collaboration (CKD-EPI) creatinine equation.
- B) proteinuria (UPCR) >2000 mg/g (UACR>1000 mg/g).
- C) Age and sex adjusted eGFR (based on CKD-Epi; male 18-23 eGFR<90 mL/min/1.73m^2
- ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 30 days
prior to screening.
- Sexually active female participants of childbearing potential and sexually mature male
participants must have agreed to practice true abstinence in line with their preferred
and usual lifestyle or to use two acceptable effective methods of contraception for
the entire duration of the study and for at least 6 weeks after last dose.
- Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines
(including ecstasy), barbiturates, benzodiazepines, and cannabinoids. At the
Investigator's discretion, participants prescribed benzodiazepines, cannabinoids, or
opiates with positive results on a drug screen were allowed.
- Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus
(HCV) antibody, and human immunodeficiency virus (HIV) antibody.
- Normal biological tests.
- Able to understand all study procedures in the informed consent form (ICF) and to
comply with all aspects of the protocol.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
55
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
Exclusion criteria:
- Causes of chronic kidney disease aside from Alport syndrome (including but not limited
to other heritable disorders leading to chronic kidney disease, diabetic nephropathy,
hypertensive nephropathy, lupus nephritis, IgA nephropathy).
- End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of
renal transplantation.
- Any clinically significant illness within 30 days before screening or surgical or
medical condition (other than Alport syndrome) that could interfere with the
participant's study compliance; confound the study results; impact participant safety;
or significantly alter the absorption, distribution, metabolism, or excretion of
drugs.
- Weight > 110 kg.
- Any history of active malignancy within the last 1 year (history of localized basal
cell or squamous cell carcinoma and cervical carcinoma in situ that has been
excised/appropriately treated or a fully excised malignant lesion with a low
probability of recurrence will not be considered exclusionary).
- Prior treatment with Bardoxolone within 90 days prior to screening.
- History or presence of alcoholism or drug abuse within 2 years before screening or
other concurrent social conditions that would potentially interfere with the
participant's study compliance, at the discretion of the Investigator.
- Participation in a recent investigational study and receipt of an investigational drug
or investigational use of a licensed drug within 30 days or 5 half-lives, whichever
was longer, prior to screening.
- History or presence of hypersensitivity or idiosyncratic, allergic, or other
clinically significant reaction to the study drug (including placebo), inactive
ingredients, or related compounds (e.g., other oligonucleotide products).
- Any other condition or circumstance that, in the opinion of the Investigator, may make
the participant unlikely to complete the study or comply with study procedures and
requirements, or may pose a risk to the participant's safety and well-being.
The above information was not intended to contain all considerations relevant to a
participant's potential participation in a clinical trial.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Terminated
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
2/11/2019
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
22/09/2022
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
43
Query!
Recruitment in Australia
Recruitment state(s)
QLD,VIC,WA
Query!
Recruitment hospital [1]
0
0
Investigational Site Number :0360003 - Herston
Query!
Recruitment hospital [2]
0
0
Investigational Site Number :0360001 - Parkville
Query!
Recruitment hospital [3]
0
0
Investigational Site Number :0360002 - Nedlands
Query!
Recruitment postcode(s) [1]
0
0
4029 - Herston
Query!
Recruitment postcode(s) [2]
0
0
3050 - Parkville
Query!
Recruitment postcode(s) [3]
0
0
6009 - Nedlands
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Minnesota
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
New York
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Ohio
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Utah
Query!
Country [6]
0
0
China
Query!
State/province [6]
0
0
Beijing
Query!
Country [7]
0
0
China
Query!
State/province [7]
0
0
Guangzhou
Query!
Country [8]
0
0
France
Query!
State/province [8]
0
0
Paris
Query!
Country [9]
0
0
France
Query!
State/province [9]
0
0
Toulouse
Query!
Country [10]
0
0
Germany
Query!
State/province [10]
0
0
Göttingen
Query!
Country [11]
0
0
Germany
Query!
State/province [11]
0
0
Köln
Query!
Country [12]
0
0
Spain
Query!
State/province [12]
0
0
Andalucia
Query!
Country [13]
0
0
Spain
Query!
State/province [13]
0
0
Barcelona [Barcelona]
Query!
Country [14]
0
0
Spain
Query!
State/province [14]
0
0
Madrid, Comunidad De
Query!
Country [15]
0
0
Spain
Query!
State/province [15]
0
0
Granada
Query!
Country [16]
0
0
United Kingdom
Query!
State/province [16]
0
0
London, City Of
Query!
Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
Nottinghamshire
Query!
Country [18]
0
0
United Kingdom
Query!
State/province [18]
0
0
Newcastle Upon Tyne
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Genzyme, a Sanofi Company
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
Primary Objectives:
- To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal
function.
- To assess the safety and tolerability of lademirsen (SAR339375) in participants with
Alport syndrome.
Secondary Objectives:
- To assess plasma pharmacokinetic (PK) parameters of the parent compound and its active
major metabolite.
- To assess the potential formation of anti-drug antibodies (ADAs) following
administration of lademirsen (SAR339375).
- To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes
in renal injury and function biomarkers.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT02855268
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Clinical Sciences & Operations
Query!
Address
0
0
Sanofi
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02855268
Download to PDF