AnemiaPhase 2NCT03552393
Ascertain the Optimal Starting Dose of Mircera Given Subcutaneously for Maintenance Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease on Dialysis or Not Yet on Dialysis.
A Phase 2 study testing Mircera in people with Anemia and Renal Insufficiency, Chronic, run by Hoffmann-La Roche at 22 sites in 7 countries.
Trial summary
Ascertain the starting dose of Mircera given subcutaneously for the maintenance treatment of anemia in pediatric participants with chronic kidney disease (CKD) on dialysis or not yet on dialysis when switching from stable subcutaneous (SC) maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa. Read more about this study
- Who is it for?
- Ages 3 months to 17 years
- Conditions the study is for
- Anemia, Renal Insufficiency, Chronic
- What is being tested?
- Mircera
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 22 sitesin 7 countries
- Study dates
- 3 Aug 2018to 19 Jul 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Pediatric participants 3 months to 17 years of age with clinically stable chronic renal anemia
- CKD with estimated glomerular filtration rate (eGFR) of < 45 mL/min/1.73 m2 (determined by the Bedside Schwartz formula) or dialysis treatment for at least 8 weeks before the first dose of Mircera
- For participants on peritoneal dialysis (PD): a weekly Kt/V≥ 1.8
- For participants on hemodialysis (HD): adequate HD, urea reduction ratio (URR) > 65% or Kt/V > 1.2 for participants on HD three times per week.
- Participants with fewer than or more than three HD sessions per week should have a weekly Kt/V≥ 3.6.
- Baseline Hb concentration 10.0-12.0 g/dL determined from the mean of two Hb values measured at Visit 1 (Week -3) and Visit 2 (Week -1)
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- Stable SC maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa with the same dosing interval for at least 6 weeks before the first dose of Mircera
- Stable dose of epoetin alfa, epoetin beta, or darbepoetin alfa treatment with no weekly dose change > 25% (increase or decrease) for at least 4 weeks before the first dose of Mircera
- Adequate iron status defined as ferritin≥100 ng/mL or transferrin saturation (TSAT)≥ 20% (or percentage of hypochromic red cells < 10%); mean of two values measured during screening.
Who cannot join
- Overt gastrointestinal bleeding within 8 weeks before screening or during the screening period
- RBC transfusions within 8 weeks before screening or during the screening period
- Hemoglobinopathies (e.g., homozygous sickle-cell disease, thalassemia of all types) Hemolytic anemia, Active malignant disease
- PD subjects with an episode of peritonitis within the past 30 days prior to screening and/or during the screening period
- Uncontrolled or symptomatic inflammatory disease (e.g., systemic lupus erythematosus)
- Uncontrolled hypertension as assessed by the investigator
Show all 14Show fewer
- Epileptic seizures within 3 months prior to screening and during the screening period
- Administration of any investigational drug within 4 weeks prior to screening or planned during the study
- Severe hyperparathyroidism (intact parathyroid hormone [PTH]≥ 1000 pg/mL or whole PTH≥ 500 pg/mL) or biopsy-proven bone marrow fibrosis
- Kidney transplant with use of immunosuppressive therapies known to exacerbate anemia
- Known hypersensitivity to recombinant human erythropoietin (EPO), polyethylene glycol, or any constituent of the study drug formulation
- Anti-EPO antibody (AEAB)-mediated pure red cell aplasia (PRCA) or history of AEAB mediated PRCA or positive AEAB test result in the absence of PRCA
- High likelihood of early withdrawal or interruption of the study (e.g., planned living donor kidney transplant within 5 months of study start)
- Planned elective surgery during the entire study period
Trial locations and contacts
22 sites in 7 countries.
22 sites in 7 countries
United States7 sites
Emory University School of Med; Pediatrics
Atlanta, Georgia, 30322
University of Alabama at Birmingham; Pediatric Nephrology
Birmingham, Alabama, 35233
UT Southwestern Medical Center; Pediatrics Dept.
Dallas, Texas, 75390
East Carolina University; Brody School of Medicine
Greenville, North Carolina, 27834
Children'S Mercy Hospital; Pediatric Nephrology
Kansas City, Missouri, 64108
Loma Linda University health
Loma Linda, California, 92354
RWJBarnabas Health
West Orange, New Jersey, 07052
Poland5 sites
Uniwersyteckie Centrum Kliniczne; Klinika Chorob Nerek i Nadciśnienia Dzieci i Mlodziezy
Gdansk, 80-952
Uniwersytecki Szpital Dziecięcy w Krakowie; Oddz.Nefrologii i Nadciśnienia Tętniczego/Stacja Dializ
Krakow, 30-663
Instytut "Centrum Zdrowia Matki Polki; Klinika Pediatrii i Immunologii i Nefrologii
Lodz, 93-338
Szpital Specjalistyczny dla Dzieci i Doroslych; Oddzial Kliniczny Pediatrii i Nefrologii
Torun, 87-100
Szpital Kliniczny nr 1 im. prof. Szyszko; Oddz. Nefrologii Dzieciecej z Pododdziałem Dializoterapii
Zabrze, 41-800
France3 sites
Hopital Jeanne De Flandre; Pediatrie
Lille, 59037
Gh Necker Enfants Malades; Nephrologie
Paris, 75743
Höpital Hautepierre; Pediatrie 1
Strasbourg, 67098
Hungary2 sites
Semmelweis University; 1st Department of Pediatrics, Pediatric Nephrology Center
Budapest, 1083
Debreceni Egyetem Klinikai Központ; Gyermekklinika
Debrecen, 4032
Italy2 sites
Clinica Pediatrica II De Marchi
Milan, Lombardy, 20122
Ospedale Infantile Regina Margherita; U.O. Autonoma di Nefrologia, Dialisi e Trapianto
Turin, Piedmont, 10126
Spain2 sites
Hospital Universitari Vall d'Hebron; Servicio de Nefrologia
Barcelona, 08035
Hospital Universitario Virgen del Rocio; Servicio de Nefrologia Pediatrica
Seville, 41013
About this study
Official title: An Open-Label, Single-Arm, Multicenter Study to Ascertain the Optimal Starting Dose of MIRCERA® Given Subcutaneously for the Maintenance Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease on Dialysis or Not Yet on Dialysis.
Ascertain the starting dose of Mircera given subcutaneously for the maintenance treatment of anemia in pediatric participants with chronic kidney disease (CKD) on dialysis or not yet on dialysis when switching from stable subcutaneous (SC) maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa.
- Conditions
- Anemia, Renal Insufficiency, Chronic
- Being tested
- Mircera
- Medical subject headings
- Anemia, Renal Insufficiency, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MirceraDrug
Also called Methoxy polyethylene glycol-epoetin beta
The initial dose of Mircera will be one of nine starting doses corresponding to the prefilled syringe strengths based on the total weekly erythropoiesis-stimulating agent (ESA) dose during the screening period.
| Study group | Role | What they receive |
|---|---|---|
| Mircera | Experimental | Mircera will be administered subcutaneously once every 4 weeks Receives: Drug: Mircera |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Hemoglobin (Hb) Concentration Between the Baseline and the Evaluation Period for Each Patient
The Hb change from baseline was calculated on a per-participant basis using an individual's average for both the baseline and evaluation periods and taking the difference. The baseline period was defined as the time between the day of first study dose and the previous 35 days. The evaluation period was defined as the period between Week 17 and Week 21, inclusive.
Measured: Baseline up to Week 21
Secondary outcomes (6)
Number of Participants With an Average Hb Concentration During the Evaluation Period Within ± 1 g/dL of Their Baseline Hb and Above, Within or Below the Range of 10-12 g/dL
Number of participants with an average Hb concentration during the evaluation period within ± 1 g/dL of their baseline Hb is reported as well as the number of participants with an average Hb concentration above, within or below the range of 10-12 g/dL. The evaluation period was defined as the period between Week 17 and Week 21 inclusive.
Measured: Week 17 up to Week 21
Mean Hb Values and Change From Baseline
The mean Hb concentration over time and the mean change in Hb from baseline over time are presented.
Measured: Baseline, Weeks 3, 5, 9, 13, 17, 19, 21, 25, 29, 33, 37, 41, 45
Change in Mircera Dose Over Time
A dose change was defined as a change in the administered dose strength compared to the preceding dose.
Measured: Week 1 to Week 17
Ratio of Mircera Starting Dose (Week 1) to the Dose at Week 17
The ratio of Mircera dose was calculated as the median (min-max) ratio of starting dose (Week 1) to the dose at Week 17. Participants who withdrew before Week 17 or who were not administered a Mircera dose at Week 17 visit due to the applicable dose adjustment rules were excluded from the ratio computation.
Measured: Week 1, Week 17
Number of Participants With Adverse Events by Severity as Assessed by Highest World Health Organization (WHO) Toxicity Grade
An adverse event is any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product, any new disease, or exacerbation of existing disease (a worsening in the character, frequency, or severity of a known condition), recurrence of an intermittent medical condition or any deterioration in a laboratory value or other clinical test.
Measured: Baseline up to Week 45
Bioavailability (F) of Mircera in Pediatric Participants Based on Population PK Model
Bioavailability (F) is defined as the percentage of the administered drug, that reaches the systemic circulation. A population PK model was developed for Mircera that adequately describes pediatric data: a 1-compartment model with first order absorption and elimination processes. The bioavailability (F) was estimated using all the data points listed under time frame using the population PK model.
Measured: Pre-dose at Week 1, 9, 17; Post-dose at Week 3, Week 19 and additional sample taken between 24 hours and 5 days at participant's convenience
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2018
- Study start3 Aug 2018
- Primary completion19 Jul 2021
- Study completion19 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AnemiaStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03552393
- Sponsor's protocol ID
- NH19708
- Other IDs
- 2016-004779-39, 2016
- Status last verified
- Mar 2022
- First submitted
- 27 Apr 2018
- Last updated
- 7 Mar 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Warady BA, Meyer Reigner S, Tirodkar C, Drozdz D. Subcutaneous C.E.R.A. for the Maintenance Treatment of Anemia in Pediatric Patients With CKD: A Phase 2, Open-Label, Single-Arm, Multicenter Study. Am J Kidney Dis. 2023 Jun;81(6):684-694.e1. doi: 10.1053/j.ajkd.2022.11.006. Epub 2022 Dec 29.
PubMed 36587890
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Single-Arm, Multicenter Study to Ascertain the Optimal Starting Dose of MIRCERA® Given Subcutaneously for the Maintenance Treatment of Anemia in Pediatric Patients With Chronic Kidney Disease on Dialysis or Not Yet on Dialysis.
- ClinicalTrials.gov ID
- NCT03552393
- Acronym / sponsor ID
- NH19708
- Conditions
- Anemia, Renal Insufficiency, Chronic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 3 Aug 2018 (actual)
- Primary completion
- 19 Jul 2021 (actual)
- Study completion
- 19 Jul 2021 (actual)
- First posted
- 11 Jun 2018 (actual)
- Results
- Posted 7 Mar 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Ascertain the starting dose of Mircera given subcutaneously for the maintenance treatment of anemia in pediatric participants with chronic kidney disease (CKD) on dialysis or not yet on dialysis when switching from stable subcutaneous (SC) maintenance treatment with epoetin alfa, epoetin beta, or darbepoetin alfa.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mircera | Experimental | Drug: MirceraMircera will be administered subcutaneously once every 4 weeks |
Who may qualify
- Ages
- 3 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Pediatric participants 3 months to 17 years of age with clinically stable chronic renal anemia
- CKD with estimated glomerular filtration rate (eGFR) of < 45 mL/min/1.73 m2 (determined by the Bedside Schwartz formula) or dialysis treatment for at least 8 weeks before the first dose of Mircera
- For participants on peritoneal dialysis (PD): a weekly Kt/V≥ 1.8
- For participants on hemodialysis (HD): adequate HD, urea reduction ratio (URR) > 65% or Kt/V > 1.2 for participants on HD three times per week.
- Participants with fewer than or more than three HD sessions per week should have a weekly Kt/V≥ 3.6.
Key exclusion criteria (14 listed)
- Overt gastrointestinal bleeding within 8 weeks before screening or during the screening period
- RBC transfusions within 8 weeks before screening or during the screening period
- Hemoglobinopathies (e.g., homozygous sickle-cell disease, thalassemia of all types) Hemolytic anemia, Active malignant disease
- PD subjects with an episode of peritonitis within the past 30 days prior to screening and/or during the screening period
- Uncontrolled or symptomatic inflammatory disease (e.g., systemic lupus erythematosus)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MirceraAlso: Methoxy polyethylene glycol-epoetin betaThe initial dose of Mircera will be one of nine starting doses corresponding to the prefilled syringe strengths based on the total weekly erythropoiesis-stimulating agent (ESA) dose during the screening period. | Drug | Mircera |
Primary outcome
Change in Hemoglobin (Hb) Concentration Between the Baseline and the Evaluation Period for Each Patient
Time frame: Baseline up to Week 21
The Hb change from baseline was calculated on a per-participant basis using an individual's average for both the baseline and evaluation periods and taking the difference. The baseline period was defined as the time between the day of first study dose and the previous 35 days. The evaluation period was defined as the period between Week 17 and Week 21, inclusive.
6 secondary outcomes
Number of Participants With an Average Hb Concentration During the Evaluation Period Within ± 1 g/dL of Their Baseline Hb and Above, Within or Below the Range of 10-12 g/dL
Time frame: Week 17 up to Week 21
Mean Hb Values and Change From Baseline
Time frame: Baseline, Weeks 3, 5, 9, 13, 17, 19, 21, 25, 29, 33, 37, 41, 45
Change in Mircera Dose Over Time
Time frame: Week 1 to Week 17
Ratio of Mircera Starting Dose (Week 1) to the Dose at Week 17
Time frame: Week 1, Week 17
Number of Participants With Adverse Events by Severity as Assessed by Highest World Health Organization (WHO) Toxicity Grade
Time frame: Baseline up to Week 45
Bioavailability (F) of Mircera in Pediatric Participants Based on Population PK Model
Time frame: Pre-dose at Week 1, 9, 17; Post-dose at Week 3, Week 19 and additional sample taken between 24 hours and 5 days at participant's convenience
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36587890Warady BA, Meyer Reigner S, Tirodkar C, Drozdz D. Subcutaneous C.E.R.A. for the Maintenance Treatment of Anemia in Pediatric Patients With CKD: A Phase 2, Open-Label, Single-Arm, Multicenter Study. Am J Kidney Dis. 2023 Jun;81(6):684-694.e1. doi: 10.1053/j.ajkd.2022.11.006. Epub 2022 Dec 29.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
22 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mircera in people with Anemia and Renal Insufficiency, Chronic, run by Hoffmann-La Roche at 22 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, Poland, France, Hungary, Italy and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Mar 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Hemoglobin (Hb) Concentration Between the Baseline and the Evaluation Period for Each Patient (time frame: Baseline up to Week 21). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03552393, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2022.