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.

NCT03552393 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: NH197082016-004779-39
• Study Type: interventional
• Target: 40
• Locations: 7 countries
• Sites: 22

Brief 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.

Conditions

Anemia; Renal Insufficiency, Chronic

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  Week 17 up to Week 21

• Mean Hb Values and Change From Baseline

  Baseline, Weeks 3, 5, 9, 13, 17, 19, 21, 25, 29, 33, 37, 41, 45

• Change in Mircera Dose Over Time

  Week 1 to Week 17

• Ratio of Mircera Starting Dose (Week 1) to the Dose at Week 17

  Week 1, Week 17

• Number of Participants With Adverse Events by Severity as Assessed by Highest World Health Organization (WHO) Toxicity Grade

  Baseline up to Week 45

Study Arms (1)

Mircera

EXPERIMENTAL

Mircera will be administered subcutaneously once every 4 weeks

Drug: Mircera

Interventions

Mircera DRUG

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.

Also known as: Methoxy polyethylene glycol-epoetin beta

Mircera

Eligibility Criteria

• Age: 3 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)
• 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.

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (22)

University of Alabama at Birmingham; Pediatric Nephrology

Birmingham, Alabama, 35233, United States

Location

Loma Linda University health

Loma Linda, California, 92354, United States

Location

Emory University School of Med; Pediatrics

Atlanta, Georgia, 30322, United States

Location

Children'S Mercy Hospital; Pediatric Nephrology

Kansas City, Missouri, 64108, United States

Location

RWJBarnabas Health

West Orange, New Jersey, 07052, United States

Location

East Carolina University; Brody School of Medicine

Greenville, North Carolina, 27834, United States

Location

UT Southwestern Medical Center; Pediatrics Dept.

Dallas, Texas, 75390, United States

Location

Hopital Jeanne De Flandre; Pediatrie

Lille, 59037, France

Location

Gh Necker Enfants Malades; Nephrologie

Paris, 75743, France

Location

Höpital Hautepierre; Pediatrie 1

Strasbourg, 67098, France

Location

Semmelweis University; 1st Department of Pediatrics, Pediatric Nephrology Center

Budapest, 1083, Hungary

Location

Debreceni Egyetem Klinikai Központ; Gyermekklinika

Debrecen, 4032, Hungary

Location

Clinica Pediatrica II De Marchi

Milan, Lombardy, 20122, Italy

Location

Ospedale Infantile Regina Margherita; U.O. Autonoma di Nefrologia, Dialisi e Trapianto

Turin, Piedmont, 10126, Italy

Location

Vilnius University Children's Hospital

Vilnius, LT-08406, Lithuania

Location

Uniwersyteckie Centrum Kliniczne; Klinika Chorob Nerek i Nadciśnienia Dzieci i Mlodziezy

Gdansk, 80-952, Poland

Location

Uniwersytecki Szpital Dziecięcy w Krakowie; Oddz.Nefrologii i Nadciśnienia Tętniczego/Stacja Dializ

Krakow, 30-663, Poland

Location

Instytut "Centrum Zdrowia Matki Polki; Klinika Pediatrii i Immunologii i Nefrologii

Lodz, 93-338, Poland

Location

Szpital Specjalistyczny dla Dzieci i Doroslych; Oddzial Kliniczny Pediatrii i Nefrologii

Torun, 87-100, Poland

Location

Szpital Kliniczny nr 1 im. prof. Szyszko; Oddz. Nefrologii Dzieciecej z Pododdziałem Dializoterapii

Zabrze, 41-800, Poland

Location

Hospital Universitari Vall d'Hebron; Servicio de Nefrologia

Barcelona, 08035, Spain

Location

Hospital Universitario Virgen del Rocio; Servicio de Nefrologia Pediatrica

Seville, 41013, Spain

Location

Related Publications (1)

• 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.

  PMID: 36587890 DERIVED

MeSH Terms

Conditions

Anemia; Renal Insufficiency, Chronic

Interventions

continuous erythropoietin receptor activator

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Medical Communications
• Organization: Hoffmann-La Roche

genentech@druginfo.com

800 821-8590

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2018
• First Posted: June 11, 2018
• Study Start: August 3, 2018
• Primary Completion: July 19, 2021
• Study Completion: July 19, 2021
• Last Updated: March 7, 2022
• Results First Posted: March 7, 2022

Record last verified: 2022-03

Locations

FR (3); PL (5); LI (1); ES (2); HU (2); US (7); IT (2)