Hemoglobin Maintenance in Pediatric ESRD (End-stage Renal Disease) Patients by Ferric Pyrophosphate Citrate (FPC)

Hemoglobin Maintenance in Pediatric ESRD Patients by Ferric Pyrophosphate Citrate (FPC)

The main purpose is to determine the safety of Triferic iron administered via dialysate and intravenously in pediatric patients with chronic kidney disease on chronic hemodialysis (CKD-5HD). It is a global, multi- center, open-label study.

Study Overview

• Status: Recruiting
• Conditions: End Stage Renal Disease
• Intervention / Treatment: Drug: Triferic

Detailed Description

This is a global, multi- center, multi dose, open-label study assessing the safety of Triferic iron administered via dialysate and intravenously to pediatric patients (< 18 years of age) receiving chronic hemodialysis (CKD-5HD).

Total participation in the study is approximately 44 weeks and is comprised of a screening visit, baseline, open label treatment, and a follow-up visit.

Upon completion of the respective Baseline observational periods, all patients will transition to the interventional period where they will receive Triferic. The Triferic will be administered via the liquid bicarbonate or via IV. Once patients enter the interventional period, IV iron will only be administered if ferritin <100 µg/L and Hgb decreases by ≥0.5g/dL from the last value obtained in the observational Baseline period. Patients will remain in the interventional period for either 36 or 28 weeks (depending on randomization assignment), at which time a final study visit will take place.

• Study Type: Interventional
• Enrollment (Anticipated): 150
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Emily Akin; Phone Number: 913-485-9082; Email: eakin@georgeclinical.com
• Study Contact Backup: Name: Terrilyn Sharpe, MSHS; Phone Number: 248-819-3088; Email: tsharpe@rockwellmed.com

Study Locations

• Puerto Rico
  • San Juan, Puerto Rico, 00963-5067
    • Recruiting
    • University of Puerto Rico School of Medicine
    • Contact: Marta Suarez, MD; Phone Number: 787-759-9595; Email: marta.suarez@upr.edu
• United States
  • California
    • Loma Linda, California, United States, 92354
      • Recruiting
      • Loma Linda University Hospital
      • Contact: Jacqueline Lopez; Email: JANLopez@llu.edu
      • Principal Investigator: Rita Sheth, MD
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Recruiting
      • Childrens Hospital National Medical Center
      • Contact: Nadia Itani; Email: njitani@childrensnational.org
      • Principal Investigator: Marva Moxey Mims, MD
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Riley Hospital for Children at Indiana University
      • Contact: Sherry Wilson; Email: slw2@iu.edu
      • Principal Investigator: Neha Pottanat, MD
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Recruiting
      • Children's Mercy Hospital
      • Contact: Stephen Morrison; Email: ssmorison@cmh.edu
  • North Carolina
    • Charlotte, North Carolina, United States, 28203
      • Recruiting
      • Carolina's Medical Center
      • Contact: Jennifer Lamothe; Phone Number: 704-446-3705; Email: jennifer.lamothe@atriumhealth.org
      • Principal Investigator: Donald Weaver, MD
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Cincinnati CHildren's Hospital
      • Contact: Bliss Magella; Phone Number: 513-636-7832; Email: Bliss.Magella@cchmc.org
      • Principal Investigator: Francisco Flores, MD
  • Texas
    • San Antonio, Texas, United States, 78229
      • Recruiting
      • University of Texas Health Science Center at San Antonio
      • Contact: Sonia Januario; Email: januario@uthscsa.edu
      • Principal Investigator: Mazen Arar, MD
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Childrens Hospital and Medical Center- Seattle
      • Principal Investigator: Jodi Smith, MD
      • Contact: Megan Kelton; Phone Number: 206-987-5539; Email: megan.kelton@seattlechildrens.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 17 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

A patient will be eligible for inclusion in the study only if all of the following criteria are met:

1. Parents/legal guardians of the patient have the ability to understand the requirements of the study and have demonstrated a willingness to have their child comply with all study procedures by signing an institutional review board-approved informed consent form. Where applicable, assent of the patient has also been obtained for all study procedures prior to any study-related activities.
2. Patient is between 6 months and <18 years of age at screening.
3. Patient has chronic kidney disease receiving in-center hemodialysis at least twice weekly for at least 3 months prior to screening.
4. Patient is receiving adequate hemodialysis as assessed by the investigator and based on a single pool Kt/V measurement >1.2.
5. Patient has a vascular access (tunneled catheter, AV fistula or AV graft) suitable to support blood flows for hemodialysis treatment.
6. Patient has a body mass of ≥11 lbs (5 kg).
7. Patient is iron-replete as measured by a TSAT ≥ 20% and a ferritin >100 µg/L at screening.
8. Patient has a whole blood Hgb concentration of ≥ 9.5 g/dL at screening.
9. If the patient is female, she must be pre-pubertal, have had documented surgical sterilization prior to Baseline admission, or be practicing adequate birth control. All female patients who have reached menarche must have a negative serum pregnancy test during screening. It is the investigator's responsibility to determine whether the patient has adequate birth control for study participation.
10. Patients who have experienced a previous adverse event with IV iron products are eligible to participate in this study if the agent that caused the event is not administered during the Baseline period.

Exclusion Criteria:

A patient will not be eligible for inclusion in the study if any of the following criteria apply:

1. Patient is positive for human immunodeficiency virus (HIV) or hepatitis B by history.
2. Patient is receiving intravenous or oral antibiotics or antifungals for any infectious process. (Prophylactic antibiotics administered on a regular basis are allowed. Patients may enter the study once the infection has cleared.)
3. Patient has evidence of an ongoing active inflammatory process (e.g., systemic lupus erythematosus, acute or chronic active hepatitis, etc.) requiring treatment.
4. Patient has been dosed in an investigational drug study within the 30 days prior to Baseline.
5. Administration of iron containing phosphate binder ferric citrate (Auryxia) or sucroferric oxyhydroxide (Velphoro) within 2 weeks prior to Baseline. (Patient is only eligible if iron based binders are stopped at least 2 weeks prior to Baseline).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Triferic via IV and Hemodialysate; Upon completion of the Baseline observational periods, all enrolled patients will transition to the interventional period where they will then receive Triferic. The Triferic will be administered via the liquid bicarbonate concentrate at a dialysate concentration of 2 uM or via IV at a dose of 0.1 mg Fe/kg, if the patient does not receive dialysis using liquid bicarbonate, for up to an additional 36 weeks (depending on duration of observational Baseline period). Hgb and CHr will continue to be measured bi-weekly and iron profiles will be obtained at 4 week intervals. In the Triferic phase of the study,changes in ESA dose will be allowed according to the study site existing protocol. IV iron will only be administered if ferritin meets the criteria for iron deficiency. Patients will remain in the interventional period for either 36 or 28 weeks (depending on randomization assignment), at which time a final study visit will take place	Drug: Triferic; Ferric Pyrophosphate Citrate; Other Names: FPC
No Intervention: Historic Control Observational Arm; Up to 75 patients will be enrolled in the Observational Arm. Patients who participate in the historical control observational arm will not receive any study medication, but will have Hgb, CHr and serum iron profiles collected at 4 week intervals for up to a total of 44 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess the incidence of adverse events of Triferic administration via dialysate and IV in pediatric CKD-5HD patients	Incidence and severity of adverse events compared to Baseline.	44 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess the ability of Triferic to maintain hemoglobin in pediatric CKD-5HD patients	Change from Baseline in hemoglobin concentration	44 weeks
Assess the proportion of patients maintaining hemoglobin between 10.5-12.0 g/dL compared to baseline	Proportion of patients maintaining hemoglobin between 10.5 - 12.0 g/dL compared toBaseline	44 weeks
Assess the change in reticulocyte hemoglobin content (CHr).	Change from Baseline in reticulocyte hemoglobin content (CHr).	44 weeks

Collaborators and Investigators

• Sponsor: Rockwell Medical Technologies, Inc.
• Investigators: Study Director: Raymond D Pratt, MD FACP, Rockwell Medical, Inc

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2020
• Primary Completion (Anticipated): August 31, 2023
• Study Completion (Anticipated): November 1, 2023

Study Registration Dates

• First Submitted: January 20, 2020
• First Submitted That Met QC Criteria: January 20, 2020
• First Posted (Actual): January 27, 2020

Study Record Updates

• Last Update Posted (Actual): April 21, 2021
• Last Update Submitted That Met QC Criteria: April 19, 2021
• Last Verified: February 1, 2021

More Information

Terms related to this study

• Keywords: pediatric; anemia; chronic kidney disease; dialysis
• Additional Relevant MeSH Terms: Urologic Diseases; Renal Insufficiency; Renal Insufficiency, Chronic; Kidney Diseases; Kidney Failure, Chronic
• Other Study ID Numbers: RMFPC-22

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No