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

NCT04239391 | Unknown Phase 3 FDA Drug

• 1 other identifier: RMFPC-22
• Study Type: interventional
• Target: 150
• Locations: 2 countries
• Sites: 9

Brief Summary

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.

Conditions

End Stage Renal Disease

Keywords

dialysis; pediatric; chronic kidney disease; anemia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Assess the ability of Triferic to maintain hemoglobin in pediatric CKD-5HD patients

  44 weeks

• Assess the proportion of patients maintaining hemoglobin between 10.5-12.0 g/dL compared to baseline

  44 weeks

• Assess the change in reticulocyte hemoglobin content (CHr).

  44 weeks

Study Arms (2)

Triferic via IV and Hemodialysate

EXPERIMENTAL

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

Historic Control Observational Arm

NO INTERVENTION

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.

Interventions

Triferic DRUG

Ferric Pyrophosphate Citrate

Also known as: FPC

Triferic via IV and Hemodialysate

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Patient is between 6 months and \<18 years of age at screening.
• Patient has chronic kidney disease receiving in-center hemodialysis at least twice weekly for at least 3 months prior to screening.
• Patient is receiving adequate hemodialysis as assessed by the investigator and based on a single pool Kt/V measurement \>1.2.
• Patient has a vascular access (tunneled catheter, AV fistula or AV graft) suitable to support blood flows for hemodialysis treatment.
• Patient has a body mass of ≥11 lbs (5 kg).
• Patient is iron-replete as measured by a TSAT ≥ 20% and a ferritin \>100 µg/L at screening.
• Patient has a whole blood Hgb concentration of ≥ 9.5 g/dL at screening.
• 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.
• 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.

You may not qualify if:

• Patient is positive for human immunodeficiency virus (HIV) or hepatitis B by history.
• 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.)
• Patient has evidence of an ongoing active inflammatory process (e.g., systemic lupus erythematosus, acute or chronic active hepatitis, etc.) requiring treatment.
• Patient has been dosed in an investigational drug study within the 30 days prior to Baseline.
• 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).

Sponsors & Collaborators

• Rockwell Medical Technologies, Inc.: lead

Study Sites (9)

Loma Linda University Hospital

Loma Linda, California, 92354, United States

RECRUITING

Childrens Hospital National Medical Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Riley Hospital for Children at Indiana University

Indianapolis, Indiana, 46202, United States

RECRUITING

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

RECRUITING

Carolina's Medical Center

Charlotte, North Carolina, 28203, United States

RECRUITING

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

RECRUITING

University of Texas Health Science Center At San Antonio

San Antonio, Texas, 78229, United States

RECRUITING

Childrens Hospital and Medical Center- Seattle

Seattle, Washington, 98105, United States

RECRUITING

University of Puerto Rico School of Medicine

San Juan, 00963-5067, Puerto Rico

RECRUITING

MeSH Terms

Conditions

Kidney Failure, Chronic; Renal Insufficiency, Chronic; Anemia

Interventions

ferric pyrophosphate

Condition Hierarchy (Ancestors)

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; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Raymond D Pratt, MD FACP

  Rockwell Medical, Inc

  STUDY DIRECTOR

Central Study Contacts

Emily Akin

CONTACT

913-485-9082; eakin@georgeclinical.com

Terrilyn Sharpe, MSHS

CONTACT

248-819-3088; tsharpe@rockwellmed.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2020
• First Posted: January 27, 2020
• Study Start: September 1, 2020
• Primary Completion: August 31, 2023
• Study Completion: November 1, 2023
• Last Updated: April 21, 2021

Record last verified: 2021-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (8); PR (1)