Clinical trial NCT04239391: Hemoglobin Maintenance in Pediatric ESRD (End-stage Renal Disease) Patients by Ferric Pyrophosphate Citrate (FPC)
Official title: Hemoglobin Maintenance in Pediatric ESRD Patients by Ferric Pyrophosphate Citrate (FPC)
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: End Stage Renal Disease
Keywords: dialysis
pediatric
chronic kidney disease
anemia
Lead sponsor: Rockwell Medical Technologies, Inc.
Sponsor class: INDUSTRY
First posted: 2020-01-27
Enrollment: 150
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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).

Primary outcomes:
measure: Assess the incidence of adverse events of Triferic administration via dialysate and IV in pediatric CKD-5HD patients
timeFrame: 44 weeks
Secondary outcomes:
measure: Assess the ability of Triferic to maintain hemoglobin in pediatric CKD-5HD patients
timeFrame: 44 weeks
measure: Assess the proportion of patients maintaining hemoglobin between 10.5-12.0 g/dL compared to baseline
timeFrame: 44 weeks
measure: Assess the change in reticulocyte hemoglobin content (CHr).
timeFrame: 44 weeks
Other outcomes:
Locations:
city: Loma Linda
country: United States
facility: Loma Linda University Hospital
state: California
city: Washington
country: United States
facility: Childrens Hospital National Medical Center
state: District of Columbia
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University
state: Indiana
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Charlotte
country: United States
facility: Carolina's Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: San Antonio
country: United States
facility: University of Texas Health Science Center At San Antonio
state: Texas
city: Seattle
country: United States
facility: Childrens Hospital and Medical Center- Seattle
state: Washington
city: San Juan
country: Puerto Rico
facility: University of Puerto Rico School of Medicine
Study officials:
affiliation: Rockwell Medical, Inc
name: Raymond D Pratt, MD FACP
role: STUDY_DIRECTOR
References: