SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney Disease

NCT06283589 | Completed Phase 1 FDA Drug

• 1 other identifier: INZ701-401
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 2

Brief Summary

This Phase 1, open-label, multicenter study evaluated the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in adults with hemodialysis-dependent end-stage kidney disease (ESKD) and low plasma inorganic pyrophosphate (PPi) levels. Participants received INZ-701 1.8 mg/kg subcutaneously once weekly for 4 weeks. The study assessed changes in plasma PPi levels, PK characteristics, ENPP1-Fc activity, safety, tolerability, and immunogenicity.

Conditions

Calciphylaxis

Keywords

calcific uremic arteriolopathy; calcinosis cutis; calcification; calcium metabolism disorders; mineral bone disorder (MBD); fusion protein; vascular calcification; end stage renal disease (ESKD); chronic kidney disease (CKD); calcinosis

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Plasma Inorganic Pyrophosphate (PPi) Concentrations

  Change from baseline in pre-hemodialysis plasma inorganic pyrophosphate concentrations throughout the study.

  Baseline through Follow-up Period (up to 12 months)

Secondary Outcomes (5)

• Serum INZ-701 Concentrations

  Day 1 to Day 26

• Assess the Maximum Serum Concentration (Cmax) of INZ-701

  26 days (Treatment Period)

• Assess the Area under the concentration-time curve over the dosing interval (AUCtau)

  26 days (Treatment Period)

• Assess the Clearance after extravascular administration of drug (CL/F)

  26 days (Treatment Period)

• Assess ENPP1 Activity

  26 days (Treatment Period)

Study Arms (1)

INZ-701

EXPERIMENTAL

Participants received INZ-701 1.8 mg/kg administered subcutaneously once weekly on Days 3, 10, 17, and 24.

Drug: INZ-701

Interventions

INZ-701 DRUG

Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Also known as: (rhENPP1-Fc).

INZ-701

Eligibility Criteria

• Age: 18 Years - 69 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Study participants must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Council for Harmonisation (ICH) Good Clinical Practice (GCP)
• Have ESKD and are receiving HD treatment
• Are compliant with receiving 3 treatments of HD per week with a functioning arteriovenous (AV) fistula, AV graft, or central venous catheter
• PPi level \<700 nmol/L at Screening
• Must be willing and able to comply with the diet prescribed by their treating physician and/or the Investigator
• Male or female aged \>18 years to \<70 years
• Women of child-bearing potential (WOCBP) as defined in Clinical Trials Coordination Group (CTFG 2020) and provided in Appendix B, must have a negative serum pregnancy test at Screening and within 3 days of INZ-701 administration
• WOCBP and partners of fertile males who are WOCBP must be using or agree to use one highly effective form of contraception (per CTFG 2020) and a barrier method from at least 1 month before the first dose of INZ-701 to 30 days after the last dose (greater than 5 half-lives of INZ-701). WOCBP and partners of fertile males who are WOCBP must also agree to not donate ova from the time of the first INZ-701 dose to 30 days after the last dose.
• Males who are sexually active must agree to use condoms from the time of the first INZ-701 dose to 30 days after the last dose. Males must also agree to not donate sperm from the time of the first INZ-701 dose to 30 days after the last dose.
• In the opinion of the Investigator, study participants are able and willing to complete all study procedures per protocol.

You may not qualify if:

• Study participants receiving other types of dialysis than HD (eg, peritoneal dialysis, hemodiafiltration)
• Study participants who are hospitalized
• In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that may impact study participation and/or confound interpretation of the study results
• Malignancy within the last year, except non-melanoma skin cancers or cervical carcinoma in situ
• Advanced liver disease manifesting as liver cirrhosis
• Myocardial infarction, stroke, or congestive heart failure requiring hospitalization within the last 6 months
• Known intolerance to INZ-701 or any of its excipients
• Weight \>125 kg
• Concurrent participation in another interventional clinical study and/or receipt of any other investigational new drug within 5 half-lives of the last dose of the other investigational product or from 4 weeks prior to the first dose of INZ-701, whichever is longer, or use of an investigational device, through completion of participation in the study

Sponsors & Collaborators

• Inozyme Pharma: lead
• BioMarin Pharmaceutical: collaborator

Study Sites (2)

South Florida Nephrology Research

Coral Springs, Florida, 33071, United States

Location

Elixia Health

Hollywood, Florida, 33024, United States

Location

MeSH Terms

Conditions

Calciphylaxis; Calcinosis Cutis; Calcinosis; Calcium Metabolism Disorders; Vascular Calcification; Kidney Failure, Chronic; Renal Insufficiency, Chronic

Condition Hierarchy (Ancestors)

Metabolic Diseases; Nutritional and Metabolic Diseases; Skin Diseases; Skin and Connective Tissue 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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single-group assignment. All enrolled participants received INZ-701 1.8 mg/kg administered subcutaneously once weekly for 4 weeks. The study was designed to evaluate the safety, pharmacokinetics, and pharmacodynamics of INZ-701 in adults with hemodialysis-dependent end-stage kidney disease and low plasma inorganic pyrophosphate (PPi) levels. No placebo or active comparator group was included.

Study Record Dates

• First Submitted: February 21, 2024
• First Posted: February 28, 2024
• Study Start: February 12, 2024
• Primary Completion: July 27, 2024
• Study Completion: December 13, 2024
• Last Updated: August 24, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)