CompletedPhase 1/2

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

United States, United Kingdom10 participantsStarted 2022-04-11

Plain-language summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant fusion protein, for the treatment of ABCC6 Deficiency. The goal of the study is to identify a dose regimen for further clinical development in the treatment of ABCC6 Deficiency.

Who can participate

Age range18 Years – 69 Years

SexALL

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Inclusion criteria

✓. Must provide written or electronic consent after the nature of the study has been explained, and prior to any research-related procedures, per International Conference on Harmonisation (ICH) Good Clinical Practice (GCP)
✓. Clinical diagnosis of PXE supported by prior genetic identification of biallelic ABCC6 mutations (ie, homozygous or compound heterozygous)
✓. Male or female, 18 to \<70 years of age at Screening
✓. Plasma PPi \<1300 nM at Screening
✓. Subjects who are being treated with statins or proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors must have been on treatment for at least 6 months prior to Screening and no new anti-lipid therapy can be introduced within 6 months of Screening
✓. Women of child-bearing potential (WOCBP) as defined in Clinical Trials Facilitation and Coordination Group (CTFG 2020) must have a negative serum pregnancy test at Screening
✓. 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 through 30 days after the last dose of INZ-701 (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 period following the first dose of INZ-701 through 30 days after last dose of INZ-701.
✓. Males who are sexually active must agree to use condoms from the period following first dose of INZ-701 through 30 days after the last dose of INZ-701. Males must also agree to not donate sperm from the period following the first dose of INZ-701 through 30 days after last dose of INZ-701.

Exclusion criteria

What they're measuring

Number of Treatment Emergent Adverse Events (TEAEs)

Timeframe: 32 days (Dose Evaluation Period)

Number of Treatment Emergent Adverse Events (TEAEs)

Timeframe: 52 weeks (Day 1 through Safety Follow-up Visit)

Trial details

NCT IDNCT05030831

SponsorInozyme Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-07-09

View on ClinicalTrials.gov More ATP-Binding Cassette Subfamily C Member 6 Deficiency trials