INZ-701 for Pseudoxanthoma Elasticum

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Clinilabs, Eatontown, NJ
Pseudoxanthoma Elasticum+4 More
INZ-701 - Drug
Eligibility
18 - 65
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug called INZ-701 is safe and tolerable for people with a rare genetic disease called ABCC6 Deficiency.

See full description

Eligible Conditions

  • Pseudoxanthoma Elasticum
  • Generalized Arterial Calcification of Infancy
  • ATP-Binding Cassette Subfamily C Member 6 Deficiency

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Pseudoxanthoma Elasticum

Study Objectives

This trial is evaluating whether INZ-701 will improve 1 primary outcome and 5 secondary outcomes in patients with Pseudoxanthoma Elasticum. Measurement will happen over the course of 32 days (Dose Evaluation Period).

Day 32
Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701
Maximum Plasma Concentration (Cmax) of INZ-701
Systemic Clearance of INZ-701
Week 52
Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels
Week 52
Incidence of Anti-Drug Antibodies (ADAs)
Number of Treatment Emergent Adverse Events (TEAEs)

Trial Safety

Safety Progress

1 of 3

Other trials for Pseudoxanthoma Elasticum

Trial Design

1 Treatment Group

INZ-701
1 of 1
Experimental Treatment

This trial requires 9 total participants across 1 different treatment group

This trial involves a single treatment. INZ-701 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

INZ-701
Drug
The study design is a MAD 3+3 with 3 dose cohorts. The planned doses will be 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg administered via subcutaneous injection twice weekly.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 52 weeks (day 1 through safety follow-up visit)
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 52 weeks (day 1 through safety follow-up visit) for reporting.

Closest Location

Clinilabs - Eatontown, NJ

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
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
Male or female, 18 to <65 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 be on stable doses for 3 years prior to enrollment and be planning to maintain stable doses through end of study unless the investigator deems, in consultation with the sponsor, that the change will not confound interpretation of the study data
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 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.
In the opinion of the Investigator, must be willing and able to complete the Dose Evaluation Period
Agree to provide access to relevant medical records

Patient Q&A Section

What causes pseudoxanthoma elasticum?

"This is a rare disease (estimated prevalence of 1 in 1,000,000). The cause is still not known, but it is likely due to a combination of genetic, environmental, and other pathogenic alterations to tissue. PSE is one of the most important causes of blindness and vision loss in the Western world." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating pseudoxanthoma elasticum?

"This is the first study to show potential therapy combining tazarotene and isotretinoin for the treatment of PXE. Tazarotene and isotretinoin demonstrated promising effects in improving clinical, histological, and genetic parameters of PXE and provided a promising therapeutic option for future trials." - Anonymous Online Contributor

Unverified Answer

How serious can pseudoxanthoma elasticum be?

"This is the first study to demonstrate that pseudoxanthoma elasticum patients have a higher prevalence of cardiovascular disease, but no association with non-cardiovascular disease, compared with healthy control subjects." - Anonymous Online Contributor

Unverified Answer

Can pseudoxanthoma elasticum be cured?

"The present case of a patient with PE presenting with systemic symptoms warrants consideration of a differential diagnosis. A history of previous dental trauma in patients with PE or a history of dental pulp neoplasm suggests a possible dental or paracapsular or accessory radicular cyst in the area. The diagnosis of PE in this case was confirmed with the help of ultrasound examination of the subcutaneous tissues and in vivo confocal laser Doppler imaging of the oral cavity. The present situation lends further credence to the use and effectiveness of an integrated approach to the treatment of PE." - Anonymous Online Contributor

Unverified Answer

What are the signs of pseudoxanthoma elasticum?

"In a recent study, findings demonstrated that a family history of PXE and pseudoxanthoma elasticum-like skin features were associated with clinical features of PXE. In a recent study, findings also showed that pseudoxanthoma elasticum can be used as a clinical marker to identify mutations of FBXL3." - Anonymous Online Contributor

Unverified Answer

What is pseudoxanthoma elasticum?

"PXE is a rare genetic condition in the general population and is inherited in an X-linked recessive fashion. PXE is thought to be inherited by parents from the same X-chromosome. Inheritance in an X-linked recessive fashion means that both parents must be carriers of the defective gene. For this reason, the disease is inherited in an X-linked dominant fashion, wherein the affected child is likely to inherit an X-chromosome containing the defective gene from either parent. More than 60% of PXE cases appear to be sporadic and may occur at an older age; women and men are affected equally." - Anonymous Online Contributor

Unverified Answer

What are common treatments for pseudoxanthoma elasticum?

"There is no cure for PXE but treatments can increase life expectancy and reduce symptoms. Common treatments for PXE include skin care, lifestyle modifications, counselling, orthoses, and medication." - Anonymous Online Contributor

Unverified Answer

How many people get pseudoxanthoma elasticum a year in the United States?

"There are no specific records-based estimates of PXE in the US. PXE-affected individuals are at all ages and are usually asymptomatic until significant organ or systemic diseases develop due to the severity of the disease. There appears to be a higher incidence of PXE in individuals of non-Caucasian descent. The cause of PXE is hereditary. No known risk factors have been shown to increase the predisposition to PXE. In fact, family and genetic counseling are recommended for all patients with PXE. It is not clear when PXE is diagnosed in early childhood, but the first recorded case of PXE was seen in a baby girl in 1860 in France." - Anonymous Online Contributor

Unverified Answer

Has inz-701 proven to be more effective than a placebo?

"The new topical agent I-701 was found to be more effective than a placebo in slowing down the progression of PXE in this patient population." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for pseudoxanthoma elasticum?

"Clinical trials are the most effective tool for the treatment of PXE. Patients with severe disease should consider clinical trials; patients with mild disease may opt for self-monitoring." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of pseudoxanthoma elasticum?

"It is common that pseudoxanthoma elasticum presents as palpable papules and plaques. In most cases, the cause is not precisely known, but it may be caused by various systemic disorders, the immune system or genetics. The primary cause has to be identified and treated to prevent the complication of eschara." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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