Not recruitingRare disease

Study on the Effects and Safety of INZ-701 for Children with ENPP1 Deficiency

Verified siteInvestigational

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition known as Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency. This condition affects the body's ability to manage certain minerals, which can lead to problems with bone development. The study will use a treatment called INZ-701, which is a special type of protein designed to help improve the symptoms of this deficiency. INZ-701 is given as an injection under the skin.

The purpose of the study is to see if INZ-701 can increase the levels of a substance called PPi in the blood and improve bone health in children with ENPP1 Deficiency. The study will last for about a year, during which participants will receive regular injections of INZ-701. Throughout the study, doctors will monitor the participants' health and check for any changes in their bone condition and overall well-being.

Participants in the study will be children who have been diagnosed with ENPP1 Deficiency. The study will involve regular visits to the clinic for check-ups and assessments. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of INZ-701. The study aims to provide valuable information on the safety and effectiveness of INZ-701 in treating this rare condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the participant must have a confirmed diagnosis of ENPP1 Deficiency with specific genetic mutations.
The participant must be between 1 and 13 years of age and have certain skeletal characteristics.
Initial assessments include a plasma PPi concentration test and a vitamin D level check.
Step 2
Treatment initiation
The participant will begin treatment with INZ-701, a medication administered as a subcutaneous injection.
The medication is provided in the form of a lyophilized powder that is prepared for injection.
Step 3
Treatment duration
The treatment with INZ-701 will continue for a period of 52 weeks.
During this time, the participant will receive regular injections as per the study protocol.
Step 4
Monitoring and assessments
Throughout the study, the participant's plasma PPi concentration and skeletal condition will be monitored.
Regular assessments will be conducted to evaluate changes in growth and skeletal abnormalities.
Step 5
Study completion
At the end of the 52-week period, final assessments will be conducted to measure the outcomes of the treatment.
The study aims to determine the efficacy and safety of INZ-701 in improving the participant's condition.

Who can join the trial?

12 criteria

The caregiver must provide written or electronic consent after understanding the study details.
Sexually active males must agree to use condoms from the first dose of the study drug, **INZ-701**, until 30 days after the last dose.
Women of childbearing potential (WOCBP) and partners of fertile males who are WOCBP must use a highly effective form of birth control from at least 1 month before the first dose of **INZ-701** until 30 days after the last dose.
The study participant must be able to complete all parts of the study, as judged by the study doctor.
The participant must agree to take part in the study according to local rules.
The participant must have a confirmed genetic diagnosis of **ENPP1 Deficiency** with specific genetic mutations, verified by a certified laboratory.
The participant must be between 1 and 12 years old at the start of the study.
The participant must have open growth plates in the thigh bone (distal femur) and shin bone (proximal tibia) in both legs.
The participant's blood level of **PPi** (a specific chemical) must be less than 1400 nM at the screening.
The participant's level of **25-hydroxyvitamin D** must be at least 12 ng/mL at the screening.
The participant must show skeletal abnormalities on an X-ray with a score of 2 or higher.
Women of childbearing potential must have a negative pregnancy test at the screening and must not be breastfeeding.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Hopital Beaujon
Clichy, France
Verified
Hospital Sant Joan De Deu Barcelona
Esplugues De Llobregat, Spain

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Investigated drugs

INZ-701 is a medication being studied for its potential to help children with a condition called ENPP1 Deficiency. This condition affects the levels of a substance in the body called PPi, which is important for bone health. The trial is looking at whether INZ-701 can increase PPi levels and improve bone problems in these children.

Recombinant Human Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Fused To The Fc Fragment Of Igg1

What is already known about the treatment

INZ-701 – This medication is administered through injection and is currently being studied in clinical trials for its effectiveness and safety in treating ENPP1 Deficiency, a rare genetic disorder. The main therapeutic goal of INZ-701 is to increase levels of inorganic pyrophosphate (PPi) and improve skeletal abnormalities. At the molecular level, INZ-701 works by targeting and modulating the activity of ectonucleotide pyrophosphatase/phosphodiesterase 1, which plays a role in bone mineralization and vascular calcification. It is classified pharmacologically as an enzyme modulator.

Investigated diseases

ENPP1 Deficiency – ENPP1 Deficiency is a rare genetic disorder that affects the body's ability to regulate certain minerals, leading to abnormal calcification in various tissues. This condition is caused by mutations in the ENPP1 gene, which is responsible for producing an enzyme that helps control the levels of pyrophosphate, a molecule that prevents calcium deposits. As a result, individuals with this deficiency may experience issues with bone development and mineralization, leading to skeletal abnormalities. Over time, these abnormalities can affect growth and physical development. The progression of the disease can vary, with some individuals experiencing more severe symptoms than others.