Chicago, ILNCT06302439Now EnrollingIRB Ready

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency Clinical Trial in Chicago, IL

Access cutting-edge ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency treatment through this clinical trial at a research site in Chicago. Study-provided care at no cost to qualified participants.

Sponsored by Inozyme Pharma

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Expert Care in Chicago

Access ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency treatment provided free

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Check if you qualify for this ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency clinical trial in Chicago, IL

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Why Participate?

  • No-Cost Study Care

  • Local to Chicago

    Convenient for IL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Chicago site if eligible
  4. 4Begin participation

About This Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency Study in Chicago

The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the infantile-onset form of adenosine triphosphate (ATP) binding cassette transporter protein subfamily C member 6 (ABCC6) Deficiency longitudinally. The registry will prospectively gather information about the genetic, biochemical, physiological, anatomic, radiographic, and functional manifestations (including patient reported outcomes \[PROs\]) of each disease during routine, standard-of-care visits, with the aim of developing a comprehensive understanding of the burden of illness and progressive nature of the disease.

Sponsor: Inozyme Pharma

Who Can Participate

Inclusion Criteria

Individuals eligible to participate must meet all the following inclusion criteria:
Must provide written or electronic consent after the nature of the registry has been explained, and prior to any research-related procedures, per International Council for Harmonisation (ICH) Good Clinical Practice (GCP)
Agree to provide access to relevant medical records
One of the following genetic or clinical criteria
A confirmed prenatal or postnatal molecular genetic diagnosis of ENPP1 Deficiency with biallelic mutations (ie, homozygous or compound heterozygous) performed by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA) certified laboratory or regional equivalent OR
Monoallelic ENPP1 mutation confirmed by a certified CAP/CLIA laboratory or regional equivalent and any of the following clinical symptoms: i. ≥ 1 traumatic vertebral fracture ii. ≥ 2 fractures as an adult (eg, long-bones, digits, vertebrae) iii. Low bone mineral density (dual-energy X-ray absorptiometry \[DXA\] Z-score \<1.5) and \<55 years of age iv. Bone or joint pain interfering with movement or daily activities v. History of myocardial infarction (MI), unstable angina, transient ischemic attack (TIA) or low cardiac output before the age of 40 yrs. vi. History of rickets or bone deformity vii. Diagnosis of ossification of the posterior longitudinal ligament (OPLL) viii. Other clinical symptoms, with approval by Inozyme OR c. A confirmed prenatal or postnatal molecular genetic diagnosis of ABCC6 Deficiency with biallelic mutations confirmed by a certified CAP/CLIA laboratory or regional equivalent, and \<18 years of age

Exclusion Criteria

Individuals who meet the following exclusion criteria will not be eligible to participate:
Participant or their legally designated representative does not have the cognitive capacity to provide informed consent
Patients who are currently participating in an INZ-701 interventional clinical study, with the exception of expanded access programs and long-term safety follow-up studies
Participants in interventional studies may be approached for inclusion in the registry once their involvement in the treatment period of the clinical study has been completed

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Chicago?

Yes, this clinical trial (NCT06302439) has an active research site in Chicago, IL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency Treatment Options in Chicago, IL

If you're searching for ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency treatment options in Chicago, IL, this clinical trial (NCT06302439) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Chicago research site is actively enrolling participants for this clinical trial. You'll receive care from experienced ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency clinical trials near you to find additional studies recruiting in your area.

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