Sioux Falls, SDNCT06168201Now EnrollingIRB Ready

Achondroplasia Clinical Trial in Sioux Falls, SD

Access cutting-edge achondroplasia treatment through this clinical trial at a research site in Sioux Falls. Study-provided care at no cost to qualified participants.

Sponsored by BioMarin Pharmaceutical

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Expert Care in Sioux Falls

Access achondroplasia specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related achondroplasia treatment provided free

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Check if you qualify for this achondroplasia clinical trial in Sioux Falls, SD

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Sioux Falls

    Convenient for SD 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 Sioux Falls site if eligible
  4. 4Begin participation

About This Achondroplasia Study in Sioux Falls

This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats for the primary pediatric study population: 1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. . 2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data. Data will be collected in the following format for the adult cohort: \- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment. The secondary study population will include 20 adolescent and adult participants.

Sponsor: BioMarin Pharmaceutical

Who Can Participate

Inclusion Criteria

Physician diagnosis of achondroplasia
Age at time of enrollment: Primary Pediatric Cohort: ≤13 years old Secondary Adolescent and Adult Cohort: ≥14 years old
Receiving medical care in the United States
Complete PicnicHealth's onboarding process, including signing informed consent and authorization for medical record retrieval

Exclusion Criteria

Lack of any medical records

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 Sioux Falls?

Yes, this clinical trial (NCT06168201) has an active research site in Sioux Falls, SD 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.

Achondroplasia Treatment Options in Sioux Falls, SD

If you're searching for achondroplasia treatment options in Sioux Falls, SD, this clinical trial (NCT06168201) 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 Sioux Falls research site is actively enrolling participants for this clinical trial. You'll receive care from experienced achondroplasia 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 achondroplasia clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Sioux Falls, SD