Chapel Hill, NCNCT06997588Now EnrollingIRB Ready

PIK3CA-related Overgrowth Spectrum (PROS) Clinical Trial in Chapel Hill, NC

Access cutting-edge pik3ca-related overgrowth spectrum (pros) treatment through this clinical trial at a research site in Chapel Hill. Study-provided care at no cost to qualified participants.

Sponsored by Novartis Pharmaceuticals

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Expert Care in Chapel Hill

Access pik3ca-related overgrowth spectrum (pros) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related pik3ca-related overgrowth spectrum (pros) treatment provided free

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Check if you qualify for this pik3ca-related overgrowth spectrum (pros) clinical trial in Chapel Hill, NC

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Why Participate?

  • No-Cost Study Care

  • Local to Chapel Hill

    Convenient for NC 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 Chapel Hill site if eligible
  4. 4Begin participation

About This PIK3CA-related Overgrowth Spectrum (PROS) Study in Chapel Hill

This study is designed to demonstrate the efficacy and assess safety and tolerability of oral daily alpelisib in participants with PIK3CA-related overgrowth spectrum (PROS).

Sponsor: Novartis Pharmaceuticals

Who Can Participate

Inclusion Criteria

Male or female participants aged ≥2 years at the time of informed consent/assent.
Participants with diagnosis of PROS (according to Clinical Diagnostic Criteria for PROS proposed by Keppler Noreuil et al 2014) with symptomatic AND progressive overgrowth, who have syndromic disease or isolated features (with the exception of isolated macrodactyly, macrocephaly or epidermal nevus) at the time of informed consent/assent.
Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local laboratories using a DNA-based test AND available archival tissue (if archival tissue sample is not available, a fresh biopsy should be performed, if it is not clinically contraindicated) at the time of informed consent/assent.
Karnofsky (in participants \>16 years of age at study entry) or Lansky (≤16 years of age at study entry) performance status index ≥50.
PGI-S score of mild, moderate, severe, or very severe at screening.
Adequate bone marrow and organ function.
Presence of at least 1 PROS-related measurable lesion (longest diameter ≥2 cm) confirmed by BIRC assessment and associated with complaints, clinical symptoms or functional limitations affecting the participant's everyday life. Key

Exclusion Criteria

Participant with only isolated macrodactyly, epidermal nevus/nevi and macroencephaly (the only clinical feature or a combination of any of three of them), in absence of other PROS-related lesions at the time of informed consent/assent.
Previous treatment with alpelisib and/or any other phosphatidylinositol 3-kinase (PI3K) inhibitor(s) (except treatment attempt, defined as the attempt to treat PROS with any of PI3K inhibitors, with treatment duration less than 2 weeks and stopped at least 4 weeks prior to the first dose of study medication with alpelisib).
Debulking or other major surgery performed within 3 months at the time of informed consent/assent.
Radiation exposure for PROS treatment purpose within 12 months prior to informed consent/assent.
Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per CTCAE v4.03) within 30 days before informed consent/assent, and/or sclerotherapy/embolization for vascular complications performed within 6 weeks before informed consent/assent.
Clinically meaningful bleeding from PROS-related lesion (Grade 2 and more as per CTCAE v4.03) within 30 days before study treatment initiation.
Participants with clinically significant worsening of PROS-related laboratory abnormalities, physical signs and symptoms (such as, but not limited to increase of D-dimers, worsening of underlying pain, newly occurring swelling or redness) indicating an uncontrolled condition during the screening phase. Other inclusion/exclusion criteria may apply

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 Chapel Hill?

Yes, this clinical trial (NCT06997588) has an active research site in Chapel Hill, NC 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.

PIK3CA-related Overgrowth Spectrum (PROS) Treatment Options in Chapel Hill, NC

If you're searching for pik3ca-related overgrowth spectrum (pros) treatment options in Chapel Hill, NC, this clinical trial (NCT06997588) 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 Chapel Hill research site is actively enrolling participants for this clinical trial. You'll receive care from experienced pik3ca-related overgrowth spectrum (pros) 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 pik3ca-related overgrowth spectrum (pros) clinical trials near you to find additional studies recruiting in your area.

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