Philadelphia, PANCT06934967Now EnrollingIRB Ready

Paroxysmal Nocturnal Hemoglobinuria (PNH) Clinical Trial in Philadelphia, PA

Access cutting-edge paroxysmal nocturnal hemoglobinuria (pnh) treatment through this phase 3 clinical trial at a research site in Philadelphia. Study-provided care at no cost to qualified participants.

Sponsored by Novartis Pharmaceuticals

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Expert Care in Philadelphia

Access paroxysmal nocturnal hemoglobinuria (pnh) specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related paroxysmal nocturnal hemoglobinuria (pnh) treatment provided free

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Check if you qualify for this paroxysmal nocturnal hemoglobinuria (pnh) clinical trial in Philadelphia, PA

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Why Participate?

  • No-Cost Study Care

  • Local to Philadelphia

    Convenient for PA 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 Philadelphia site if eligible
  4. 4Begin participation

About This Paroxysmal Nocturnal Hemoglobinuria (PNH) Study in Philadelphia

The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \< 18 years of age.

Sponsor: Novartis Pharmaceuticals

Who Can Participate

Inclusion Criteria

Male and female participants 2 to \< 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes/monocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.
Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.
Patients who are anti-C5 treatment naive: mean hemoglobin level \< 10 g/dL confirmed by central laboratory assessment during screening.
Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) \> 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.
Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.
Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.

Exclusion Criteria

History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.
Known or suspected hereditary complement deficiency at screening.
History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).
Patients with laboratory evidence of bone marrow failure (reticulocytes \< 100 x 10 to the ninth/L; platelets \< 30 × 10 to the ninth/L; neutrophils \< 0.5 × 10 to the ninth/L).
Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.
Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration. Other protocol-defined 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 Philadelphia?

Yes, this clinical trial (NCT06934967) has an active research site in Philadelphia, PA 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.

Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Options in Philadelphia, PA

If you're searching for paroxysmal nocturnal hemoglobinuria (pnh) treatment options in Philadelphia, PA, this clinical trial (NCT06934967) 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 Philadelphia research site is actively enrolling participants for this phase 3 clinical trial. You'll receive care from experienced paroxysmal nocturnal hemoglobinuria (pnh) 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 paroxysmal nocturnal hemoglobinuria (pnh) clinical trials near you to find additional studies recruiting in your area.

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