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Tests treatment safety and results for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
ClinicalTrials.gov ID: NCT06934967
What this study is testing
What is LNP023?
LNP023 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for paroxysmal nocturnal hemoglobinuria (pnh).
Also referred to as Iptacopan.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 18
You may be able to join if
- 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)...
- 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...
- 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...
- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the...
You likely can't join if
- 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...
- 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.
See the full eligibility 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%...
- 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...
- 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...
- 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...
- Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- New Brunswick, New Jersey, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Brasília, Federal District, Brazil
- Natal, Rio Grande do Norte, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Santo André, São Paulo, Brazil
- São Paulo, São Paulo, Brazil
- Cali, Valle del Cauca Department, Colombia
- Berlin, Germany
- Genova, GE, Italy
- Utrecht, Netherlands
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New Brunswick, New Jersey, United States; Philadelphia, Pennsylvania, United States; Memphis, Tennessee, United States; Brasília, Federal District, Brazil; Natal, Rio Grande do Norte, Brazil; Porto Alegre, Rio Grande do Sul, Brazil and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.