New oral drug tested in kids with rare blood disease

NCT ID NCT06934967

Recruiting now Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

Summary

This study aims to understand how a drug called iptacopan works in the bodies of children and teenagers with PNH, a rare and serious blood disease. It will test the drug's safety and how well it is tolerated in about 12 participants aged 2 to 18 years old. The goal is to see if this oral medication can help control the disease by improving blood counts and reducing the need for blood transfusions.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-••••

Locations

Cancer Institute of New Jersey
RECRUITING

New Brunswick, New Jersey, 08901, United States

Contact

Contact Phone: •••-•••-•••• Email: •••••@•••••
Childrens Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104 4399, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Contact
Novartis Investigative Site
RECRUITING

Brasília, Federal District, 70684-831, Brazil
Novartis Investigative Site
RECRUITING

Natal, Rio Grande do Norte, 59012 300, Brazil
Novartis Investigative Site
RECRUITING

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil
Novartis Investigative Site
RECRUITING

Santo André, São Paulo, 09090-401, Brazil
Novartis Investigative Site
RECRUITING

São Paulo, São Paulo, 01323001, Brazil
Novartis Investigative Site
RECRUITING

São Paulo, São Paulo, 04038-002, Brazil
Novartis Investigative Site
RECRUITING

Cali, Valle del Cauca Department, 760012, Colombia
Novartis Investigative Site
RECRUITING

Berlin, 13353, Germany
Novartis Investigative Site
RECRUITING

Genova, GE, 16147, Italy
Novartis Investigative Site
RECRUITING

Utrecht, 3584 CX, Netherlands

Conditions

Explore the condition pages connected to this study.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)