Netherton syndrome

This early-stage trial tests a cream called ATR12-351 for Netherton syndrome, a rare genetic skin condition. The cream contains live bacteria that produce a protein missing in people with the disease. Twelve adults will apply the cream to one side of their body and a placebo to t…

This study tests a new intravenous immunoglobulin (IGIV) called PlasmaCap IG in adults and children aged 2 to 70 with primary immune deficiency diseases (PIDD). Participants receive regular infusions of the therapy for 12 months. The goal is to see if it prevents serious bacteria…

This Phase 3 study tested an ointment called TMB-001 in 153 people aged 6 and older with X-linked or ARCI ichthyosis, conditions that cause dry, scaly, and cracked skin. Participants applied the ointment or a placebo daily for 12 weeks, with some continuing for maintenance. The g…

This study checked the long-term safety of TAK-771 in 15 Japanese patients with primary immunodeficiency disease (PID). Participants received TAK-771 injections for up to 3 years. The main goal was to monitor side effects and immune reactions. The study is now complete.

This study tested a new intravenous immunoglobulin (IVIg) treatment called Kedrion IVIg 10% in 47 people with primary immunodeficiency, a condition where the immune system cannot make enough antibodies. The goal was to see if regular infusions of this antibody therapy could preve…

This Phase 2 trial tested whether Humira (adalimumab), a drug used for arthritis, can help people with Netherton syndrome, a rare genetic skin condition that causes severe inflammation, itching, and pain. Eleven patients received six injections over three months, followed by thre…

This study tested a new medicine called TAK-881, a concentrated antibody solution given under the skin, in 65 people with primary immunodeficiency diseases (PIDD). The goal was to see how well the body absorbs it and if it is safe compared to an existing treatment (HYQVIA). Parti…

This early study tested a cream called SXR1096 on 9 people with Netherton syndrome, a rare inflammatory skin disease. The cream aims to block certain enzymes that cause skin problems. Participants applied the cream and a placebo to different skin areas for one month to check safe…

This study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…

This study tested a combination of two immune-targeting drugs, secukinumab and dupilumab, in 15 children with severe Netherton syndrome, a rare genetic skin disorder. The goal was to see if the treatment could improve survival and control the disease. The children received weight…

This study analyzed DNA from 9 patients with primary immune deficiencies whose genetic cause was unknown. Researchers used whole exome or genome sequencing to look for new mutations that might explain their condition. The goal was to identify new inborn errors of immunity, which …

This study looked for hidden immune system problems in adults who get frequent or severe bacterial infections, like pneumonia or meningitis. Researchers screened 120 adults aged 18-65 to see how many had an undiagnosed primary immunodeficiency. The goal was to find out if these c…

This study looked at 99 women with primary immune deficiency to learn more about their pregnancies and birth outcomes. Researchers tracked infections during pregnancy and after birth, as well as how well contraception worked. The goal is to help doctors give better advice to wome…

This study looked at Netherton syndrome, a rare genetic skin condition, to better understand how it affects the body and immune system. Researchers studied 18 people with the disease, measuring immune markers in the blood and tracking skin changes. The goal was to find new target…

This study looked at whether epidural or spinal anesthesia during childbirth leads to more infections in women with primary immunodeficiencies (weak immune systems). Researchers tracked 30 women from a national registry for 28 days after delivery to see if they developed serious …