HYPOPHOSPHATASIA
Clinical trials for HYPOPHOSPHATASIA explained in plain language.
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New hope for rare bone disease: experimental drug aims to help patients walk better
Disease control OngoingThis phase 3 trial tests an experimental drug called ALXN1850 in 124 adolescents and adults with hypophosphatasia, a rare genetic bone disease. Participants receive either the drug or a placebo by injection under the skin. The main goal is to see if the drug improves walking dist…
Matched conditions: HYPOPHOSPHATASIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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New hope for kids with rare bone disease: experimental drug enters final testing
Disease control OngoingThis study tests a new drug called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The trial compares the drug to a placebo to see if it improves bone health and movement. About 30 children who have not received prior treatmen…
Matched conditions: HYPOPHOSPHATASIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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New hope for kids with brittle bones: experimental drug faces off against standard care
Disease control OngoingThis study compares a new medicine, ALXN1850, to the current standard treatment (asfotase alfa) in 43 children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. All children have been on the standard treatment for at least 6 months before joining. T…
Matched conditions: HYPOPHOSPHATASIA
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:29 UTC
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Withdrawn study aimed to counteract antibodies blocking hypophosphatasia drug
Disease control TerminatedThis study planned to test a combination of immunosuppressive drugs (methotrexate, rituximab, bortezomib, IVIg, and folic acid) in people with hypophosphatasia whose bodies had stopped responding to asfotase alfa due to antibodies. The goal was to see if suppressing the immune sy…
Matched conditions: HYPOPHOSPHATASIA
Phase: PHASE4 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: HYPOPHOSPHATASIA
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 04:00 UTC