Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia

Launched by ALEXION PHARMACEUTICALS, INC. · Aug 23, 2023

Keywords

ClinConnect Summary

This clinical trial, called STRENSIQ®, is studying a treatment approach for people with a condition known as hypophosphatasia, which affects bone health. The main goal of the trial is to see how well a type of medication called immunosuppressive therapy (IST) works for patients who have been using a different treatment, asfotase alfa, but are experiencing a decrease in its effectiveness due to their immune system. This trial will involve individuals aged between 730 days (about 2 years) and 6574 days (about 18 years), and it is open to all genders.

To participate in the trial, candidates must have shown some improvement from their previous treatment but have recently had a worsening of their symptoms for at least three months. They also need to have certain immune system markers present. It's important that participants, or their guardians, can give informed consent and follow specific guidelines about contraception if they could become pregnant. Throughout the study, participants will visit the clinic for check-ups, and they will be closely monitored to ensure their safety. This trial is not currently recruiting participants, so it's a good idea to stay updated if you or someone you know might be interested in taking part.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Reoccurrence or worsening of rickets for at least the past 3 months in participants who showed an initial efficacy response to asfotase alfa after at least 6 months of continuous treatment and currently receiving asfotase alfa. RSS will be used to determine severity at Baseline.
• • Presence of ADAs, with or without NAbs, irrespective of their titers.
• • Confirmation by the TMB that both the clinical evidence and immunogenicity-mediated association noted above are present.
• • Female participants of childbearing potential and male participants with partners of childbearing potential must follow protocol-specified contraception guidance as described in Section 10.5.
• • Participant, or participant's legal guardian, is capable of signing informed consent or assent as described in Section 10.1.3, which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol.
• Exclusion Criteria:
• • Known history of human immunodeficiency virus (HIV) infection (evidenced by HIV type 1 or type 2 \[HIV 1, HIV 2\] antibody) or hepatitis B or C viral infection.
• • Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to Screening.
• • Inability of the participant, or the participant's legal guardian, to provide informed consent.
• • Pregnant, breastfeeding, or intending to conceive during the course of the study.
• • Inability to travel to the clinic for specified visits during the Treatment Period caused by disease per se or logistics (does not apply to external travel restrictions).
• • The participant is at risk of reactivation or has an active significant viral infection such as hepatitis B, cytomegalovirus, herpes simplex, human polyomavirus (also known as John Cunningham \[JC\] virus), parvovirus, or Epstein Barr virus.
• • The participant is at risk of reactivation of tuberculosis or has regular contact (eg, in the household) with individuals who are being actively treated for tuberculosis.
• • The participant has had or is required to have any live vaccination within 1 month prior to enrollment.