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STRENSIQ® Immunogenicity Study
EU CTIS ID: 2022-502793-17-00
What this study is testing
To Evaluate the effect of Immunosuppressive therapies (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE)
- Therapeutic use (Phase IV)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Participants must be ≥ 2 years of age and < 18 years with open epiphyseal growth plates at the time of signing the informed assent/consent by the participant or their legal guardian.
- 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 Treatment Monitoring Board (TMB) that both the clinical evidence and immunogenicity-mediated association noted above are present.
- Male or female
- Female participants of childbearing potential and male participants with partners of childbearing potential must follow protocol-specified contraception guidance
You likely can't join if
- Non-immune-mediated LoE. Potential causes of non-immune-mediated LoE could include poor treatment adherence, inappropriate injection technique, vitamin D deficiency (vitamin D level should be > 20 ng/mL. If < 20 ng/mL at Screening, rescreening is allowed after vitamin D supplementation), undernutrition, or concomitant diseases, and should be ruled out when assessing participants for a suspected LoE of asfotase alfa.
- Female participants who have a positive pregnancy test at Screening or Day 1.
- 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.
See the full eligibility criteria
Who can join
- Participants must be ≥ 2 years of age and < 18 years with open epiphyseal growth plates at the time of signing the informed assent/consent by the participant or their legal guardian.
- 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 Treatment Monitoring Board (TMB) that both the clinical evidence and immunogenicity-mediated association noted above are present.
- Male or female
- Female participants of childbearing potential and male participants with partners of childbearing potential must follow protocol-specified contraception guidance
- Participant, or participant’s legal guardian, is capable of signing informed consent or assent, which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol.
What rules you out
- Non-immune-mediated LoE. Potential causes of non-immune-mediated LoE could include poor treatment adherence, inappropriate injection technique, vitamin D deficiency (vitamin D level should be > 20 ng/mL. If < 20 ng/mL at Screening, rescreening is allowed after vitamin D supplementation), undernutrition, or concomitant diseases, and should be ruled out when assessing participants for a suspected LoE of asfotase alfa.
- Female participants who have a positive pregnancy test at Screening or Day 1.
- 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.
- Any medical condition (eg, cardiac, pulmonary, renal, hepatic, hematologic, oncologic,or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study (such as any interference with rituximab, methotrexate, and bortezomib), pose any added risk to the participant, or confound the assessment of the participant.
- 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.
- History of hypersensitivity to any ingredient contained in any of the study interventions.
- Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to Screening.
- Evidence of severe hepatic or renal impairment or any other medical conditions that are contraindications for use of any of the components of the IST regimens according to the Reference Safety Information (RSI) or local approved product labeling.
- History of malignancy within 5 years of Screening that has been treated with no evidence of recurrence.
- Any concomitant medications contraindicated for rituximab, methotrexate, and bortezomib (including, but not limited to, those listed in the RSI for each IST).
- Participation in another investigational drug or investigational device study within 30 days before the first dose administered in this study. Participants currently enrolled in the HPP Global Registry and/or HPP Registry Substudy should be discontinued prior to the Baseline of this study
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. 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 EU CTIS; the study team decides eligibility.