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New treatment option for Hereditary Angioedema (HAE)
Official title Donidalorsen Treatment in Children With Hereditary Angioedema
ClinicalTrials.gov ID: NCT07298447
What this study is testing
What is Donidalorsen?
Donidalorsen is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for hereditary angioedema (hae).
Also referred to as ISIS 721744, IONIS-PKK-LRx.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 11
You may be able to join if
- Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
- Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
- Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
- Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
- Diagnostic testing results that confirm HAE-1/HAE-2: C1-inhibitor (C1-INH) functional level \<50% normal level AND complement factor C4 level below...
You likely can't join if
- Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a...
- Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of...
- Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III)...
The study team makes the final eligibility decision.
Where it's taking place
- Santa Monica, California, United States
- Miami, Florida, United States
- St Louis, Missouri, United States
- Cincinnati, Ohio, United States
- Hershey, Pennsylvania, United States
- Milan, Italy
- Padova, Italy
- San Donato Milanese, Italy
- Warsaw, Poland
- Barcelona, Spain
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, 2 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Santa Monica, California, United States; Miami, Florida, United States; St Louis, Missouri, United States; Cincinnati, Ohio, United States; Hershey, Pennsylvania, United States; Milan, Italy and 4 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.