NCT05127226 HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
| NCT ID | NCT05127226 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Angelman Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 70 participants |
| Start Date | 2021-12-22 |
| Primary Completion | 2029-03 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 70 participants in total. It began in 2021-12-22 with a primary completion date of 2029-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Eligibility Criteria
Key Inclusion Criteria: 1. Participant has a documented and certified diagnosis of Angelman syndrome (AS) (ubiquitin-protein ligase E3A \[UBE3A\] deletion or UBE3A mutation) 2. Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s) 3. Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose. 4. Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed. Key Exclusion Criteria: 1. Has documented molecular AS confirmation of paternal uniparental disomy (UPD) or imprinting defect (ID). 2. Any clinically significant (CS) cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable for participation in, and/or unable to complete the study procedures. Has poorly controlled seizures as determined by the Investigator or has documented Status Epilepticus in the past 6 months that could pose a safety risk while on study. 3. Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid, antisense oligonucleotide \[ASOs\]). COVID-19 vaccinations are allowed. 4. Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05127226 clinical trial?
This trial is open to participants of all sexes, aged 0 Years or older, up to 50 Years, studying Angelman Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05127226 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05127226 currently recruiting?
Yes, NCT05127226 is actively recruiting participants. Contact the research team at IonisAngelmanStudy@clinicaltrialmedia.com for enrollment information.
Where is the NCT05127226 trial being conducted?
This trial is being conducted at San Diego, United States, Aurora, United States, Chicago, United States, Boston, United States and 7 additional locations.
Who is sponsoring the NCT05127226 clinical trial?
NCT05127226 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 70 participants.