Angelman SyndromeNCT07136454
Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
A study testing Rugonersen in people with Angelman Syndrome, run by Oak Hill Bio Ltd.
Trial summary
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE), and continue to meet the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Angelman Syndrome
- What is being tested?
- Rugonersen
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to the initiation of any study-required procedures. The participant has a parent, caregiver, or legal representative (referred to as "caregiver") who is reliable and able to consent for the participant according to ICH and local regulations
- At least 18 years of age
- Willing and able to accompany the participant to clinic visits and be available to the site by phone, email, or other electronic form as needed
- Is and will likely remain sufficiently knowledgeable of the participant's condition in order to respond to queries as requested.
- Patient had previously signed informed consent for the TANGELO Optional OOE and completed a final safety follow-up visit for the TANGELO Study.
- Participant is able to comply with all requirements including blood draws.
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- Participant is able to undergo lumbar puncture (LP) and intrathecal (IT) injection under sedation or anesthesia as deemed appropriate.
- Will comply with requirements regarding contraception and is confirmed by caregiver consent.
- In the opinion and clinical judgement of the Physician, potential benefit outweighs potential risk of continuing rugonersen as an investigational therapy, based on the individual patient's medical history and program eligibility criteria, and judged by the treating physician to still being medically suitable for treatment with rugonersen.
Who cannot join
- History of clinically significant post lumbar puncture headache of moderate or severe intensity and/or blood patch.
- Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of screening or planned during treatment.
- Ascertained or presumptive hypersensitivity to the rugonersen or its excipients.
- Permitted sleep medications have not been stable for 4 weeks prior to screening and at time of enrolment.
- Permitted medications for epilepsy have not been stable for 12 weeks prior to screening and at the time of enrolment.
- Prohibited use of antiplatelet or anticoagulant therapy for 2 weeks prior to screening and at the time of enrolment.
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- Concurrent psychotropic medications have not been stable for 4 weeks prior to screening and at time of enrolment.
- Concurrent or planned concurrent participation in any clinical trial (including observational, non-drug or non-interventional).
- Previous participation in cellular therapy, gene therapy or gene editing, or any other gene expression modulating clinical trial, such as an antisense oligonucleotide (ASO) treatment other than rugonersen.
- Participation in any other research study with drug or non-drug treatments during this EAP.
- Participants who discontinued participation in the TANGELO Study (BP41674) due to safety reasons are not eligible.
Trial locations and contacts
No sites are listed yet.
Sharon Morriss contact
About this study
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE), and continue to meet the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the first dose in the EAP.
The Physician making the request for expanded access considers continued treatment via the EAP with rugonersen to be a suitable treatment option for the patient under consideration.
Read the detailed description
The goal of this EAP is to make rugonersen available to patients who have previously consented to participate in the TANGELO Optional Open-Label Extension (OOE).
Eligibility reassessment is required for patients who are entering the EAP after they have completed a final visit in the TANGELO study. The purpose of the reassessment is to ensure that the patient continues to meet the eligibility criteria. The relevant assessments to confirm eligibility (i.e., coagulation, platelets, renal function) are part of pre-dosing assessment and can be conducted prior to EAP dosing either on the day before dosing or on the dosing day.
An updated medical history for participants should be completed prior to entering the EAP.
For the EAP the dose regimen will be 120 mg administered every 16 weeks.
- Conditions
- Angelman Syndrome
- Being tested
- Rugonersen
- Medical subject headings
- Angelman Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RugonersenDrug
Rugonersen is a selective synthetic 20-mer antisense oligonucleotide (ASO).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Angelman SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Oak Hill Bio Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Early Access Care LLC
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07136454
- Sponsor's protocol ID
- OHB-EAP
- Status last verified
- Aug 2025
- First submitted
- 14 Aug 2025
- Last updated
- 22 Aug 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
- ClinicalTrials.gov ID
- NCT07136454
- Acronym / sponsor ID
- OHB-EAP
- Conditions
- Angelman Syndrome
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- Oak Hill Bio LtdIndustry (pharma, biotech, devices)
- Collaborators
- Early Access Care LLC
- First posted
- 22 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE), and continue to meet the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the first dose in the EAP.
The Physician making the request for expanded access considers continued treatment via the EAP with rugonersen to be a suitable treatment option for the patient under consideration.
Detailed description
The goal of this EAP is to make rugonersen available to patients who have previously consented to participate in the TANGELO Optional Open-Label Extension (OOE).
Eligibility reassessment is required for patients who are entering the EAP after they have completed a final visit in the TANGELO study. The purpose of the reassessment is to ensure that the patient continues to meet the eligibility criteria. The relevant assessments to confirm eligibility (i.e., coagulation, platelets, renal function) are part of pre-dosing assessment and can be conducted prior to EAP dosing either on the day before dosing or on the dosing day.
An updated medical history for participants should be completed prior to entering the EAP.
For the EAP the dose regimen will be 120 mg administered every 16 weeks.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Signed informed consent must be obtained prior to the initiation of any study-required procedures. The participant has a parent, caregiver, or legal representative (referred to as "caregiver") who is reliable and able to consent for the participant according to ICH and local regulations
- Patient had previously signed informed consent for the TANGELO Optional OOE and completed a final safety follow-up visit for the TANGELO Study.
- Participant is able to comply with all requirements including blood draws.
- Participant is able to undergo lumbar puncture (LP) and intrathecal (IT) injection under sedation or anesthesia as deemed appropriate.
- Will comply with requirements regarding contraception and is confirmed by caregiver consent.
Key exclusion criteria (11 listed)
- History of clinically significant post lumbar puncture headache of moderate or severe intensity and/or blood patch.
- Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of screening or planned during treatment.
- Ascertained or presumptive hypersensitivity to the rugonersen or its excipients.
- Permitted sleep medications have not been stable for 4 weeks prior to screening and at time of enrolment.
- Permitted medications for epilepsy have not been stable for 12 weeks prior to screening and at the time of enrolment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RugonersenRugonersen is a selective synthetic 20-mer antisense oligonucleotide (ASO). | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sharon Morriss contact
Questions about this trial
What is this clinical trial studying?
A study testing Rugonersen in people with Angelman Syndrome, run by Oak Hill Bio Ltd.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 22 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Annette Silvia (001-203-441-7938 ext. 180, rugonersen.eap@earlyaccesscare.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2025.