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GTX-102 for Angelman Syndrome (KIK-AS Trial)
Phase 1 & 2
Waitlist Available
Research Sponsored by GeneTX Biotherapeutics, LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Documented genetic confirmation of full maternal UBE3A gene deletion causing AS in the region of 15q11.2-q13 including class I, II or III
Normal renal function with serum creatinine and spot urine protein ≤ 1.4 x the upper limit of normal (ULN)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to day 337
Awards & highlights
KIK-AS Trial Summary
This trial is testing if a new medication is safe and tolerated when given to people with AS.
Who is the study for?
This trial is for children with Angelman Syndrome confirmed by genetic testing, who have stable seizure control and can walk independently or with help. They must not be wheelchair-bound, have normal blood clotting and organ function tests, agree to follow study procedures including lumbar puncture (spinal tap), and use contraception if of childbearing age.Check my eligibility
What is being tested?
The trial studies the safety of multiple doses of GTX-102 given through spinal injections in kids with Angelman Syndrome. It aims to see how well participants tolerate this treatment over time.See study design
What are the potential side effects?
While specific side effects are not listed here, common risks may include discomfort at the injection site, potential allergic reactions to GTX-102 or anesthesia used during lumbar punctures, and general risks associated with intrathecal injections such as headache or back pain.
KIK-AS Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My AS is caused by a complete UBE3A gene deletion from my mother.
Select...
My kidney function is normal, with acceptable creatinine and protein levels.
Select...
I can walk on my own or with help from a device.
KIK-AS Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to day 337
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to day 337
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), Adverse Events of Special Interest (AESIs), AEs Leading to Discontinuation and Severity of AEs
Secondary outcome measures
Pharmacokinetics of GTX-102 over time
Other outcome measures
Exploratory: Adaptive behaviors
Exploratory: Change in communication
Exploratory: Change in development
+5 moreKIK-AS Trial Design
13Treatment groups
Experimental Treatment
Group I: GTX-102 Expanded Enrollment Cohort DExperimental Treatment1 Intervention
Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in U.S participants ≥ 8 to 17 years of age)
Group II: GTX-102 Expanded Enrollment Cohort CExperimental Treatment1 Intervention
Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in U.S participants 4 to <8 years of age)
Group III: GTX-102 Expanded Enrollment Cohort BExperimental Treatment1 Intervention
Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age)
Group IV: GTX-102 Expanded Enrollment Cohort AExperimental Treatment1 Intervention
Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in Ex-U.S participants 4 to <8 years of age)
Group V: GTX-102 Cohort USExperimental Treatment1 Intervention
2 mg for 4 monthly doses followed by a quarterly maintenance regimen
Group VI: GTX-102 Cohort EExperimental Treatment1 Intervention
Sponsor selected dose followed by slow intra-patient dose escalation and then a maintenance phase (in participants that transition from GTX-102 US Cohort only)
Group VII: GTX-102 Cohort 7Experimental Treatment1 Intervention
10 mg starting dose followed by slow intra-patient dose escalation up to 12 mg and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age)
Group VIII: GTX-102 Cohort 6Experimental Treatment1 Intervention
7.5 mg starting dose followed by slow intra-patient dose escalation up to 10 mg and then a maintenance phase (in Ex-U.S participants 4 to <8 years of age)
Group IX: GTX-102 Cohort 5Experimental Treatment1 Intervention
5 mg starting dose followed by slow intra-patient dose escalation up to 7.5 mg and then a maintenance phase (in Ex-U.S participants ≥ 8 to 17 years of age)
Group X: GTX-102 Cohort 4Experimental Treatment1 Intervention
3.3 mg starting dose followed by slow intra-patient dose escalation up to 5 mg and then a maintenance phase (in Ex-U.S participants 4 to <8 years of age)
Group XI: GTX-102 Cohort 3Experimental Treatment1 Intervention
20 mg starting dose followed by intra-patient dose escalation up to 55 mg and then a maintenance phase (in U.S participants 4 to <17 years of age)
Group XII: GTX-102 Cohort 2Experimental Treatment1 Intervention
10 mg starting dose followed by intra-patient dose escalation up to 36 mg and then a maintenance phase (in U.S participants 4 to <17 years of age)
Group XIII: GTX-102 Cohort 1Experimental Treatment1 Intervention
3.3 mg starting dose followed by intra-patient dose escalation up to 36 mg and then a maintenance phase (in U.S participants 4 to <17 years of age)
Find a Location
Who is running the clinical trial?
GeneTX Biotherapeutics, LLCLead Sponsor
Ultragenyx Pharmaceutical IncLead Sponsor
89 Previous Clinical Trials
179,000 Total Patients Enrolled
1 Trials studying Angelman Syndrome
11 Patients Enrolled for Angelman Syndrome
Scott Stromatt, MDStudy DirectorGeneTX Biotherapeutics, LLC
4 Previous Clinical Trials
217 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My liver tests are within normal limits, or slightly higher due to medication for epilepsy or Gilbert syndrome.You have been diagnosed with hepatitis B, hepatitis C, or HIV.I can walk on my own or with help from a device.You have had a serious allergic reaction to oligonucleotide or have shown signs of a severe allergic reaction like hives or rash.My seizures are under control and my medication hasn't changed in the last month.I am not pregnant or breastfeeding and will not become pregnant for 3 months after treatment.I haven't taken any experimental drugs recently, except for GTX 102.I have a bleeding or platelet disorder.I do not have any major health issues that would make it unsafe for me to join the study.My AS is caused by a complete UBE3A gene deletion from my mother.I am not taking medications that increase bleeding risk.My kidney function is normal, with acceptable creatinine and protein levels.Your blood clotting and platelet levels are within the normal range.I have previously undergone gene therapy.I am a male and agree to avoid sex or use contraception for 3 months after my last GTX-102 dose.I haven't changed my medications or diet to manage my AS symptoms in the last month.I currently have an active infection.I can safely undergo anesthesia for a lumbar puncture.I have a condition that could make a spinal tap risky or difficult.I need a breathing tube for anesthesia due to a medical condition.
Research Study Groups:
This trial has the following groups:- Group 1: GTX-102 Expanded Enrollment Cohort A
- Group 2: GTX-102 Cohort 2
- Group 3: GTX-102 Cohort 1
- Group 4: GTX-102 Cohort 4
- Group 5: GTX-102 Cohort 3
- Group 6: GTX-102 Cohort 5
- Group 7: GTX-102 Cohort 6
- Group 8: GTX-102 Cohort 7
- Group 9: GTX-102 Cohort US
- Group 10: GTX-102 Expanded Enrollment Cohort B
- Group 11: GTX-102 Expanded Enrollment Cohort C
- Group 12: GTX-102 Expanded Enrollment Cohort D
- Group 13: GTX-102 Cohort E
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is this research accessible to those aged 65 and above?
"This research project seeks to enroll participants between the ages of 4 and 17."
Answered by AI
Is it possible to register for this experiment?
"Patients aged between 4 and 17 who have been diagnosed with Angelman syndrome are eligible to partake in this study, which aims to recruit a total of 83 participants."
Answered by AI
What is the total participant count for this clinical trial?
"Affirmative, according to the data posted on clinicaltrials.gov this investigation is actively looking for participants. This project was originally initiated on February 24th 2020 and has been updated as recently as November 2nd 2022. 83 patients need to be enrolled from 4 distinct locations."
Answered by AI
Are there multiple institutions in this state executing the experiment?
"Several medical facilities are now accepting patients, such as Children's Hospital of Western Ontario in London, McGill University Health Centre in Montréal, and Children's Hospital of Eastern Ontario in Ottawa. Several other locations have also opened enrollment for this trial."
Answered by AI
Are there still vacancies in this investigation for participants?
"Affirmative. Data hosted on clinicaltrials.gov reveals this medical experiment, which went live on February 24th 2020, is still in need of participants. 83 individuals should be recruited from 4 different sites for the trial to take place."
Answered by AI
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