All > Angelman Syndrome

GTX-102 for Angelman Syndrome

Queensland Children's Hospital, Queensland, Australia
Angelman SyndromeGTX-102 - Drug
Eligibility
4 - 17
All Sexes

Study Summary

This trial is testing if a new medication is safe and tolerated when given to people with AS.

Eligible Conditions
  • Angelman Syndrome

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 1 & 2

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1
STRO-002
1
STRO-002
1
LY3410738

Study Objectives

4 Primary · 1 Secondary · Reporting Duration: Day 2, Day 3, Day 4, Day 30, Day 58, Day 86, Day 100/128

Day 128
Exploratory: Adaptive behaviors
Exploratory: Change in communication
Exploratory: Change in development
Day 128
Exploratory: Change in motor function by wearable device
Exploratory: Change in sleep
Exploratory: Global Clinical Status
Exploratory: Seizure frequency
Day 100
Pharmacokinetics of GTX-102
Day 86
Pharmacodynamics of GTX-102
Up to Day 128
Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs (SAEs), TEAEs Leading to Discontinuation and Severity of TEAEs
Safety: incidence of adverse events
Safety: incidence of serious adverse events
Safety: severity of adverse events

Trial Safety

Phase-Based Safety

1 of 3

Similar Trials

1
STRO-002
1
STRO-002
1
LY3410738

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

7 Treatment Groups

GTX-102 Cohort A
1 of 7
GTX-102 Cohort B
1 of 7
GTX-102 US Cohort
1 of 7
GTX-102 Cohort 7
1 of 7
GTX-102 Cohort 6
1 of 7
GTX-102 Cohort 5
1 of 7
GTX-102 Cohort 4
1 of 7

Experimental Treatment

83 Total Participants · 7 Treatment Groups

Primary Treatment: GTX-102 · No Placebo Group · Phase 1 & 2

GTX-102 Cohort A
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 Cohort B
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 US Cohort
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 Cohort 7
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 Cohort 6
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 Cohort 5
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug
GTX-102 Cohort 4
Drug
Experimental Group · 1 Intervention: GTX-102 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: day 2, day 3, day 4, day 30, day 58, day 86, day 100/128

Who is running the clinical trial?

GeneTX Biotherapeutics, LLCLead Sponsor
Ultragenyx Pharmaceutical IncLead Sponsor
86 Previous Clinical Trials
179,039 Total Patients Enrolled
1 Trials studying Angelman Syndrome
50 Patients Enrolled for Angelman Syndrome
Scott Stromatt, MDStudy DirectorGeneTX Biotherapeutics, LLC
4 Previous Clinical Trials
217 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,670 Previous Clinical Trials
7,959,129 Total Patients Enrolled
1 Trials studying Angelman Syndrome
20 Patients Enrolled for Angelman Syndrome

Eligibility Criteria

Age 4 - 17 · All Participants · 9 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Your parent(s) or legal guardian(s) gave permission for you to participate in the study by signing a form.
You have a specific genetic confirmation of Angelman Syndrome caused by a complete deletion of the UBE3A gene from a specific area on chromosome 15.
Your seizures have been under control for at least one month, and you have not changed your seizure medication unless it was due to changes in your weight.
Your kidneys are working normally, with no signs of kidney damage.
Your liver is functioning normally, as shown by blood tests for bilirubin, AST, ALT, and alkaline phosphatase.
You are between the ages of 4 and 17 years old (or between 4 and 7 years old if you are in the US) when you are screened for the trial.
Your platelet count and blood clotting factors are within a normal range.
You are able and willing to follow the study's schedule, take medication as directed, undergo required tests, follow study restrictions, and complete all study procedures.
You can handle the anesthesia needed for the lumbar puncture procedure.
References

Frequently Asked Questions

Is this research accessible to those aged 65 and above?

"This research project seeks to enroll participants between the ages of 4 and 17." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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