GXR for Prader-Willi Syndrome

Phase-Based Progress Estimates
3
Effectiveness
3
Safety
Maimonides Medical Center, Brooklyn, NYPrader-Willi Syndrome+20 MoreGuanfacine Extended Release - Drug
Eligibility
6 - 35
All Sexes
What conditions do you have?
Select

Study Summary

This trial will study if a medication can reduce aggressive and self-harming behavior in people with PWS, and its safety, esp. its metabolic effects.

Eligible Conditions
  • Prader-Willi Syndrome
  • Behavioral Symptoms
  • Intellectual Disability
  • Neurobehavioral Symptoms
  • Neurologic Symptoms
  • Nervous System Disorders
  • Chromosome Disorder
  • Antihypertensive Drugs
  • Pathology
  • Self-Harm
  • Multiple Abnormalities
  • Malnutrition
  • Obesity
  • Congenital Anomalies
  • Genetic Disorders, Congenital
  • Overnutrition
  • Neurotransmitter Agents
  • Molecular Pharmacology
  • Drug Effects
  • Skin Picking Disorder
  • Aggression

Treatment Effectiveness

Effectiveness Progress

3 of 3
This is further along than 93% of similar trials

Study Objectives

2 Primary · 6 Secondary · Reporting Duration: 16 Weeks

16 Weeks
A change in Aberrant Behavior Checklist from baseline to week 16
A change in Modified Overt Aggression Scale from baseline to week 16
A change in Self-Injury Trauma Scale from baseline to week 16
Aberrant Behavior Checklist
Clinical Global Impression- Improvement
Clinical Global Impression- Improvement change from baseline to week 16
Modified Overt Aggression Scale
Self-Injury Trauma Scale

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Guanfacine Extended Release
31%Sedation
19%Fatigue
6%Dizziness
6%Stomachache
This histogram enumerates side effects from a completed 2011 Phase 4 trial (NCT01069523) in the Guanfacine Extended Release ARM group. Side effects include: Sedation with 31%, Fatigue with 19%, Dizziness with 6%, Stomachache with 6%.

Trial Design

2 Treatment Groups

GXR
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

33 Total Participants · 2 Treatment Groups

Primary Treatment: GXR · Has Placebo Group · Phase 4

GXR
Drug
Experimental Group · 1 Intervention: Guanfacine Extended Release · Intervention Types: Drug
Placebo
Other
ShamComparator Group · 1 Intervention: Placebo · Intervention Types: Other
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Guanfacine Extended Release
2010
Completed Phase 4
~50

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 16 weeks

Who is running the clinical trial?

Maimonides Medical CenterLead Sponsor
64 Previous Clinical Trials
14,332 Total Patients Enrolled
Deepan Singh, MDPrincipal InvestigatorMaimonides Medical Center
2 Previous Clinical Trials
20 Total Patients Enrolled
1 Trials studying Prader-Willi Syndrome

Eligibility Criteria

Age 6 - 35 · All Participants · 2 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have been rated as moderately or severely affected on the Clinical Global Impression- Severity Scale.
References

Frequently Asked Questions

Are juveniles of a certain age qualified to participate in this experiment?

"This research is open to applicants aged between 6 and 35 years old." - Anonymous Online Contributor

Unverified Answer

Who has the opportunity to take part in this examination?

"To qualify for this medical trial, applicants must have a neurodegenerative disease and be between 6-35 years old. 33 individuals are accepted in total." - Anonymous Online Contributor

Unverified Answer

Has GXR attained regulatory clearance from the FDA?

"Our assessment at Power rates the safety of GXR as a 3, since this is an approved Phase 4 trial which has already been deemed safe for general use." - Anonymous Online Contributor

Unverified Answer

What is the overall size of this research project's participant pool?

"Affirmative. Clinicaltrials.gov showcases that this medical trial is currently looking for participants; the initial posting was on December 17th 2020 and there has since been a recent update on December 12th 2022. This study requires recruitment of 33 individuals from 1 location." - Anonymous Online Contributor

Unverified Answer

Are there any vacancies for participation in this trial?

"Affirmative, clinicaltrials.gov data indicates that this medical trial is presently in search of participants. Begun on December 17th 2020 and most recently updated on the 12th of December 2022, 33 patients are being invited to join from a single facility." - 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.