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NNZ-2591 for Pitt Hopkins Syndrome (PTHS-001 Trial)
PTHS-001 Trial Summary
This trial is testing a new drug to see if it is safe and effective in children and adolescents with Pitt Hopkins Syndrome.
PTHS-001 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowPTHS-001 Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.PTHS-001 Trial Design
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Who is running the clinical trial?
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- I am currently experiencing major or long-term depression.I am scheduled for surgery during the study period.I can swallow liquid medication.My body weight is less than 12kg.Your heart's electrical activity is abnormal.I am between 3 and 17 years old.You are pregnant.You have important abnormal results in your safety tests and vital signs when you were checked before starting the study.You weigh at least 12 kilograms at the time of screening.Any other important discovery on the heart test done at the beginning of the study.I tested positive for COVID-19 and was hospitalized for it in the last year.You have a score of 4 or higher on the Clinical Global Impression-Severity (CGI-S) test during the screening visit.You haven't been losing skills or abilities you already had in the last 3 months.You are currently losing abilities or skills.I do not have serious thyroid issues or diabetes needing insulin that is not well-managed.You have a score of 4 or higher on the Clinical Global Impression-Severity (CGI-S) scale during the screening visit.I have been diagnosed with PTHS and have a confirmed genetic cause.I have a serious kidney condition.My seizures are not under control.I do not have any major heart, liver, stomach, lung, hormone, or organ problems.I have significant vision or hearing loss that hasn't been corrected.You are allergic to strawberries.I have had a stroke, brain injury, or similar brain condition.I have had or currently have catatonia.My mental health medication has been stable for the last 2 weeks.I have or had cancer before.I can swallow liquid medication.I am between 3 and 17 years old.I have been diagnosed with PTHS and have a genetic test confirming it.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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.
- Group 1: NNZ-2591
Frequently Asked Questions
Has any other research been conducted using NNZ-2591?
"Sydney Children's Hospital was the site of NNZ-2591's initial clinical trial, which concluded in 2022. Currently, there are 3 separate active studies occurring throughout San Francisco, California."
Has NNZ-2591 been given the green light by the FDA?
"While no efficacy data exists on NNZ-2591, there is enough safety information present to give this drug a score of 2."
What criteria must patients meet in order to participate in this experiment?
"This medical research is currently open to 20 minors, aged between 3 and 17 years old, who are diagnosed with PTHS. To qualify for this trial the patient must have a documented genetic cause of their disorder; weigh at least 12kgs; be assessed as having moderate or greater symptoms severity according to CGI-S scores; not have lost any developmental skills in the past three months; be able to swallow liquid medication provided by researchers; and lastly caregivers need sufficient English language abilities."
How many individuals is the research team recruiting for this clinical investigation?
"To meet the requirements for this clinical study, 20 qualified participants are required. These individuals can register to take part at either University of California in San Francisco or University of Alabama in Birmingham."
Is this research endeavor actively seeking elderly participants?
"As delineated in the study requirements, 3 is the minimum age and 17 is the maximum age of enrolment."
Is this a precedent-setting clinical trial?
"Presently, Neuren Pharmaceuticals Limited's NNZ-2591 has 3 ongoing trials spanning 11 cities and 2 countries. The initial trial of this drug was launched in 2022 and featured 20 participants undergoing its Phase 2 clinical testing phase. Since then, 1 study has been completed."
Are there opportunities to join this medical research at the present moment?
"Correct. Clinicaltrials.gov reports that the trial, first publicized on October 14th 2022, is actively seeking volunteers. 20 participants need to be sourced from 5 participating medical centres."
What is the extent of this trial's geographic coverage?
"The trial is conducted in a variety of locations, including the University of California at San Francisco (San Francisco, CA), University of Alabama at Birmingham (Birmingham, AL), and UT Southwestern (Dallas, TX)."
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