What is the mechanism of action of this treatment?

The most common treatments for Pitt Hopkins Syndrome (PTHS) focus on improving neurological development and managing symptoms. NNZ-2591, a drug being studied, aims to enhance cognitive function and reduce behavioral issues by modulating neurotrophic factors and synaptic plasticity. This is crucial for PTHS patients as it addresses the core deficits in brain development and function, potentially improving quality of life and daily functioning. Other treatments may include supportive therapies like physical, occupational, and speech therapy to aid in overall development and skill acquisition.

What side effects are associated with this type of intervention?

Common treatments for neurological development disorders, which may be similar to those being studied for Pitt Hopkins Syndrome, often include medications such as antiepileptics and neuroleptics. These treatments can have side effects including drowsiness, dizziness, gastrointestinal issues, weight gain, and potential behavioral changes. It is important to monitor these side effects closely and discuss them with a healthcare provider to ensure the benefits of the treatment outweigh the risks.

What prior FDA approvals exist?

There is no specific information provided about prior FDA approvals for the treatment of Pitt Hopkins Syndrome. The trial NNZ-2591 is being studied for its potential therapeutic effects on neurological development in children and adolescents with Pitt Hopkins Syndrome. Generally, treatments for similar neurological conditions focus on managing symptoms and improving quality of life, but specific FDA-approved drugs for Pitt Hopkins Syndrome are not mentioned in the provided context.

What other types of research exist?

Promising novel alternatives to NNZ-2591 for Pitt Hopkins Syndrome patients include gene therapy approaches targeting the TCF4 gene, which is implicated in PTHS. Additionally, therapies that modulate synaptic function and neuroplasticity, such as trofinetide, which has shown potential in related neurodevelopmental disorders like Rett syndrome, could be considered. Advances in CRISPR/Cas9 gene editing and other genome editing technologies also hold promise for correcting genetic mutations at the source. Finally, therapies aimed at enhancing neurotrophic factors or using small molecules to modulate neural pathways involved in cognitive and developmental processes may offer new avenues for treatment.

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NNZ-2591 for Pitt Hopkins Syndrome (PTHS-001 Trial)

Phase 2

Waitlist Available

Research Sponsored by Neuren Pharmaceuticals Limited

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Each subject must be able to swallow the study medication provided as a liquid solution.

Males or females aged 3-17 years

Must not have

Current major or persistent depressive disorder (including bipolar depression)

Has planned surgery during the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 13 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a liquid medicine called NNZ-2591 in children and teenagers with Pitt Hopkins Syndrome. The goal is to see if the medicine is safe, how the body handles it, and if it helps with symptoms. Participants will take the medicine for several weeks.

Who is the study for?

This trial is for children and adolescents aged 3-17 with Pitt Hopkins Syndrome, who can swallow liquid medication, weigh at least 12kg, have a certain severity of disease symptoms, and haven't lost skills in the past 3 months. They must not be pregnant or have significant health issues like heart or kidney problems.

What is being tested?

The study tests NNZ-2591's safety and how well it works in kids with Pitt Hopkins Syndrome. It looks at how the body processes the drug (pharmacokinetics) and its effectiveness on various symptoms such as hyperventilation and intellectual disability.

What are the potential side effects?

While specific side effects are not listed here, they typically include reactions to the drug's tolerability which could range from mild discomforts like nausea to more serious conditions depending on individual responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can swallow liquid medication.

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I am between 3 and 17 years old.

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I have been diagnosed with PTHS and have a confirmed genetic cause.

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I can swallow liquid medication.

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I am between 3 and 17 years old.

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I have been diagnosed with PTHS and have a genetic test confirming it.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently experiencing major or long-term depression.

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I am scheduled for surgery during the study period.

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My body weight is less than 12kg.

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I tested positive for COVID-19 and was hospitalized for it in the last year.

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I do not have serious thyroid issues or diabetes needing insulin that is not well-managed.

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I have a serious kidney condition.

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My seizures are not under control.

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I do not have any major heart, liver, stomach, lung, hormone, or organ problems.

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I have significant vision or hearing loss that hasn't been corrected.

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I have had a stroke, brain injury, or similar brain condition.

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I have had or currently have catatonia.

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I have or had cancer before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 13 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~13 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 13 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Pharmacokinetic - Measurement of AUC

Pharmacokinetic - Measurement of Cmax

Pharmacokinetic - Measurement of t1/2

+2 more

Secondary study objectives

Exploratory efficacy measurement

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: NNZ-2591Experimental Treatment1 Intervention

NNZ-2591 oral solution (50mg/mL) to be administered twice daily dose for 13 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

NNZ-2591

2020

Completed Phase 2

~80

0 / 18Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Pitt Hopkins Syndrome (PTHS) focus on improving neurological development and managing symptoms. NNZ-2591, a drug being studied, aims to enhance cognitive function and reduce behavioral issues by modulating neurotrophic factors and synaptic plasticity. This is crucial for PTHS patients as it addresses the core deficits in brain development and function, potentially improving quality of life and daily functioning. Other treatments may include supportive therapies like physical, occupational, and speech therapy to aid in overall development and skill acquisition.

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