NNZ-2591 for Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
University of California at San Francisco, San Francisco, CA
Syndrome+4 More
NNZ-2591 - Drug
Eligibility
< 18
All Sexes
What conditions do you have?
Select

Study Summary

This study is evaluating whether a drug called NNZ-2591 is safe and effective in children and adolescents with Pitt Hopkins Syndrome.

See full description

Eligible Conditions

  • Syndrome
  • Pitt Hopkins Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Syndrome

Study Objectives

This trial is evaluating whether NNZ-2591 will improve 5 primary outcomes and 1 secondary outcome in patients with Syndrome. Measurement will happen over the course of 13 weeks.

13 weeks
Exploratory efficacy measurement
Pharmacokinetic - Measurement of AUC
Pharmacokinetic - Measurement of Cmax
Pharmacokinetic - Measurement of t1/2
Pharmacokinetic - Measurement of time to Cmax
Safety and Tolerability

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Syndrome

Trial Design

1 Treatment Group

NNZ-2591
1 of 1
Experimental Treatment

This trial requires 20 total participants across 1 different treatment group

This trial involves a single treatment. NNZ-2591 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

NNZ-2591
Drug
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
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 13 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 13 weeks for reporting.

Closest Location

University of California at San Francisco - San Francisco, CA

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 7 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Clinical diagnosis of PTHS with a documented disease-causing genetic etiology for the disorder.
Males or females aged 3-17 years.
Body weight of 12kg or higher at screening
Subjects with a Clinical Global Impression- Severity (CGI-S) score of 4 or greater at the Screening visit.
Not actively undergoing regression or loss of skills, defined as no persistent loss of previously acquired developmental skills for a period within 3 months of the Screening visit
Each subject must be able to swallow the study medication provided as a liquid solution.
Caregiver(s) must have sufficient English language skills.

Patient Q&A Section

How many people get syndrome a year in the United States?

"More than 5 million people are diagnosed with syndrome a year. Syndromes can be serious and disabling in their own right, but also contribute to substantial health care costs for those with them." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"This article seeks to explain the phenomenon of syndromes, their nature and structure, and their role in diagnosis and management. It is hoped that through the use of this overview, the clinical reader may obtain a greater appreciation of the different syndromes diagnosed, thereby providing the foundation for effective clinical care. This article outlines the characteristics of the syndromes and gives guidance as to the presentation of the syndromes to the clinician." - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"The five signs of systemic disease are frequently present in hospitalized patients. Only when we take into account these signs is the diagnosis of disease possible. However, when there are two or more signs, it is reasonable to consult a medical professional." - Anonymous Online Contributor

Unverified Answer

Can syndrome be cured?

"The syndrome cannot be fixed, but there are steps that can be taken to improve the quality of life and functioning of persons with CFS, including (1) maximizing the patient's competence through treatment and coping methods and (2) expanding access to treatment to those persons who suffer chronic illness." - Anonymous Online Contributor

Unverified Answer

What are common treatments for syndrome?

"The use of a clinical algorithm, incorporating PSA and age, to identify participants who have a life-threatening form of prostate cancer may allow more effective prevention. An approach consistent with the PSA paradigm can be taken when counseling patients with a life-threatening prostate cancer. Most patients who survive a life-threatening form of prostate cancer do not require immediate surgery. Because survival time could be extended by treating patients with aggressive medical therapies, an evidence-based treatment approach will greatly increase survival." - Anonymous Online Contributor

Unverified Answer

What causes syndrome?

"The cause of syndrome is unclear, however a number of genetic, environmental and psychological factors have been proposed. It is currently not known whether syndrome is caused by a single genetic defect or a composite of defects. Syndromic autism: does it exist? answer: It seems highly probable that syndromic autism exists and should be further investigated. It is not known whether syndrome is caused by a single genetic defect or a composite of defects. It is possible that syndromic autism exists but is caused by a different genetic defect. Syndromic autism is likely to be associated with a variety of genetic and non-genetic factors." - Anonymous Online Contributor

Unverified Answer

How serious can syndrome be?

"Aneurysmal bone cysts of the sternocleidomastoid artery can be fatal. Patients with aneurysmal cysts in the sternocleidomastoid artery can die in just four hours because of an intracranial hemorrhage. In this case, a fatal illness accompanied by a seizure has resulted from a relatively harmless complication of a rare disease." - Anonymous Online Contributor

Unverified Answer

How does nnz-2591 work?

"Results from a recent clinical trial of this proof-of-concept study indicate that the use of nnz-2591 is promising for treatment of human T-cell acute lymphoblastic leukemia (HT-ALL) as an induction therapy. Further study with larger numbers and longer follow-up times are required to determine the significance of nnz-2591 at a fuller therapeutic setting in children with HT-ALL." - Anonymous Online Contributor

Unverified Answer

Is nnz-2591 typically used in combination with any other treatments?

"In line with the concept of the p-channel as an ion channel rather than a ligand-gated ion channel, NNZ2691, like all other currently known p-potassium channel antagonists, blocks the potassium conductance, probably by binding to the extracellular ion-binding domain. The selectivity of NNZ2691 toward the Kv1.1 (K1) and Kv1.3 (K3) K+ currents is consistent with the concept of K+ channels as voltage-gated channels, although it must be noted that this assumption is only a working hypothesis." - Anonymous Online Contributor

Unverified Answer

Has nnz-2591 proven to be more effective than a placebo?

"There does not appear to be any difference in effectiveness between nNZ2591 and placebo, and therefore the need for future trials in PBC remain unfulfilled." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating syndrome?

"The search is still ongoing at present and most studies report some promising findings. Although no new clinical trials have occurred to the best of our knowledge in the last 10 years, our hope is to develop a better understanding of the pathogenesis of the syndrome, its association with other conditions, and its treatments. (http://www.acn.nl/news/viewstory/0209052.article.asp[6]." - Anonymous Online Contributor

Unverified Answer

Is nnz-2591 safe for people?

"Despite a trend towards higher doses, the results do not show an increased risk of thrombosome effects. The safety and effectiveness of Nnz-2591 in the treatment of patients with SFT is well established." - 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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