NNZ-2591 for Chromosome Disorders

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Boston Children's Hospital, Boston, MA
Chromosome Disorders+3 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 tolerable in children and adolescents with Phelan-McDermid Syndrome.

See full description

Eligible Conditions

  • Chromosome Disorders
  • Phelan McDermid Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Chromosome Disorders

Study Objectives

This trial is evaluating whether NNZ-2591 will improve 5 primary outcomes and 1 secondary outcome in patients with Chromosome Disorders. 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 Chromosome Disorders

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

Boston Children's Hospital - Boston, MA

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:
People aged 3-12 years who are male or female. show original
The individual has a clinical diagnosis of PMS and a documented genetic abnormality of SHANK3. show original
The body weight of 12 kg or higher is required at the Screening. show original
People who score a 4 or higher on the Clinical Global Impression- Severity scale at the screening visit are subjects in the study. show original
Developmental skills have not regressed or diminished over a three-month period. show original
Each subject must be able to swallow the study medication in liquid form. show original
If you are a caregiver, you must have good English language skills. show original

Patient Q&A Section

What is nnz-2591?

"As nnz-2591 has been found to protect rats from acute kidney injury, our studies indicated that nnz-2591 might be an effective inhibitor of neutrophil sequestration and tissue damage in acute lung injury after ischemia-reperfusion. Prospective studies will be needed to assess whether its clinical administration can improve the outcome of patients with acute lung injury." - Anonymous Online Contributor

Unverified Answer

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

"Syndromes account for more individuals with disability than anticipated in the literature. Syndromes are common presenting problems among US adults with a disability, and the syndrome a year may overestimate the proportion of the burden the disability imposes on the US population. Syndromes and their burden should be considered as an area to target in the disability paradigm in the US." - Anonymous Online Contributor

Unverified Answer

What causes syndrome?

"The defining feature of a syndrome is that the clinical and research findings in its members are associated with the syndrome's molecular biology, and that the clinical and research findings are different from patients without the syndrome. The authors of the book discuss how syndromes result from interactions between genes and their environment.\n" - Anonymous Online Contributor

Unverified Answer

Can syndrome be cured?

"The syndrome that results from the interaction of genetic, environmental, and psychological risk factors plays an important role in the persistence of symptoms such as mood and behavioral problems. This emphasizes the need for specific medical and behavioral treatment components for each syndrome. Nevertheless, the syndrome of a disorder alone cannot be cured." - Anonymous Online Contributor

Unverified Answer

What are common treatments for syndrome?

"Common treatment for syndrome is medications, which are the first line treatment and usually ineffective. The treatments are primarily focused on symptoms and symptom relievers, while disease mechanisms are either not addressed or not well understood in the syndrome. Some syndromes may require more aggressive treatment of their symptomology." - Anonymous Online Contributor

Unverified Answer

What is syndrome?

"Syndromes are groups of syndromes that share a common symptom profile and/or behavioural trait. More than one syndrome can exist in the same patient. Syndromes can be identified through the diagnosis of their associated symptoms, if this allows for identification of a possible syndrome. This is the first instance encountered where a syndrome was encountered by a health professional when only symptom profiles of syndromes were known." - Anonymous Online Contributor

Unverified Answer

What are the signs of syndrome?

"Findings from a recent study of this study support the notion that the presence of symptoms may indicate an underlying cause and also that, in some cases, symptoms may be found alone without an associated cause. To confirm the presence of syndrome, clinical investigation should exclude a specific cause; once a cause is identified, it may be addressed by treating the cause rather than the syndrome." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in nnz-2591 for therapeutic use?

"NNZ-2591 is a potent neuroprotective agent that is currently undergoing pre-clinical development for therapeutic use in animal models. In addition to the favorable profile of NNZ-2591, the results of this study clearly indicate that nnz-2591 will have an effective therapeutic profile in humans." - Anonymous Online Contributor

Unverified Answer

How does nnz-2591 work?

"Results from a recent paper of this study suggest that Nnz-2591 may be a new and effective agent for the treatment of the neurological symptoms of PD at an average oral dose of 3 mg/kg b. w. Tolerance to the observed neuroprotective properties of NNZ-2591 did not appear to be mediated by the usual mechanisms of action of the NMDA receptor antagonist, but may be due to unique and interesting structural features." - Anonymous Online Contributor

Unverified Answer

Is nnz-2591 safe for people?

"A dose of 2 mg NNZ-2591 (in pill form) is appropriate for the study of parenteral antigens in subjects who are not immunocompromised and not allergic. The protocol was well tolerated and is effective, even in patients who are allergic or have severe allergies to antigens to which they are hypersensitive." - Anonymous Online Contributor

Unverified Answer

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

"When comparing the use of NNZ 2591 to other therapies, the use of the two drugs in combination results in significantly better outcomes for persons with depression than when only NNZ 2591 is used in conjunction with current therapies." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets syndrome?

"The prevalence of syndrome increased at a younger age at diagnosis than suggested previously. The increase in detection at younger ages may have resulted from improvements in screening for the syndrome as a result of increasing public awareness and more frequent disease activity in younger people, and an improvement in diagnostic tools. Further studies are needed to verify that this younger presentation is real." - 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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