About the Safety Study of NNZ-2566

Results of the Phase II Trial (that Katelin participated in) Released Nov. 11, 2014

Phase II Announcement

For an updated list of Neuren Announcements click here!

Taken from the International Rett Syndrome Foundation Website

Safety Study of NNZ-2566 in Patients With Rett Syndrome

Location: Baylor College of Medicine/Texas Children’s Hosptial, University of Alabama, Birmingham

Collaborators: Neuren, Texas Children’s Hospital, University of Alabama, Birmingham,  International Rett Syndrome Foundation

Summary: Rett Syndrome is a developmental disorder primarily if not exclusively affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of regression with onset of systemic and neurological signs. The CNS symptoms of Rett Syndrome include learning disability, autism and epilepsy and these can be severe and highly debilitating. Affected individuals also show signs of autonomic dysfunction, reflected in cardiovascular and respiratory abnormalities. There is no currently effective treatment for Rett Syndrome.

This study will investigate the safety and tolerability of treatment with oral administration of NNZ-2566 at 35 mg/kg or 70 mg/kg BID in adolescent or adult females with Rett Syndrome. The study also will also investigate measures of efficacy during treatment.

Eligibility: Ages Eligible for Study: 16 Y ears to 45 Years Genders Eligible for Study: Female


Inclusion Criteria:

  • Diagnosis of Rett Syndrome with proven mutation of the MeCP2 gene
  • Age 16 to 45 years
  • Severity rating of between 14 and 36 (Rett Syndrome Natural History/Clinical Severity Scale)
  • Subjects with a Clinical Global Impression – Severity (CGI-S) score of 4 or greater.
  • Permitted concomitant medications must be stable for at least 4 weeks prior to enrolment. The following concomitant medications are permitted: anticonvulsants which do not have liver inducing effects; beta-blockers; medications for the treatment of gastroesophageal reflux disease (GERD); medications for the treatment of chronic respiratory conditions such as asthma; medications for the treatment of anxiety; medications for the treatment of depression; medications for the treatment of psychosis; hormonal contraceptives. Melatonin for difficulties with sleep onset is also permissible.
  • Ability to swallow study medication provided as a liquid solution, or via gastrostomy tube

Exclusion Criteria:

  • No detectable abnormality of the EEG at baseline
  • Actively undergoing regression
  • QTc exclusions (any of the following): baseline/screening QT/QTcf interval of 450 msec; history of risk factors for torsade de pointes (e.g. heart failure, hypokalemia (serum potassium at screening < 3.0 mmol/L) or family history of long QT syndrome; QT/QTc prolongation previously or currently controlled with medication
  • Current treatment with insulin
  • Hgb A1C values outside the normal reference range at screening
  • Current or past treatment with IGF-1
  • Current or past treatment with growth hormone
  • Current treatment with NMDA antagonists
  • Current or planned use of non-medication based interventional therapy during the period of the study (defined as 6 week screening period followed by 6 week dosing and follow-up period)
  • Current clinically significant cardiovascular, renal, hepatic or respiratory disease
  • Gastrointestinal disease which may interfere with the absorption, distrbution, metabolism or excretion of the the study medication
  • History of, or current cerebrovascular disease or brain trauma
  • History of, or current significant endocrine disorder e.g. hypo or hyperthyroidism or diabetes mellitus
  • History of, or current malignancy
  • Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as measured at screening or baseline
  • Confirmed pregnancy
  • Significant hearing and/or visual impairment that may affect ability to complete the test procedures
  • Enrollment in another clinical trial within the previous 30 days
  • Previously randomized in this clinical trial
  • Allergy to strawberries.

– See more at: http://rettsyndrome.org/research-programs/clinical-trials-and-databases/rett-syndrome-specific-trials#NNZ2566

2 Responses to About the Safety Study of NNZ-2566

  1. Glenn says:


    About 6 months ago i invested in NEU on the Australian Stock Exchange. Before this i had never heard of Retts Syndrome; the more i researched the stock the more i learnt about this disorder. During my research i have come across your website and wanted to personally share my thoughts on what a great website you have.

    Everyone invests to make money however after reading many stories about families affected by this disorder i truly hope trofinetide proves to be a drug that helps. Whilst it is a very exciting time to be an investor in this stock i also empathize with the families who have alot more at stake than just those invested. I truly hope the FDA approve the Phase 3 design NEU will propose this quarter and trofinetide is readily available and beneficial to those in need.

    Yours sincerely

    • melelllan says:

      Thank you for your kind words. I am, in return, grateful for every investor no matter their reasons. I truly believe in Trofinetide and there are many at Neuren that I consider my and my daughter’s heroes. Even if ultimately Trofinetide were to fail (as if 🙄) I would think the same. Best of luck to all of us. I want my daughter helped and I want all of you to make millions. It’s a fair trade off in my eyes. 🙂

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