Clinical trial of the drug Sarizotan involving individuals with Rett syndrome, is now underway in Australia

April 2018 – A study to evaluate the effectiveness, safety and tolerability of the drug Sarizotan involving individuals with Rett syndrome who have breathing abnormalities, has finally got underway in Australia. Other participating countries are India, Italy, the United Kingdom and the United States.

This research is being sponsored by Newron Pharmaceuticals S.p.A, a biopharmaceutical company based in Milan, which is focussed on the development of novel therapies for patients with diseases of the central and peripheral nervous systems. RSAA’s involvement was accepted by Newron in October 2017, with the Association’s role being to manage reimbursement of the accommodation, travel and meal costs incurred by participating families. Newron has provided RSAA with funds for such.

The Fiona Stanley Hospital, Perth, is where Australian participants in the trial are required to attend.

ELIGIBILITY (Source: Australian and New Zealand Clinical Trials Register):

  • Participant is female or male aged 4 years or older and a body weight of 10 kg or more
  • Participant has a diagnosis of Rett syndrome based on consensus clinical criteria. Patients with MECP2 duplications will not be eligible.
  • Participant has at least 10 episodes of breathing dysrhythmia, defined by episodes =10 seconds of breath holding (apnea) per hour during cardiorespiratory monitoring
  • Participant has an ability to take study medication provided, either as capsules or combined with food/drink
  • Participant is cooperative, willing to complete all aspects of the study, and capable of doing so with assistance of a caregiver.

Key EXCLUSION criteria:

  • Participant meets any of the diagnostic exclusion criteria for Rett syndrome, Typical (Neul et al, 2010);
  • Participant is participating in a clinical trial with another investigational drug
  • Participant has a hypersensitivity to Sarizotan or other 5-HT1a agonists
  • Participant has a current clinically significant (as determined by Investigator) cardiovascular, respiratory (e.g. severe asthma), gastrointestinal, renal, hepatic, hematologic or other medical disorders, in addition to those directly related to the patient’s Rett syndrome
  • Participant has a QTcF interval on the electrocardiogram (ECG) greater than 450 millisecs
  • Participant has surgery planned during the study (except for insertion of a gastrostomy tube)
  • Participant has severe diabetes mellitus or a fatty acid oxidation disorder
  • Participant has an ophthalmologic history including any of the following conditions: albino patients, family history of hereditary retinal disease, retinitis pigmentosa, any active retinopathy or severe diabetic retinopathy
  • Participant is a female who is pregnant, breastfeeding, or of childbearing potential and is not using a hormonal contraceptive.

What does participation in this research involve?

The clinical trial consists of two parts –

An initial treatment period of 24 weeks

The trial begins with prospective participants being subjected to a number of tests or procedures will include a review of his or her medical history; examinations (both physical and neurological); vital signs measurement (weight, height, blood pressure, pulse, temperature, respiratory rate); electrocardiogram; collection of both blood and urine samples; and respiratory monitoring at home. Screening can last up to 4 weeks and will require the participant to visit the Fiona Stanley Hospital at least once during that time.

Once having successfully been through the screening process, a participant may need to spend 2 nights at the Fiona Stanley Hospital when taking part in those activities set down for Days 0, 1 and 2.

Day 0 (the first day) – A number of assessments involving the participant will be carried out at the Fiona Stanley Hospital;

Day 1 (the second day) – The participant will be given the first dose of the Study medication (study drug or placebo) at the Hospital during the evening;

Day 2 (the third day) – The participant will receive a morning dose of the Study medication, 4 hours after which he or she can then leave the Hospital;

Day 7 – Study staff will telephone the participating family;

Days 14 and 15 – A number examinations/procedures are to be conducted at the Hospital, therefore a visit there is required. It may be that a participant may also require an overnight stay;

Day 56 (during week 8 of the Study) – Another visit to the Hospital is necessary to enable a number of assessments, examinations and procedures, to be conducted;

Day 112 (during week 16 of the Study) – Another visit to the Hospital is required to enable similar assessments, examinations and procedures, to those that were conducted on Day 56;

Day 168 (during week 24 of the Study) – Another visit to the Hospital is required to enable similar assessments, examinations and procedures that were conducted on Days 56 and 112. Day 168 which marks the end of the initial treatment period, will involve the participant taking his or her final dose of study medication at the Hospital, unless he or she is continuing in the trial’s Extension Study;

The second part of the clinical trial is the safety follow-up period and takes place on Day 182.

Day 182 (or 14 days after receiving his or her last dose of the Study medication) – Another visit to the Hospital is required to enable a number of assessments to be conducted. This visit only applies to those participants who completed the initial 24 week treatment period but are not continuing treatment in the extension treatment period or were participants who discontinued their involvement prior to completing the initial 24 week period.

The extension period also goes for 24 weeks and follows on from the initial treatment phase. Involvement in the extension has to be at the wish of the participant’s family. All participants will receive Sarizotan. If at the end of this period, the participant is doing well as result of receiving the medication, he or she may be able to have their treatment extended by being provided with the drug.

An enormous commitment is being asked of those Australian Rett syndrome families who wish to be involved in the trial, particularly those who live outside Western Australia. As stated earlier, RSAA will assist with the payment of travel, accommodation, and meal expenses, incurred by participating families.

If wanting further information on the Australian trial of Sarizotan, please contact:

Dr Janine Spencer (Head of Paediatric Medicine, Fiona Stanley Hospital)
phone: 0419 577 234 email: [email protected]

31 October 2018 – BUSINESS WIRE advised that Newron Pharmaceuticals S.p.A. , a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system, welcomed investors, analysts and media today to its well-attended research and development (R&D) day in New York City………………………………………………………………………

Stefan Weber, Chief Executive Officer of Newron Pharmaceuticals, commented: “We were delighted to host presentations from leading clinicians on the treatment options for Rett syndrome and treatment resistant schizophrenia at today’s R&D event, as well as to provide an update on our clinical development programs………………………………………………………………………………………………………

Ravi Anand, MD, Newron’s Chief Medical Officer, presented the status of the STARS study. By the third week of October (2018), 116 patients had been randomized to treatment, with another four qualifying for randomization. Newron is therefore on track to reach its target of enrolling 129 patients during the remaining months of 2018 and now expects to report results from the study in Q3 2019 ……………………………………………………………………………………………………………………..

April 27, 2018
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RETT Syndrome Association of Australia