Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02790034
• Date of registration: 24/05/2016
• Prospective Registration: Yes
• Primary sponsor: Newron Pharmaceuticals SPA
• Public title: Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
• Scientific title: A Randomised, Double-Blind, Placebo-Controlled 6-month Study to Evaluate the Efficacy, Safety, and Tolerability of Sarizotan in Patients With Rett Syndrome With Respiratory Symptoms
• Date of first enrolment: August 2016
• Target sample size: 129
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02790034
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

Australia, India, Italy, United Kingdom, United States

Contacts

• Name: Ravi Anand, MD
• Affiliation: Newron Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

- Body weight = 10 kg

- Age = 4 years

- Diagnosis of Rett syndrome based on consensus clinical criteria and patients with
MECP2 duplications will not be eligible.

- Has at least 10 episodes of breathing dysrhythmia, defined by episodes =10 seconds of
breath holding (apnea), per hour during cardiorespiratory monitoring

- Ability to take study medication provided either as capsules or combined with
food/drink.

- Patient is cooperative, willing to complete all aspects of the study, and capable of
doing so with assistance of a caregiver.

Exclusion Criteria:

- Meets any of the diagnostic exclusion criteria for Rett syndrome, Typical (Neul et al,
2010);

- Patient is participating in a clinical trial with another investigational drug

- Hypersensitivity to sarizotan or other 5-HT1a agonists;

- 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;

- QTcF interval on the ECG is greater than 450 msec.

- Surgery planned during the study (except for insertion of gastrostomy tube);

- Severe diabetes mellitus or fatty acid oxidation disorder.

- 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.

- Females who are pregnant, breastfeeding, or of childbearing potential and not using a
hormonal contraceptive.



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Rett Syndrome

Intervention(s)

Drug: Placebo

Drug: Sarizotan

Primary Outcome(s)

Reduction in respiratory abnormality in patients with Rett syndrome [Time Frame: 3 days prior to Baseline up to week 24]

Secondary Outcome(s)

Global change from baseline [Time Frame: 24 weeks]

Pharmacokinetics profile of sarizotan and its comparison with the profile in adults [Time Frame: Baseline, 1 and 4 hr post-dose on Day 1, and 1 and 4 hr post-dose on Day 15]

Overall assessment of symptoms of Rett syndrome [Time Frame: 24 weeks]

Efficacy of sarizotan assessed by the caregiver [Time Frame: 24 weeks]

Motor behaviour [Time Frame: 24 weeks]

Caregiver burden [Time Frame: 24 weeks]

Respiratory symptoms - Incidence of breathing dysrhythmia episodes [Time Frame: 24 weeks]

Respiratory symptoms - Oxygen saturation [Time Frame: 24 weeks]

Respiratory symptoms - Number of hyperventilation episodes [Time Frame: 24 weeks]

Respiratory symptoms - Respiratory Distress Index; [Time Frame: 24 weeks]

Safety and tolerability of sarizotan in patients with Rett syndrome with respiratory symptoms. [Time Frame: 24 weeks]

Respiratory symptoms - Percent time spent with breathing dysrhythmia per hour [Time Frame: 24 weeks]

Secondary ID(s)

Sarizotan/001/II/2015

Source(s) of Monetary Support

Please refer to primary and secondary sponsors