Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02790034




Registration number
NCT02790034
Ethics application status
Date submitted
24/05/2016
Date registered
3/06/2016
Date last updated
21/12/2021

Titles & IDs
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
Secondary ID [1] 0 0
Sarizotan/001/II/2015
Universal Trial Number (UTN)
Trial acronym
STARS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sarizotan low dose
Treatment: Drugs - Sarizotan high dose
Treatment: Drugs - Placebo

Experimental: Sarizotan low dose - 2 mg or 5 mg bid based on age and weight criteria for 24 wks DB 2 mg bid (4 to \<13 years; =13 years of age and weighing \<25 kg 5 mg bid (=13 years of age and weighing =25 kg)

Experimental: Sarizotan high dose - 5 mg or 10 mg bid based on age and weight criteria for 24 wks DB 5 mg bid (4 to \<13 years; =13 years of age and weighing \<25 kg 10 mg bid (=13 years of age and weighing =25 kg)

Placebo comparator: Placebo - Placebo bid for 24 wks DB age 4 and above


Treatment: Drugs: Sarizotan low dose
2 mg or 5 mg bid based on age and weight criteria for 24 wks DB 2 mg bid (4 to \<13 years; =13 years of age and weighing \<25 kg; 5 mg bid (=13 years of age and weighing =25 kg). Assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.

Treatment: Drugs: Sarizotan high dose
5 mg or 10 mg bid based on age and weight criteria for 24 wks DB 5 mg bid (4 to \<13 years; =13 years of age and weighing \<25 kg; 10 mg bid (=13 years of age and weighing =25 kg) Assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients

Treatment: Drugs: Placebo
Placebo BID followed by assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Reduction in Respiratory Abnormality in Patients With Rett Syndrome
Timepoint [1] 0 0
Baseline up to week 24
Secondary outcome [1] 0 0
Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change
Timepoint [1] 0 0
24 weeks

Eligibility
Key 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.
Minimum age
4 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/10/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/05/2020
Sample size
Target
Accrual to date
Final
129
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
South Metropolitan Health Service Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
6961 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
India
State/province [6] 0 0
Kerala
Country [7] 0 0
India
State/province [7] 0 0
Tamilnadu
Country [8] 0 0
India
State/province [8] 0 0
Mumbai
Country [9] 0 0
India
State/province [9] 0 0
New Delhi
Country [10] 0 0
Italy
State/province [10] 0 0
Tuscany
Country [11] 0 0
Italy
State/province [11] 0 0
Milano
Country [12] 0 0
United Kingdom
State/province [12] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Newron Pharmaceuticals SPA
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ravi Anand, MD
Address 0 0
Newron Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02790034