A Phase 2/3 interventional study of Sarizotan low dose and Sarizotan high dose in Rett Syndrome, sponsored by Newron Pharmaceuticals SPA. Terminated at 14 sites in 5 countries. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2021-12-21.
Sponsored by Newron Pharmaceuticals SPA · Phase 2/3, Interventional, and Treatment
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks (the latter for patients with no safety and tolerability issues).
This is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and efficacy of multiple doses of sarizotan in patients with Rett syndrome with respiratory abnormalities. The study participants will be randomized to either sarizotan between 2 and 10 mg bid or placebo bid, based on age and weight criteria.
All patients who have completed the final evaluations at Week 24 (Day 168) and have no safety or tolerability issues that would preclude continuing on the study medication and have been compliant with the trial requirements will have the option of continuing open-label treatment with Sarizotan for up to 168 weeks.
96 studies on the registry are indexed under Rett Syndrome; 19 are open to participants now.
This study's enrollment of 129 is above the median of 30 across 64 interventional studies indexed under Rett Syndrome.
Browse Rett Syndrome studies →Newron Pharmaceuticals SPA is the lead sponsor of 22 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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)
Drug: Sarizotan low 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)
Drug: Sarizotan high dose
Placebo bid for 24 wks DB age 4 and above
Drug: Placebo
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.
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
Placebo BID followed by assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.
Reduction in Respiratory Abnormality in Patients With Rett Syndrome
Measured as the percent change in the number of apnea episodes per hour during awake time, calculated using an ambulatory data acquisition system (BioRadioTM) as part of home monitoring procedure. BioRadioTM record specific respiratory and cardiac parameters.
Time frame: Baseline up to week 24
Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change
Caregiver-rated Impression of Change (CIC): 7-point scale requiring the caregiver to rate how much the patient's illness has improved or worsened relative to the baseline state. 7-point Likert-type scale for which ratings range from 1 = very much improved to 7 = very much worse, with 4 = no change. This caregiver-rated measure considered activities, behavior, mood and functioning. This rating was performed in consultation with the study Investigator but was based largely on the caregivers' evaluation during the reporting period. The single rating of the CIC was to be based on changes in the following domains: • Activities (watching TV, interest in conversations around her, cooperation during toileting, dressing/bathing, etc.), • Communication (verbal or by eye movements, hand movements, or head movements), • Behavior (agitation, refusal to feed, scratching, social avoidance), • Participation in family/outdoor/social events)
Time frame: 24 weeks
The study was conducted at 14 sites in 5 countries. Enrollment occurred from 26-Oct-2016 through February 2019 . An analysis was planned when the primary efficacy data at Week 24 were available for all patients (06 August 2019 - last patient last visit for double-blind part of the study). Patients without safety and tolerability issues continued in the open label with sarizotan for an additional 24 weeks and then another 48 weeks if no safety issues were present.
| Milestone | Sarizotan Low Dose | Sarizotan High Dose | Placebo |
|---|---|---|---|
| Started | 33 | 56 | 40 |
| Completed | 27 | 46 | 36 |
| Not completed | 6 | 10 | 4 |
Measured as the percent change in the number of apnea episodes per hour during awake time, calculated using an ambulatory data acquisition system (BioRadioTM) as part of home monitoring procedure. BioRadioTM record specific respiratory and cardiac parameters.
| % of change in mean counts per hr | Sarizotan Low Dose | Sarizotan High Dose | Placebo |
|---|---|---|---|
| Reduction in Respiratory Abnormality in Patients With Rett Syndrome | 1.54 ± 10.1228 | 13.211 ± 7.5075 | 18.503 ± 8.441 |
Caregiver-rated Impression of Change (CIC): 7-point scale requiring the caregiver to rate how much the patient's illness has improved or worsened relative to the baseline state. 7-point Likert-type scale for which ratings range from 1 = very much improved to 7 = very much worse, with 4 = no change. This caregiver-rated measure considered activities, behavior, mood and functioning. This rating was performed in consultation with the study Investigator but was based largely on the caregivers' evaluation during the reporting period. The single rating of the CIC was to be based on changes in the following domains: • Activities (watching TV, interest in conversations around her, cooperation during toileting, dressing/bathing, etc.), • Communication (verbal or by eye movements, hand movements, or head movements), • Behavior (agitation, refusal to feed, scratching, social avoidance), • Participation in family/outdoor/social events)
| score on a scale | Sarizotan Low Dose | Sarizotan High Dose | Placebo |
|---|---|---|---|
| Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change | 3.6 ± 0.67 | 3.5 ± 1.12 | 3.4 ± 0.79 |
Collected over AEs will be collected, from the time of signing of Informed Consent to the end of the safety follow-up period (14 days after last dose of study medication at 24 weeks). In addition, all subjects will be followed up for 30 days after last dose for SAEs, up to a total of 28 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Sarizotan Low Dose | 0/33 (0%) | 2/33 (6.1%) | 25/33 (75.8%) |
| Sarizotan High Dose | 0/56 (0%) | 10/56 (17.9%) | 46/56 (82.1%) |
| Placebo | 0/39 (0%) | 5/39 (12.8%) | 30/39 (76.9%) |
| Event | Sarizotan Low Dose | Sarizotan High Dose | Placebo |
|---|---|---|---|
| Accidental OverdoseInjury, poisoning and procedural complications | 0/33 | 0/56 | 2/39 |
| SomnolenceNervous system disorders | 0/33 | 2/56 | 1/39 |
| SeizureNervous system disorders | 1/33 | 0/56 | 0/39 |
| Gingival HypertrophyGastrointestinal disorders | 1/33 | 0/56 | 0/39 |
| Lower respiratory tract infectionInfections and infestations | 1/33 | 1/56 | 0/39 |
| GingivitisInfections and infestations | 1/33 | 0/56 | 0/39 |
| Medication errorInjury, poisoning and procedural complications | 0/33 | 1/56 | 1/39 |
| Electrocardiogram QT prolongedInvestigations | 0/33 | 1/56 | 1/39 |
| Femur FractureInjury, poisoning and procedural complications | 0/33 | 0/56 | 1/39 |
| NeutropeniaBlood and lymphatic system disorders | 0/33 | 1/56 | 0/39 |
| Event | Sarizotan Low Dose | Sarizotan High Dose | Placebo |
|---|---|---|---|
| SeizureNervous system disorders | 8/33 | 15/56 | 5/39 |
| SomnolenceNervous system disorders | 2/33 | 10/56 | 6/39 |
| VomitingGastrointestinal disorders | 5/33 | 2/56 | 2/39 |
| Psychomotor hyperactivityNervous system disorders | 4/33 | 1/56 | 3/39 |
| PyrexiaGeneral disorders | 4/33 | 3/56 | 0/39 |
| CoughRespiratory, thoracic and mediastinal disorders | 1/33 | 3/56 | 4/39 |
| DiarrhoeaGastrointestinal disorders | 2/33 | 4/56 | 4/39 |
| DystoniaNervous system disorders | 3/33 | 3/56 | 1/39 |
| Sleep DisorderPsychiatric disorders | 3/33 | 4/56 | 1/39 |
| InsomniaPsychiatric disorders | 3/33 | 2/56 | 2/39 |
| Age, Categorical(Participants) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| <=18 years | 23 | 35 | 27 | 85 |
| Between 18 and 65 years | 10 | 21 | 13 | 44 |
| >=65 years | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| Mean | 13.23 ± 7.195 | 15.55 ± 9.362 | 14.13 ± 9.155 | 14.51 ± 8.780 |
| Sex: Female, Male(Participants) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| Female | 33 | 56 | 40 | 129 |
| Male | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB)(Participants) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| Hispanic or Latino | 4 | 5 | 4 | 13 |
| Not Hispanic or Latino | 29 | 51 | 36 | 116 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 |
| Asian | 8 | 16 | 13 | 37 |
| Native Hawaiian or Other Pacific Islander | 0 | 1 | 0 | 1 |
| Black or African American | 1 | 1 | 3 | 5 |
| White | 23 | 37 | 22 | 82 |
| More than one race | 1 | 0 | 0 | 1 |
| Unknown or Not Reported | 0 | 1 | 2 | 3 |
| Region of Enrollment(Participants) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| United States | 11 | 19 | 11 | 41 |
| Italy | 8 | 13 | 9 | 30 |
| United Kingdom | 5 | 10 | 6 | 21 |
| Australia | 1 | 0 | 2 | 3 |
| India | 8 | 14 | 12 | 34 |
| Duration of Rett syndrome(months) | Sarizotan Low Dose | Sarizotan High Dose | Placebo | Total |
|---|---|---|---|---|
| Mean | 87.90 ± 72.536 | 97.18 ± 72.922 | 73.94 ± 65.800 | 87.94 ± 70.696 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is terminated, as verified in Nov 2021. You cannot join it, but the record below documents what was studied.
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