DDrare: Database of Drug Development for Rare Diseases

Searched query = "Rett syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ANA001XHCl (Syntagon) or VEXA-04 (Patheon); ANAVEX2-73; ANAVEX2-73 (blarcamesine); ANAVEX2-73 oral liquid; Acetate; Administration of a high dose of desipramine; Administration of a low dose of desipramine; Alpha-tocotrienol quinone; Blarcamesine; CANNABIDIOL; CBD; CBD - Oral Solution, is known as Epidyolex, and is the approved name; CBD Oral solution, is known as Epidyolex and is the approved name in the EU; Cannabidiol; Cannabidiol (CBD); Creatine; Creatine monohydrate; Desipramine; Dextromethorphan; Donepezil; Donepezil hydrochloride; EMD 128130; EPI-743; Epidyolex; Epidyolex and is the approved name in the EU; Fingolimod; Fingolimod (FTY720); GWP42003-P; Glatiramer; Glatiramer Acetate; Glatiramer Acetate (Copaxone®); Human insulin; Ketamine; Lactobacillus plantarum; Lovastatin; NNZ-2566; Probiotic-Lactobacillus plantarum PS128; Recombinant Human Insulin Growth Factor 1 (rhIGF-1); Rett-T; RhIGF-1; Risperidone; SARIZOTAN HYDROCHLORIDE; Sarizotan; Sarizotan hydrochloride; Tocotrienol; Topiramate; Tridecanoic Acid; Triheptanoin; Trofinetide

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2015-004448-20-GB (EUCTR)	28/11/2016	23/09/2016	Sarizotan: Treatment in patients with Rett Syndrome with Respiratory Symptoms.	A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with Rett Syndrome with Respiratory Symptoms	Rett syndrome MedDRA version: 20.0;Level: LLT;Classification code 10039000;Term: Rett's disorder;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: sarizotan hydrochloride Product Code: EMD 128130 INN or Proposed INN: sarizotan hydrochloride Other descriptive name: SARIZOTAN HYDROCHLORIDE Product Name: sarizotan hydrochloride Product Code: EMD 128130 INN or Proposed INN: sarizotan hydrochloride Other descriptive name: SARIZOTAN HYDROCHLORIDE Product Name: sarizotan hydrochloride Product Code: EMD 128130 INN or Proposed INN: sarizotan hydrochloride Other descriptive name: SARIZOTAN HYDROCHLORIDE	Newron Pharmaceuticals S.p.A.	NULL	Not Recruiting			Female: yes Male: yes	129	Phase 2	United States;Australia;Italy;United Kingdom;India
2	NCT02790034 (ClinicalTrials.gov)	August 2016	24/5/2016	Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms	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	Rett Syndrome	Drug: Sarizotan;Drug: Placebo	Newron Pharmaceuticals SPA	NULL	Terminated	4 Years	N/A	All	129	Phase 2;Phase 3	United States;Australia;India;Italy;United Kingdom

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2015-004448-20-GB
(EUCTR)

28/11/2016

23/09/2016

Sarizotan: Treatment in patients with Rett Syndrome with Respiratory Symptoms.

A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with Rett Syndrome with Respiratory Symptoms

Rett syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10039000;Term: Rett's disorder;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: sarizotan hydrochloride
Product Code: EMD 128130
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE
Product Name: sarizotan hydrochloride
Product Code: EMD 128130
INN or Proposed INN: sarizotan hydrochloride
Other descriptive name: SARIZOTAN HYDROCHLORIDE

Newron Pharmaceuticals S.p.A.

NULL

Not Recruiting

Female: yes
Male: yes

129

Phase 2

United States;Australia;Italy;United Kingdom;India

NCT02790034
(ClinicalTrials.gov)

August 2016

24/5/2016

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

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

Rett Syndrome

Drug: Sarizotan;Drug: Placebo

Newron Pharmaceuticals SPA

NULL

Terminated

4 Years

N/A

All

129

Phase 2;Phase 3

United States;Australia;India;Italy;United Kingdom