DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
13	多発性硬化症／視神経脊髄炎	4
51	全身性強皮症	1
86	肺動脈性肺高血圧症	1

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	NCT04468165 (ClinicalTrials.gov)	January 2021	8/7/2020	Effectiveness and Safety of Generic Delayed-Release Dimethyl Fumarate (Sclera® or Marovarex ®, Hikma) in Routine Medical Practice in the Treatment of Relapsing-Remitting Multiple Sclerosis in MENA Region	Effectiveness and Safety of Generic Delayed-Release Dimethyl Fumarate (Sclera® or Marovarex ®, Hikma) in Routine Medical Practice in the Treatment of Relapsing-Remitting Multiple Sclerosis in MENA Region	Relapsing Remitting Multiple Sclerosis	Drug: Dimethyl Fumarate (DMF)	Hikma Pharmaceuticals LLC	NULL	Not yet recruiting	18 Years	N/A	All	155		NULL
2	NCT03101735 (ClinicalTrials.gov)	September 23, 2016	3/3/2017	Study to Assess Resource Utilization and Quality of Life of Patients With RRMS Treated With Tecfidera in Greece	A Real Life, Non-interventional, Multicentre Study to Assess Resource Utilization and Quality of Life of Patients With Relapsing Forms of Multiple Sclerosis Treated With Dimethyl Fumarate in Greece - the FIDELITY Study	RRMS	Drug: Dimethyl Fumarate (DMF)	Genesis Pharma CNS & Specialty	NULL	Active, not recruiting	18 Years	65 Years	All	455		Greece
3	EUCTR2015-004116-38-SE (EUCTR)	18/12/2015	20/10/2015	A clincial study comparing the effectiveness of two drugs, named Rituximab and Dimethyl Fumarate (Tecfidera®), for the neurological disease Multiple Sclerosis.	RItuximab versus FUmarate in Newly Diagnosed Multiple Sclerosis – RIFUND-MSA randomized phase 3 study comparing Rituximab with Dimethyl Fumarate in early Relapsing-Remitting Multiple SclerosisObjective: To compare the efficacy of rituximab on the ability to prevent relapses in early RRMS and CIS compared with dimethyl fumarate (DMF), which is an approved first-line medication for RRMS today, using a phase 3 design.Population: Patients with newly diagnosed RRMS or CIS with no more than 10 years disease duration (since diagnosis), 18 – 50 years of age and previously not treated with immunomodulating drugs OR treated with first-line injectables. Patients should display protocol-defined clinical or radiological disease activity during the preceding year before screening for inclusion.Intervention: Treatment with rituximab (Mabthera®) with an initial dose of 1000 mg intravenously (iv) followed by 500 mg iv every six months.Control: Treatment with DMF (Tecfidera®) 240 mg twice daily. The two treatments are randomised in a 1:1 proportion.Outcome: Primary outcome is the relative risk of experiencing a relapse during the two–year period for either compound. As secondary endpoints worsening on neurological disability, magnetic-resonance imaging-defined disease activity and effect on cerebrospinal fluid biomarkers will be analysed. In addition, health-economic evaluations of using rituximab as first-line treatment for RRMS will be performed.	Patients with multiple sclerosis (MS) or patients presenting with symptoms highly suspicious of MS while not completely fulfilling diagnostic criteria, 18 - 50 years of age and no more than 10 years of disease duration (from diagnosis). Patients treated with immunomodulatory drugs or treated with first-line injectable therapies (eg interferons or glatiramer acetate) may be included.;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: Mabthera INN or Proposed INN: RITUXIMAB Trade Name: Tecfidera INN or Proposed INN: DIMETHYL FUMARATE Other descriptive name: DIMETHYL FUMARATE	Department of Clinical Sciences, Danderyd Hospital	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 3	Sweden
4	NCT02047097 (ClinicalTrials.gov)	November 19, 2013	24/1/2014	Dimethyl Fumarate (DMF) Observational Study	A Multicenter, Global, Observational Study to Collect Information on Safety and to Document the Drug Utilization of Tecfidera™ (Dimethyl Fumarate) When Used in Routine Medical Practice in the Treatment of Multiple Sclerosis (ESTEEM)	Multiple Sclerosis	Drug: dimethyl fumarate	Biogen	NULL	Active, not recruiting	18 Years	N/A	All	5496		United States;Argentina;Australia;Austria;Canada;Czechia;Denmark;France;Germany;Hungary;Ireland;Italy;Netherlands;New Zealand;Norway;Poland;Portugal;Puerto Rico;Slovakia;Spain;Switzerland;United Kingdom;Czech Republic

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

NCT04468165
(ClinicalTrials.gov)

January 2021

8/7/2020

Effectiveness and Safety of Generic Delayed-Release Dimethyl Fumarate (Sclera® or Marovarex ®, Hikma) in Routine Medical Practice in the Treatment of Relapsing-Remitting Multiple Sclerosis in MENA Region

Relapsing Remitting Multiple Sclerosis

Drug: Dimethyl Fumarate (DMF)

Hikma Pharmaceuticals LLC

NULL

Not yet recruiting

18 Years

N/A

All

155

NULL

NCT03101735
(ClinicalTrials.gov)

September 23, 2016

3/3/2017

Study to Assess Resource Utilization and Quality of Life of Patients With RRMS Treated With Tecfidera in Greece

A Real Life, Non-interventional, Multicentre Study to Assess Resource Utilization and Quality of Life of Patients With Relapsing Forms of Multiple Sclerosis Treated With Dimethyl Fumarate in Greece - the FIDELITY Study

RRMS

Drug: Dimethyl Fumarate (DMF)

Genesis Pharma CNS & Specialty

NULL

Active, not recruiting

18 Years

65 Years

All

455

Greece

EUCTR2015-004116-38-SE
(EUCTR)

18/12/2015

20/10/2015

A clincial study comparing the effectiveness of two drugs, named Rituximab and Dimethyl Fumarate (Tecfidera®), for the neurological disease Multiple Sclerosis.

RItuximab versus FUmarate in Newly Diagnosed Multiple Sclerosis – RIFUND-MSA randomized phase 3 study comparing Rituximab with Dimethyl Fumarate in early Relapsing-Remitting Multiple SclerosisObjective: To compare the efficacy of rituximab on the ability to prevent relapses in early RRMS and CIS compared with dimethyl fumarate (DMF), which is an approved first-line medication for RRMS today, using a phase 3 design.Population: Patients with newly diagnosed RRMS or CIS with no more than 10 years disease duration (since diagnosis), 18 – 50 years of age and previously not treated with immunomodulating drugs OR treated with first-line injectables. Patients should display protocol-defined clinical or radiological disease activity during the preceding year before screening for inclusion.Intervention: Treatment with rituximab (Mabthera®) with an initial dose of 1000 mg intravenously (iv) followed by 500 mg iv every six months.Control: Treatment with DMF (Tecfidera®) 240 mg twice daily. The two treatments are randomised in a 1:1 proportion.Outcome: Primary outcome is the relative risk of experiencing a relapse during the two–year period for either compound. As secondary endpoints worsening on neurological disability, magnetic-resonance imaging-defined disease activity and effect on cerebrospinal fluid biomarkers will be analysed. In addition, health-economic evaluations of using rituximab as first-line treatment for RRMS will be performed.

Patients with multiple sclerosis (MS) or patients presenting with symptoms highly suspicious of MS while not completely fulfilling diagnostic criteria, 18 - 50 years of age and no more than 10 years of disease duration (from diagnosis). Patients treated with immunomodulatory drugs or treated with first-line injectable therapies (eg interferons or glatiramer acetate) may be included.;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Trade Name: Mabthera
INN or Proposed INN: RITUXIMAB
Trade Name: Tecfidera
INN or Proposed INN: DIMETHYL FUMARATE
Other descriptive name: DIMETHYL FUMARATE

Department of Clinical Sciences, Danderyd Hospital

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

200

Phase 3

Sweden

NCT02047097
(ClinicalTrials.gov)

November 19, 2013

24/1/2014

Dimethyl Fumarate (DMF) Observational Study

A Multicenter, Global, Observational Study to Collect Information on Safety and to Document the Drug Utilization of Tecfidera™ (Dimethyl Fumarate) When Used in Routine Medical Practice in the Treatment of Multiple Sclerosis (ESTEEM)

Multiple Sclerosis

Drug: dimethyl fumarate

Biogen

NULL

Active, not recruiting

18 Years

N/A

All

5496

United States;Argentina;Australia;Austria;Canada;Czechia;Denmark;France;Germany;Hungary;Ireland;Italy;Netherlands;New Zealand;Norway;Poland;Portugal;Puerto Rico;Slovakia;Spain;Switzerland;United Kingdom;Czech Republic