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	9
145	ウエスト症候群	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	EUCTR2014-005493-11-FR (EUCTR)	31/08/2015	15/03/2018	Study the action of a single dose of intrathecally-infused rituximab upon cerebro-spinal fluid (CSF) biological targets, assuming that a definitive action upon CSF biological targets would be strongly predictive of a delayed clinical action in patients with Progressive Multiple Sclerosis.	Intrathecal Rituximab in Progressive Multiple Sclerosis - EFFRITE	Patients included in our study suffer from a progressive form of Multiple sclerosis and will be treated by intravenous and intrathecal rituximab, with intravenous methylprednisolone.;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]		Centre Hospitalier de Pau	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	France
2	NCT02398994 (ClinicalTrials.gov)	March 2015	23/3/2015	A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis	A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin (IVIg) Versus Standard Therapy for the Treatment of Transverse Myelitis in Adults and Children	Myelitis, Transverse;Neuromyelitis Optica	Drug: Intravenous Methylprednisolone;Drug: Intravenous Immunoglobulin	Guy's and St Thomas' NHS Foundation Trust	King's College London;Barts and the London School of Medicine and Dentistry;Cardiff University;University College, London;King's College Hospital NHS Trust;Great Ormond Street Hospital for Children NHS Foundation Trust;Barts & The London NHS Trust;Alder Hey Children's NHS Foundation Trust;Walton Centre NHS Foundation Trust;Oxford University Hospitals NHS Trust;Birmingham Children's Hospital NHS Foundation Trust;University Hospital Birmingham NHS Foundation Trust;Cardiff and Vale University Health Board;North Bristol NHS Trust;University Hospitals Bristol NHS Foundation Trust;Central Manchester University Hospitals NHS Foundation Trust;Salford Royal NHS Foundation Trust;University Hospital Southampton NHS Foundation Trust.;Newcastle-upon-Tyne Hospitals NHS Trust;Nottingham University Hospitals NHS Trust;NHS Lothian	Terminated	1 Year	N/A	Both	2	Phase 3	United Kingdom
3	NCT01900093 (ClinicalTrials.gov)	July 2013	11/7/2013	Evaluation of the Response to Acthar Gel Therapy in Patients Who Failed Intravenous Methylprednisolone (IVMP) for MS Relapses	An Open-Label Prospective Proof-Of-Concept Trial to Evaluate the Response to Acthar Gel Therapy in Patients With Multiple Sclerosis (MS) Relapses Who Have Failed To Make an Adequate Recovery After Treatment With High-Dose Intravenous Methylprednisolone	Multiple Sclerosis	Drug: Acthar Gel	Aaron Miller	Mallinckrodt	Recruiting	18 Years	65 Years	All	10	N/A	United States
4	EUCTR2009-013884-21-PL (EUCTR)	20/05/2010	13/01/2010	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome MedDRA version: 14.1;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: PEG-liposomal Prednisolone Sodium Phosphate Product Code: Nanocort INN or Proposed INN: Prednisolone Sodium Phosphate Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE Trade Name: Solu-Medrol INN or Proposed INN: METHYLPREDNISOLONE SODIUM SUCCINATE Other descriptive name: METHYLPREDNISOLONE SODIUM SUCCINATE	Enceladus Pharmaceuticals BV	NULL	Not Recruiting			Female: yes Male: yes	90		Germany;Belgium;Poland
5	NCT01039103 (ClinicalTrials.gov)	December 2009	20/12/2009	Nanocort in Acute Exacerbation of Relapsing-Remitting Multiple Sclerosis (MS)	A Randomized, International, Multi Centre Study to Assess the Efficacy and Safety of Intravenous PEG-liposomal Prednisolone Sodium Phosphate (Nanocort®) vs Intravenous Methylprednisolone (Solu-Medrol®) Treatment in Patients With Acute Exacerbation of Relapsing-remitting Multiple Sclerosis or in Patients With Clinically Isolated Syndrome (CIS)	Acute Exacerbation of Remitting Relapsing Multiple Sclerosis;Clinically Isolated Syndrome	Drug: PEG-liposomal prednisolone sodium phosphate;Drug: Methylprednisolone	Galapagos NV	NULL	Terminated	18 Years	65 Years	Both	15	Phase 2	Belgium;Germany;Poland
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
6	EUCTR2009-013884-21-DE (EUCTR)	18/11/2009	09/09/2009	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome MedDRA version: 14.0;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: PEG-liposomal Prednisolone Sodium Phosphate Product Code: Nanocort INN or Proposed INN: Prednisolone Sodium Phosphate Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE Trade Name: Solu-Medrol INN or Proposed INN: METHYLPREDNISOLONE SODIUM SUCCINATE Other descriptive name: METHYLPREDNISOLONE SODIUM SUCCINATE	Enceladus Pharmaceuticals BV	NULL	Not Recruiting			Female: yes Male: yes	90		Poland;Belgium;Germany
7	EUCTR2009-013884-21-BE (EUCTR)	13/11/2009	10/09/2009	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)	Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome MedDRA version: 12.0;Level: LLT;Classification code 10028245;Term: Multiple sclerosis	Product Name: PEG-liposomal Prednisolone Sodium Phosphate Product Code: Nanocort INN or Proposed INN: Prednisolone Sodium Phosphate Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE Trade Name: Solu-Medrol INN or Proposed INN: METHYLPREDNISOLONE SODIUM SUCCINATE Other descriptive name: METHYLPREDNISOLONE SODIUM SUCCINATE	Galapagos NV	NULL	Not Recruiting			Female: yes Male: yes	90	Phase 2	Poland;Belgium;Germany
8	NCT00947895 (ClinicalTrials.gov)	October 2009	2/7/2009	Efficacy Study of Adrenocorticotropin Hormone to Treat Multiple Sclerosis (MS) Relapses After Sub-responding to an Initial 3 Day Course of Intravenous (IV) Methylprednisolone	Relative Efficacy of Repeat Course of Intravenous methyLprednisolone and Intramuscular ACTH in the Treatment of Acute Relapse of Multiple Sclerosis After Sub Response to Initial Course of Intravenous Methylprednisolone (RECLAIM): a Single Center Pilot Study	Multiple Sclerosis	Drug: Methylprednisolone;Drug: ACTH;Other: IV placebo;Other: IM placebo	Neurologique Foundation, Inc.	Mallinckrodt	Terminated	18 Years	N/A	Both	30	Phase 2;Phase 3	United States
9	NCT00112034 (ClinicalTrials.gov)	June 2003	27/5/2005	AVONEX® Combination Trial - ACT	A Multi-Center, Randomized, Blinded, Parallel-Group Study of AVONEX Compared With AVONEX in Combination With Oral Methotrexate, Intravenous Methylprednisolone, or Both in Subjects With Relapsing Remitting MS Who Have Breakthrough Disease on AVONEX Monotherapy.	Multiple Sclerosis, Relapsing-Remitting	Drug: Methotrexate;Drug: IV methylprednisolone	Biogen Idec	NULL	Completed	18 Years	55 Years	Both	350	Phase 4	United States

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

EUCTR2014-005493-11-FR
(EUCTR)

31/08/2015

15/03/2018

Study the action of a single dose of intrathecally-infused rituximab upon cerebro-spinal fluid (CSF) biological targets, assuming that a definitive action upon CSF biological targets would be strongly predictive of a delayed clinical action in patients with Progressive Multiple Sclerosis.

Intrathecal Rituximab in Progressive Multiple Sclerosis - EFFRITE

Patients included in our study suffer from a progressive form of Multiple sclerosis and will be treated by intravenous and intrathecal rituximab, with intravenous methylprednisolone.;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Centre Hospitalier de Pau

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

France

NCT02398994
(ClinicalTrials.gov)

March 2015

23/3/2015

A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis

A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin (IVIg) Versus Standard Therapy for the Treatment of Transverse Myelitis in Adults and Children

Myelitis, Transverse;Neuromyelitis Optica

Drug: Intravenous Methylprednisolone;Drug: Intravenous Immunoglobulin

Guy's and St Thomas' NHS Foundation Trust

King's College London;Barts and the London School of Medicine and Dentistry;Cardiff University;University College, London;King's College Hospital NHS Trust;Great Ormond Street Hospital for Children NHS Foundation Trust;Barts & The London NHS Trust;Alder Hey Children's NHS Foundation Trust;Walton Centre NHS Foundation Trust;Oxford University Hospitals NHS Trust;Birmingham Children's Hospital NHS Foundation Trust;University Hospital Birmingham NHS Foundation Trust;Cardiff and Vale University Health Board;North Bristol NHS Trust;University Hospitals Bristol NHS Foundation Trust;Central Manchester University Hospitals NHS Foundation Trust;Salford Royal NHS Foundation Trust;University Hospital Southampton NHS Foundation Trust.;Newcastle-upon-Tyne Hospitals NHS Trust;Nottingham University Hospitals NHS Trust;NHS Lothian

Terminated

1 Year

N/A

Both

Phase 3

United Kingdom

NCT01900093
(ClinicalTrials.gov)

July 2013

11/7/2013

Evaluation of the Response to Acthar Gel Therapy in Patients Who Failed Intravenous Methylprednisolone (IVMP) for MS Relapses

An Open-Label Prospective Proof-Of-Concept Trial to Evaluate the Response to Acthar Gel Therapy in Patients With Multiple Sclerosis (MS) Relapses Who Have Failed To Make an Adequate Recovery After Treatment With High-Dose Intravenous Methylprednisolone

Multiple Sclerosis

Drug: Acthar Gel

Aaron Miller

Mallinckrodt

Recruiting

18 Years

65 Years

All

N/A

United States

EUCTR2009-013884-21-PL
(EUCTR)

20/05/2010

13/01/2010

A randomized, international, multi centre study to assess the efficacy and safety of intravenous PEG-liposomal prednisolone sodium phosphate (Nanocort®) vs intravenous methylprednisolone (Solu-Medrol®) treatment in patients with acute exacerbation of relapsing-remitting Multiple Sclerosis or in patients with clinically isolated syndrome (CIS)

Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome
MedDRA version: 14.1;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: PEG-liposomal Prednisolone Sodium Phosphate
Product Code: Nanocort
INN or Proposed INN: Prednisolone Sodium Phosphate
Other descriptive name: PREDNISOLONE SODIUM PHOSPHATE
Trade Name: Solu-Medrol
INN or Proposed INN: METHYLPREDNISOLONE SODIUM SUCCINATE
Other descriptive name: METHYLPREDNISOLONE SODIUM SUCCINATE

Enceladus Pharmaceuticals BV

NULL

Not Recruiting

Female: yes
Male: yes

Germany;Belgium;Poland

NCT01039103
(ClinicalTrials.gov)

December 2009

20/12/2009

Nanocort in Acute Exacerbation of Relapsing-Remitting Multiple Sclerosis (MS)

A Randomized, International, Multi Centre Study to Assess the Efficacy and Safety of Intravenous PEG-liposomal Prednisolone Sodium Phosphate (Nanocort®) vs Intravenous Methylprednisolone (Solu-Medrol®) Treatment in Patients With Acute Exacerbation of Relapsing-remitting Multiple Sclerosis or in Patients With Clinically Isolated Syndrome (CIS)

Acute Exacerbation of Remitting Relapsing Multiple Sclerosis;Clinically Isolated Syndrome

Drug: PEG-liposomal prednisolone sodium phosphate;Drug: Methylprednisolone

Galapagos NV

NULL

Terminated

18 Years

65 Years

Both

Phase 2

Belgium;Germany;Poland

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

EUCTR2009-013884-21-DE
(EUCTR)

18/11/2009

09/09/2009

Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome
MedDRA version: 14.0;Level: PT;Classification code 10028245;Term: Multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Enceladus Pharmaceuticals BV

NULL

Not Recruiting

Female: yes
Male: yes

Poland;Belgium;Germany

EUCTR2009-013884-21-BE
(EUCTR)

13/11/2009

10/09/2009

Patients with acute exacerbation of Relapsing-Remitting Multiple Sclerosis or Clinically Isolated Syndrome
MedDRA version: 12.0;Level: LLT;Classification code 10028245;Term: Multiple sclerosis

Galapagos NV

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Poland;Belgium;Germany

NCT00947895
(ClinicalTrials.gov)

October 2009

2/7/2009

Efficacy Study of Adrenocorticotropin Hormone to Treat Multiple Sclerosis (MS) Relapses After Sub-responding to an Initial 3 Day Course of Intravenous (IV) Methylprednisolone

Relative Efficacy of Repeat Course of Intravenous methyLprednisolone and Intramuscular ACTH in the Treatment of Acute Relapse of Multiple Sclerosis After Sub Response to Initial Course of Intravenous Methylprednisolone (RECLAIM): a Single Center Pilot Study

Multiple Sclerosis

Drug: Methylprednisolone;Drug: ACTH;Other: IV placebo;Other: IM placebo

Neurologique Foundation, Inc.

Mallinckrodt

Terminated

18 Years

N/A

Both

Phase 2;Phase 3

United States

NCT00112034
(ClinicalTrials.gov)

June 2003

27/5/2005

AVONEX® Combination Trial - ACT

A Multi-Center, Randomized, Blinded, Parallel-Group Study of AVONEX Compared With AVONEX in Combination With Oral Methotrexate, Intravenous Methylprednisolone, or Both in Subjects With Relapsing Remitting MS Who Have Breakthrough Disease on AVONEX Monotherapy.

Multiple Sclerosis, Relapsing-Remitting

Drug: Methotrexate;Drug: IV methylprednisolone

Biogen Idec

NULL

Completed

18 Years

55 Years

Both

350

Phase 4

United States