DDrare: Database of Drug Development for Rare Diseases

17. 多系統萎縮症
［臨床試験数：107，薬物数：153（DrugBank：48），標的遺伝子数：59，標的パスウェイ数：104］

Searched query = "Multiple system atrophy", "MSA-C", "MSA-P", "Olivopontocerebellar atrophy", "OPCA", "Striatonigral degeneration", "Shy-Drager 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 = 1) Inosine 5'-monophosphate; 1-(2-Isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one; 11C-methylreboxetine; 13N-Ammonia; 18F-Dopa; 18F-Dopamne; AFFITOPE® PD01A + Adjuvant; AFFITOPE® PD03A + Adjuvant; AZD3241; AZD3241 100mg; AZD3241 300mg; AZILECT; AZILECT 1 mg comprimidos; Adjuvant without active component; Ammonia; Ampreloxetine; Ampreloxetine chydrochloride; Ampreloxetine hydrochloride; Angiotensin; Angiotensin-(1-7); Anti-Parkinson medication; Atomexetine; Autologous mesenchymal stem cells; Azilect; BHV-3241; BHV-4157; BIIB101; BOTULINUM TOXIN TYPE A; BQ123; Behavioral: BCI-FIT active querying; Behavioral: BCI-FIT adaptive signal modeling; Behavioral: BCI-FIT language modeling; Behavioral: BCI-FIT multi-modal access; Behavioral: Clinical characteristics of AMS patients; Behavioral: Conservative Measures for Orthostatic Hypotension; Behavioral: MOntreal Cognitive Assessment (MoCA); Blood sampling; Botulinum toxin type A; Bq123; Bydureon; CS10BR05; Captopril; Carbidopa; Carbidopa 200mg oral dose; Carbonate; DMI; DOPS; DaTSCAN™ Ioflupane (123I) Injection; DaTscan; Datscan; Desipramine; Desipramine (DMI); Device: Abdominal compression; Device: Deep Brain Stimulation; Device: sham compression; Diagnostic Test: Elecsys (Roche) Abeta42, Ttau and Ptau; Diagnostic Test: Flutemetamol F18 Injection; Diagnostic Test: Lumipulse (Fujirebio) Abeta42, Ttau and Ptau; Diagnostic Test: [18F]-RO6958948; Domperidone; Droxidopa; EGCG; EGCG as putative neuroprotective agent; Emeramide; Entacapone; Entacapone 400mg oral dose; Exenatide; Exenatide Pen Injector [Bydureon]; FLUOXETINE; Fipamezole; Fipamezole hydrochloride; Fipamezole hydrochloride 30 mg oral disintegrating tablets; Fipamezole hydrochloride 60 mg oral disintegrating tablets; Fipamezole hydrochloride 90 mg oral disintegrating tablets; Fludrocortisone; Flumazenil; Fluoxetine; Fluoxétine; Flutemetamol; Flutemetamol F18; GREEN TEA LEAF; Gold; Green Tea; Green tea leaf; Homeostasis Model Assessment of insulin resistance (HOMA); Hydrogen; IUPAC name: 1-(2-Isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one; IVIg; Inosine; Intranasal Insulin; Intravenous immunoglobulin (IVIg); Intravesical injection of Botulinum A toxin; JP-1730/F01; JP-1730/F02; JP-1730/F03; L-Threo DOPS; LITHIUM CARBONATE; LITIO CARBONATO; Levodopa; Levodopa test; Lithium Carbonate; Lithium carbonate; Losartan; Metoprolol; Metoprolol tartrate 50 mg; Midodrine; Modal; N1,N3-BIS(2-MERCAPTOETHYL) ISOPHTHALAMIDE; NBMI; NT 101; Nebivolol; Nebivolol 5 mg; Nitrogen; Normal saline; Other: Abdominal compression; Other: Brain MRI (magnetic resonance imaging); Other: Sham abdominal compression; PROZAC; Procedure: Brain Magnetic Resonance Imaging (MRI); Procedure: determination of objective and subjective pain threshold; Pseudoephedrine; Pseudoephedrine + 480 ml water; Pseudoephedrine + 50 ml water; RASAGILINA MESILATO; Raclopride; Radiation: H215O PET; Radiation: PET (Positron Emission Tomography) Study; Rasagilina; Rasagiline; Rasagiline mesylate; Rifampicin; Riluzole; Rituximab; SAFINAMIDE; Safinamide; Safinamide Methanesulfonate; Safinamide methanesulfonate; Saline; Sildenafil25 mg; Sirolimus; Sirolimus 2 MG; Sunphenon EGCg; TD-9855; Tea leaf; Tllsh2910; Trimethaphan; Ubiquinol; Verdiperstat; Water; Xadago; Xeomin; [18F]FDOPA; [F-18]PBR06

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	NCT04616456 (ClinicalTrials.gov)	December 2020	26/10/2020	Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients	Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients	Multiple System Atrophy;Multiple System Atrophy, Cerebellar Variant;Multiple System Atrophy, Parkinson Variant (Disorder);Multiple System Atrophy (MSA) With Orthostatic Hypotension	Drug: [F-18]PBR06;Drug: Verdiperstat	Brigham and Women's Hospital	Biohaven Pharmaceuticals, Inc.	Not yet recruiting	18 Years	N/A	All	8	Early Phase 1	United States
2	EUCTR2019-001100-38-DE (EUCTR)	07/01/2020	07/10/2019	A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)	Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)	Multiple System Atrophy MedDRA version: 21.1;Level: PT;Classification code 10064060;Term: Multiple system atrophy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: BHV-3241 INN or Proposed INN: Verdiperstat	Biohaven Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	422	Phase 3	United States;France;Austria;Germany;United Kingdom;Italy;China
3	EUCTR2019-001100-38-AT (EUCTR)	20/12/2019	29/10/2019	A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)	Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)	Multiple System Atrophy MedDRA version: 21.1;Level: PT;Classification code 10064060;Term: Multiple system atrophy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: BHV-3241 INN or Proposed INN: Verdiperstat	Biohaven Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	422	Phase 3	United States;France;Austria;Germany;United Kingdom;Italy;China
4	EUCTR2019-001100-38-FR (EUCTR)	13/12/2019	03/10/2019	A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy(M-STAR Study)	Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)	Multiple System Atrophy MedDRA version: 21.1;Level: PT;Classification code 10064060;Term: Multiple system atrophy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: BHV-3241 INN or Proposed INN: Verdiperstat	Biohaven Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	252	Phase 3	United States;France;Austria;Germany;Italy;United Kingdom
5	EUCTR2019-001100-38-GB (EUCTR)	06/12/2019	11/10/2019	A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy(M-STAR Study)	Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)	Multiple System Atrophy MedDRA version: 21.1;Level: PT;Classification code 10064060;Term: Multiple system atrophy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: BHV-3241 INN or Proposed INN: Verdiperstat	Biohaven Pharmaceuticals, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	422	Phase 3	United States;France;Austria;Germany;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
6	NCT03952806 (ClinicalTrials.gov)	July 29, 2019	15/5/2019	Study of BHV-3241 in Subjects With Multiple System Atrophy	A Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy (M-STAR Study)	Multiple System Atrophy	Drug: Verdiperstat;Drug: Placebo	Biohaven Pharmaceuticals, Inc.	NULL	Active, not recruiting	40 Years	80 Years	All	336	Phase 3	United States;Austria;France;Germany;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

NCT04616456
(ClinicalTrials.gov)

December 2020

26/10/2020

Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients

Multiple System Atrophy;Multiple System Atrophy, Cerebellar Variant;Multiple System Atrophy, Parkinson Variant (Disorder);Multiple System Atrophy (MSA) With Orthostatic Hypotension

Drug: [F-18]PBR06;Drug: Verdiperstat

Brigham and Women's Hospital

Biohaven Pharmaceuticals, Inc.

Not yet recruiting

18 Years

N/A

All

Early Phase 1

United States

EUCTR2019-001100-38-DE
(EUCTR)

07/01/2020

07/10/2019

A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)

Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)

Multiple System Atrophy
MedDRA version: 21.1;Level: PT;Classification code 10064060;Term: Multiple system atrophy;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: BHV-3241
INN or Proposed INN: Verdiperstat

Biohaven Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

422

Phase 3

United States;France;Austria;Germany;United Kingdom;Italy;China

EUCTR2019-001100-38-AT
(EUCTR)

20/12/2019

29/10/2019

A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)

Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)

Product Name: BHV-3241
INN or Proposed INN: Verdiperstat

Biohaven Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

422

Phase 3

United States;France;Austria;Germany;United Kingdom;Italy;China

EUCTR2019-001100-38-FR
(EUCTR)

13/12/2019

03/10/2019

A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy(M-STAR Study)

Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)

Product Name: BHV-3241
INN or Proposed INN: Verdiperstat

Biohaven Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

252

Phase 3

United States;France;Austria;Germany;Italy;United Kingdom

EUCTR2019-001100-38-GB
(EUCTR)

06/12/2019

11/10/2019

A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy(M-STAR Study)

Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects with Multiple System Atrophy (M-STAR Study)

Product Name: BHV-3241
INN or Proposed INN: Verdiperstat

Biohaven Pharmaceuticals, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

422

Phase 3

United States;France;Austria;Germany;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

NCT03952806
(ClinicalTrials.gov)

July 29, 2019

15/5/2019

Study of BHV-3241 in Subjects With Multiple System Atrophy

A Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy (M-STAR Study)

Multiple System Atrophy

Drug: Verdiperstat;Drug: Placebo

Biohaven Pharmaceuticals, Inc.

NULL

Active, not recruiting

40 Years

80 Years

All

336

Phase 3

United States;Austria;France;Germany;Italy;United Kingdom