DDrare: Database of Drug Development for Rare Diseases

64. 血栓性血小板減少性紫斑病
［臨床試験数：74，薬物数：76（DrugBank：19），標的遺伝子数：15，標的パスウェイ数：57］

Searched query = "Thrombotic thrombocytopenic purpura", "TTP", "Upshaw-Schulman syndrome", "USS"
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 = ALX-0081; ARC 1779; ARC1779; ARC1779 Injection; Acetate; Anfibatide; Anti-von Willebrand Factor Nanobody; Anti-von Willebrand Factor Nanobody, Caplacizumab; Anti-von Willebrand Factor Nanobody, INN = Caplacizumab; Apadamtase alfa; Apademtase alfa; BAX930; BAX930 - RECOMBINANT HUMAN ADAMTS13; BAX930 or SHP655; CABLIVI; CAPLACIZUMAB; Cablivi; Caplacizumab; Caplacizumab (ALX-0081); Caplacizumab (an anti-von Willebrand Factor Nanobody ALX-0081); Caplacizumab (an anti-von Willebrand Factor Nanobody); Corticosteroid treatment (Methylprednisolone or prednisolone); Corticosteroids; Cryosupernatant plasma; Cyclosporine; Danazol; Desmopressin; Desmopressin acetate; Dexamethasone; HMED-IdeS; IDEC-C2B8, Ro 45-2294; IdeS; IdeS (0.25 mg/kg); IdeS (0.50 mg/kg); Immunosuppressive treatment (eg, rituximab); MABTHERA; MASP-2 antibody; MabThera; Mabthera; Magnesium; Methylprednisolone; Minirin; OMS721; OMS721 100 mg/ml Injection solution, OMS00620646, OMS620646, MASP-2 Antibody; OMS721 185 mg/ml Injection solution, OMS00620646, OMS620646, MASP-2 Antibody; Octaplas; Octaplas SD; Octaplas infusion; OctaplasLG®; Octaplas®LG; Octostim; Other: Standard of Care; Plasma exchange (PE); Pooled Plasma (Human), Solvent/Detergent Treated; Prednisolone; Prednisone; Procedure: Plasma exchange; RADAMTS-13; Recombinant A Disintegrin And Metalloproteinase with Thrombospondin Type-1 Motifs 13 (rADAMTS13); Recombinant A Disintegrin and Metalloproteinase Thrombospondin Type-1 Motifs 13; Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1; Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs; Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13; Recombinant ADAMTS13; Rituximab; Romiplostim; SHP 655 (BAX 930); SHP655; Standard Plasma; Standard of care; Standrd of Care treatment; Sulfate; Sulfate, Magnesium; TAK-755 orBAX930 or SHP655; Uniplas; Von willebrand factor

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	NCT00726544 (ClinicalTrials.gov)	December 2008	30/7/2008	Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy	A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy	Thrombotic Microangiopathy;Thrombotic Thrombocytopenic Purpura	Drug: ARC 1779 Placebo;Drug: ARC1779 Injection	Archemix Corp.	NULL	Terminated	18 Years	75 Years	Both	100	Phase 2	United States;Austria;Canada;Italy;United Kingdom
2	EUCTR2007-004371-19-AT (EUCTR)	20/11/2007	12/12/2007	A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients with von Willebrand Factor-Related Platelet Function Disorders	A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients with von Willebrand Factor-Related Platelet Function Disorders	von Willebrand Factor- related platelet function disorders : thrombotic thrombocytopenic purpura(TTP) in remission, acute TTP, and von Willebrand Disease Type- 2b (vWD-2b). MedDRA version: 9.1;Level: LLT;Classification code 10037563;Term: Purpura thrombopenic thrombotic MedDRA version: 9.1;Classification code 10047715;Term: Von Willebrand's disease	Product Name: ARC1779 Injection Product Code: ARC1779 Other descriptive name: ARC 1779 Trade Name: Octostim Product Name: desmopressin acetate Product Code: desmopressin acetate INN or Proposed INN: desmopressin acetate Trade Name: Minirin Product Name: desmopressin Product Code: desmopressin INN or Proposed INN: desmopressin acetate Product Name: ARC1779 Injection Product Code: ARC1779 Other descriptive name: ARC 1779	Archemix Corp.	NULL	Not Recruiting			Female: yes Male: yes	28	Phase 2	Austria

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

NCT00726544
(ClinicalTrials.gov)

December 2008

30/7/2008

Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy

A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy

Thrombotic Microangiopathy;Thrombotic Thrombocytopenic Purpura

Drug: ARC 1779 Placebo;Drug: ARC1779 Injection

Archemix Corp.

NULL

Terminated

18 Years

75 Years

Both

100

Phase 2

United States;Austria;Canada;Italy;United Kingdom

EUCTR2007-004371-19-AT
(EUCTR)

20/11/2007

12/12/2007

A Phase 2 Pilot Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ARC1779 Injection in Patients with von Willebrand Factor-Related Platelet Function Disorders

von Willebrand Factor- related platelet function disorders : thrombotic thrombocytopenic purpura(TTP) in remission, acute TTP, and von Willebrand Disease Type- 2b (vWD-2b).
MedDRA version: 9.1;Level: LLT;Classification code 10037563;Term: Purpura thrombopenic thrombotic
MedDRA version: 9.1;Classification code 10047715;Term: Von Willebrand's disease

Product Name: ARC1779 Injection
Product Code: ARC1779
Other descriptive name: ARC 1779
Trade Name: Octostim
Product Name: desmopressin acetate
Product Code: desmopressin acetate
INN or Proposed INN: desmopressin acetate
Trade Name: Minirin
Product Name: desmopressin
Product Code: desmopressin
INN or Proposed INN: desmopressin acetate
Product Name: ARC1779 Injection
Product Code: ARC1779
Other descriptive name: ARC 1779

Archemix Corp.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Austria