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	EUCTR2017-000858-18-AT (EUCTR)	20/03/2018	05/12/2017	A Phase 3, randomized, controlled study of severe congenital thrombotic thrombocytopenic purpura, with Bax 930.	A phase 3, prospective, randomized, controlled, open-label, multicenter, 2-period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 (rADAMTS13) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP]) - A phase 3, randomized, controlled study of prophylactic and on-demand treatment of cTTP with BAX 930	severe congenital thrombotic thrombocytopenic purpura(cTTP, Upshaw-Schulman Syndrome [USS], hereditary thromboticthrombocytopenic purpura [hTTP]) MedDRA version: 20.0;Level: LLT;Classification code 10043562;Term: Thrombocytopenic purpura, thrombotic;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13 Product Code: BAX930 or SHP655 INN or Proposed INN: apadamtase alfa Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs Trade Name: Octaplas SD Product Name: Octaplas ®LG INN or Proposed INN: Octaplas Other descriptive name: Pooled Plasma (Human), Solvent/Detergent Treated Product Name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13 Product Code: BAX930 or SHP655 INN or Proposed INN: apademtase alfa Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1	Baxalta Innovations GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	68	Phase 3	United States;France;Canada;Poland;Spain;Austria;Germany;United Kingdom;Switzerland;Japan;Italy

No.

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agemin

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EUCTR2017-000858-18-AT
(EUCTR)

20/03/2018

05/12/2017

A Phase 3, randomized, controlled study of severe congenital thrombotic thrombocytopenic purpura, with Bax 930.

A phase 3, prospective, randomized, controlled, open-label, multicenter, 2-period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 (rADAMTS13) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP, Upshaw-Schulman Syndrome [USS], hereditary thrombotic thrombocytopenic purpura [hTTP]) - A phase 3, randomized, controlled study of prophylactic and on-demand treatment of cTTP with BAX 930

severe congenital thrombotic thrombocytopenic purpura(cTTP, Upshaw-Schulman Syndrome [USS], hereditary thromboticthrombocytopenic purpura [hTTP])
MedDRA version: 20.0;Level: LLT;Classification code 10043562;Term: Thrombocytopenic purpura, thrombotic;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
Product Code: BAX930 or SHP655
INN or Proposed INN: apadamtase alfa
Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
Trade Name: Octaplas SD
Product Name: Octaplas ®LG
INN or Proposed INN: Octaplas
Other descriptive name: Pooled Plasma (Human), Solvent/Detergent Treated
Product Name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs 13
Product Code: BAX930 or SHP655
INN or Proposed INN: apademtase alfa
Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1

Baxalta Innovations GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;France;Canada;Poland;Spain;Austria;Germany;United Kingdom;Switzerland;Japan;Italy