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	EUCTR2020-003348-10-GB (EUCTR)	17/11/2020	14/09/2020	A continuation study to evaluate the prophylactic and on demand treatment of congenital Thrombotic Thrombocytopenic Purpura (cTTP) with the drug TAK-755 (rADAMTS-13, also known as BAX 930/SHP655)	A Phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also known as BAX 930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura) - Baxalta TAK-755-3002 Continuation study to 281102	congenital Thrombotic thrombocytopenic purpura (TTP);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Recombinant A Disintegrin and Metalloproteinase Thrombospondin Type-1 Motifs 13 Product Code: TAK-755 orBAX930 or SHP655 INN or Proposed INN: Apadamtase alfa Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs INN or Proposed INN: Apadamtase alfa Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs	Baxalta Innovations GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	88	Phase 3	United States;Portugal;Serbia;Taiwan;Thailand;Spain;Ukraine;Israel;Russian Federation;Colombia;Switzerland;Italy;India;France;Denmark;Australia;Netherlands;China;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Germany;Norway;Japan;Sweden

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

EUCTR2020-003348-10-GB
(EUCTR)

17/11/2020

14/09/2020

A continuation study to evaluate the prophylactic and on demand treatment of congenital Thrombotic Thrombocytopenic Purpura (cTTP) with the drug TAK-755 (rADAMTS-13, also known as BAX 930/SHP655)

A Phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also known as BAX 930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura) - Baxalta TAK-755-3002 Continuation study to 281102

congenital Thrombotic thrombocytopenic purpura (TTP);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: Recombinant A Disintegrin and Metalloproteinase Thrombospondin Type-1 Motifs 13
Product Code: TAK-755 orBAX930 or SHP655
INN or Proposed INN: Apadamtase alfa
Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs
INN or Proposed INN: Apadamtase alfa
Other descriptive name: Recombinant A Disintegrin and Metalloproteinase with Thrombospondin Type-1 Motifs

Baxalta Innovations GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Portugal;Serbia;Taiwan;Thailand;Spain;Ukraine;Israel;Russian Federation;Colombia;Switzerland;Italy;India;France;Denmark;Australia;Netherlands;China;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Hungary;Canada;Brazil;Belgium;Poland;Germany;Norway;Japan;Sweden