DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2018-003775-35-DE (EUCTR)	18/10/2019	30/04/2019	A Phase 2, randomized, placebo-controlled, double-blind study ofrADAMTS-13 (SHP655) in the treatment of patients with aTTP	A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment	acquired thrombotic thrombocytopenic purpura (aTTP) MedDRA version: 20.0;Level: PT;Classification code 10043648;Term: Thrombotic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Recombinant A Disintegrin And Metalloproteinase with Thrombospondin Type-1 Motifs 13 (rADAMTS13) Product Code: SHP 655 (BAX 930) INN or Proposed INN: apadamtase alfa Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13 INN or Proposed INN: apadamtase alfa Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13	Baxalta Innovation GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	33	Phase 2	United States;France;Canada;Spain;Netherlands;Germany;United Kingdom;Switzerland;Italy
2	NCT03922308 (ClinicalTrials.gov)	October 9, 2019	5/4/2019	Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)	A Phase 2, Multicenter, Randomized, Placebo-Controlled, Double-blind Study in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) to Evaluate the Pharmacokinetics,Safety and Efficacy of rADAMTS-13 (SHP655) Administered in Addition to Standard Of Care (SoC) Treatment	Acquired Thrombotic Thrombocytopenic Purpura (aTTP)	Other: Placebo;Drug: SHP655;Other: Standard of Care	Shire	NULL	Recruiting	18 Years	75 Years	All	30	Phase 2	United States;France;Germany;Spain;United Kingdom
3	EUCTR2018-003775-35-GB (EUCTR)	03/07/2019	13/02/2019	A Phase 2, randomized, placebo-controlled, double-blind study of rADAMTS-13 (SHP655) in the treatment of patients with aTTP	A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment - Baxalta SHP655-201	acquired thrombotic thrombocytopenic purpura (aTTP) MedDRA version: 20.0;Level: PT;Classification code 10043648;Term: Thrombotic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: Recombinant A Disintegrin And Metalloproteinase with Thrombospondin Type-1 Motifs 13 (rADAMTS13) Product Code: SHP 655 (BAX 930) INN or Proposed INN: apadamtase alfa Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13 INN or Proposed INN: apadamtase alfa Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13	Baxalta Innovations GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	33	Phase 2	United States;France;Canada;Spain;Germany;Italy;United Kingdom
4	EUCTR2018-003775-35-ES (EUCTR)	13/06/2019	11/06/2019	A Phase 2, randomized, placebo-controlled, double-blind study of rADAMTS-13 (SHP655) in the treatment of patients with aTTP	A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment	acquired thrombotic thrombocytopenic purpura (aTTP) MedDRA version: 20.0;Level: PT;Classification code 10043648;Term: Thrombotic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]		Baxalta Innovations GmbH	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	33	Phase 2	France;United States;Canada;Spain;Netherlands;Germany;Italy;United Kingdom;Switzerland
5	NCT03393975 (ClinicalTrials.gov)	October 20, 2017	5/10/2017	A Study of Prophylactic and On-demand Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP) With BAX 930 (rADAMTS13)	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 Participants With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP) (Upshaw-Schulman Syndrome)	Congenital Thrombotic Thrombocytopenic Purpura	Biological: BAX930;Biological: Standard of care	Baxalta now part of Shire	Baxalta Innovations GmbH, now part of Shire	Recruiting	N/A	70 Years	All	68	Phase 3	United States;Austria;France;Germany;Italy;Japan;Poland;Spain;Switzerland;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

EUCTR2018-003775-35-DE
(EUCTR)

18/10/2019

30/04/2019

A Phase 2, randomized, placebo-controlled, double-blind study ofrADAMTS-13 (SHP655) in the treatment of patients with aTTP

A Phase 2, multicenter, randomized, placebo-controlled, double-blind study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) to evaluate the pharmacokinetics, safety, and efficacy of rADAMTS-13 (SHP655) administered in addition to standard of care (SoC) treatment

acquired thrombotic thrombocytopenic purpura (aTTP)
MedDRA version: 20.0;Level: PT;Classification code 10043648;Term: Thrombotic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: Recombinant A Disintegrin And Metalloproteinase with Thrombospondin Type-1 Motifs 13 (rADAMTS13)
Product Code: SHP 655 (BAX 930)
INN or Proposed INN: apadamtase alfa
Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13
INN or Proposed INN: apadamtase alfa
Other descriptive name: BAX930 - RECOMBINANT HUMAN ADAMTS13

Baxalta Innovation GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;France;Canada;Spain;Netherlands;Germany;United Kingdom;Switzerland;Italy

NCT03922308
(ClinicalTrials.gov)

October 9, 2019

5/4/2019

Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

A Phase 2, Multicenter, Randomized, Placebo-Controlled, Double-blind Study in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) to Evaluate the Pharmacokinetics,Safety and Efficacy of rADAMTS-13 (SHP655) Administered in Addition to Standard Of Care (SoC) Treatment

Acquired Thrombotic Thrombocytopenic Purpura (aTTP)

Other: Placebo;Drug: SHP655;Other: Standard of Care

Shire

NULL

Recruiting

18 Years

75 Years

All

Phase 2

United States;France;Germany;Spain;United Kingdom

EUCTR2018-003775-35-GB
(EUCTR)

03/07/2019

13/02/2019

A Phase 2, randomized, placebo-controlled, double-blind study of rADAMTS-13 (SHP655) in the treatment of patients with aTTP

Baxalta Innovations GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;France;Canada;Spain;Germany;Italy;United Kingdom

EUCTR2018-003775-35-ES
(EUCTR)

13/06/2019

11/06/2019

A Phase 2, randomized, placebo-controlled, double-blind study of rADAMTS-13 (SHP655) in the treatment of patients with aTTP

acquired thrombotic thrombocytopenic purpura (aTTP)
MedDRA version: 20.0;Level: PT;Classification code 10043648;Term: Thrombotic thrombocytopenic purpura;System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Baxalta Innovations GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

France;United States;Canada;Spain;Netherlands;Germany;Italy;United Kingdom;Switzerland

NCT03393975
(ClinicalTrials.gov)

October 20, 2017

5/10/2017

A Study of Prophylactic and On-demand Treatment of Congenital Thrombotic Thrombocytopenic Purpura (cTTP) With BAX 930 (rADAMTS13)

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 Participants With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP) (Upshaw-Schulman Syndrome)

Congenital Thrombotic Thrombocytopenic Purpura

Biological: BAX930;Biological: Standard of care

Baxalta now part of Shire

Baxalta Innovations GmbH, now part of Shire

Recruiting

N/A

70 Years

All

Phase 3

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