DDrare: Database of Drug Development for Rare Diseases

61. 自己免疫性溶血性貧血
［臨床試験数：90，薬物数：72（DrugBank：23），標的遺伝子数：19，標的パスウェイ数：147］

Searched query = "Autoimmune hemolytic anemia", "AIHA", "WAIHA", "Cold agglutinin disease", "CAD", "Paroxysmal cold hemoglobinuria", "MAIHA", "Evans 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 = ALXN1830; ANX005; APL-2; Acalabrutinib; BENDAMUSTINE HYDROCHLORIDE; BIV009; BIVV009; BIVV020; Bendamustine; Bendamustine, Rituximab; Blood sample; Blood samples; Bortezomib; CINRYZE® (C1 esterase inhibitor [human]); COMPLEMENT C1 ESTERASE INHIBITOR; Ciclosporin; Cinryze; Combination of a single dose anti-CD20 antibody and bortezomib; Complement C1 esterase inhibitor; EFALIZUMAB; Eculizumab; Efalizumab; Fludarabine; Fostamatinib; Fostamatinib 150 mg bid; Fostamatinib Disodium; Fostamatinib disodium; INCB050465; INCB050465 1 mg; INCB050465 2.5 mg; INCB050465 HYDROCHLORIDE; Ibrutinib; Interleukine-2; Isatuximab; Isatuximab SAR650984; KZR-616; Levact; Levamisole; M281; MabThera; Ninlaro; ORILANOLIMAB; Orilanolimab; Parsaclisib; Prednisolone; Prednisolone + mabthera; Prednisone; Prednisone, low dose rituximab; Procedure: spleen sample; R788, fostamatinib disodium hexahydrate (in USPI/FPI); R935788; RITUXIMAB; RVT-1401; RVT-1401 340 mg/weekly; RVT-1401 680 mg/weekly; Rapamycin; Raptiva; Raptiva 100 mg/ml powder and solvent for solution for injection; Rituximab; Rituximab (MABTHERA® or RITUXAN®).; Rituximab (Mabthera®); SYNT001; Sirolimus; Soliris; Soliris (R); Sutimlimab; Sutimlimab (BIVV009); TNT009; Tacrolimus; Urine sample; VELCADE*EV 1FL 3,5MG 1MG/ML; Velcade

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	EUCTR2017-003363-35-IT (EUCTR)	05/07/2018	04/11/2020	Clinical study to assess the safety, tolerability, efficacy and pharmacokinetics of APL-2 in patients with Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)	An open label, prospective, study to assess the safety, tolerability, efficacy and pharmacokinetics of APL-2 in patients with Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD). - Treatment of wAIHA with APL-2	Patients affected by Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD) MedDRA version: 20.0;Level: LLT;Classification code 10002045;Term: Anaemia haemolytic;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: APL-2 Product Name: APL-2 Product Code: APL-2 INN or Proposed INN: APL-2	APELLIS PHARMACEUTCIALS, INC.	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	United States;Italy
2	NCT03226678 (ClinicalTrials.gov)	August 31, 2017	7/7/2017	Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)	An Open Label, Prospective, Study to Assess the Safety, Tolerability, Efficacy and Pharmacokinetics of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)	Warm Autoimmune Hemolytic Anemia;Cold Agglutinin Disease	Drug: APL-2	Apellis Pharmaceuticals, Inc.	NULL	Active, not recruiting	18 Years	N/A	All	24	Phase 2	United States

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

EUCTR2017-003363-35-IT
(EUCTR)

05/07/2018

04/11/2020

Clinical study to assess the safety, tolerability, efficacy and pharmacokinetics of APL-2 in patients with Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

An open label, prospective, study to assess the safety, tolerability, efficacy and pharmacokinetics of APL-2 in patients with Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD). - Treatment of wAIHA with APL-2

Patients affected by Warm Antibody Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)
MedDRA version: 20.0;Level: LLT;Classification code 10002045;Term: Anaemia haemolytic;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: APL-2
Product Name: APL-2
Product Code: APL-2
INN or Proposed INN: APL-2

APELLIS PHARMACEUTCIALS, INC.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Italy

NCT03226678
(ClinicalTrials.gov)

August 31, 2017

7/7/2017

Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

An Open Label, Prospective, Study to Assess the Safety, Tolerability, Efficacy and Pharmacokinetics of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)

Warm Autoimmune Hemolytic Anemia;Cold Agglutinin Disease

Drug: APL-2

Apellis Pharmaceuticals, Inc.

NULL

Active, not recruiting

18 Years

N/A

All

Phase 2

United States