DDrare: Database of Drug Development for Rare Diseases

ID	Disease name (Link within this page)	Number of trials
60	Aplastic anemia	2
63	Idiopathic thrombocytopenic purpura	10

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	ChiCTR1800019669	2019-01-01	2018-11-21	A randomized, open-label, single-center controlled trial for the efficacy and safety of recombinant human thrombopoietin injection in the treatment of severe Aplastic Anemia with thrombocytopenia	A randomized, open-label, single-center controlled trial for the efficacy and safety of recombinant human thrombopoietin injection in the treatment of severe Aplastic Anemia with thrombocytopenia	Aplastic anemia,Thrombocytopenia	Group 1:Add rhTPO, 300U/kg qd after ATG treatment for 8 weeks;Group 2:Add rhTPO, 300U/kg qod after ATG treatment for 8 weeks;	Institute of Hematology, People's Hospital, Peking University	NULL	Pending			Both	Group 1:30;Group 2:30;		China
2	NCT02857530 (ClinicalTrials.gov)	August 2016	26/7/2016	Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia	Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia	Anemia, Aplastic	Drug: rhTPO;Drug: placebo	Tianjin Medical University General Hospital	NULL	Not yet recruiting	6 Years	60 Years	Both	250	Phase 2	China

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	ChiCTR1900027735	2020-01-01	2019-11-24	Clinical study for high-dose dexamethasone combined with versus without human recombinant thrombopoietin (rhTPO) in the treatment of newly diagnosed primary immune thrombocytopenia (ITP)	Clinical study for high-dose dexamethasone combined with versus without human recombinant thrombopoietin (rhTPO) in the treatment of newly diagnosed primary immune thrombocytopenia (ITP)	Adult newly diagnosed immune thrombocytopenia	Study group:Dexamethasone 40mg/d×4d, recombinant human thrombopoietin (rhTPO) 300U/Kg subcutaneous injection, once/d×14d, if PLT>100×109/L or PLT increase =50×109/L during treatment period RhTPO drug was discontinued; rhTPO and dexamethasone were replaced with prednisone 0.5mg~1mg/kg once a day, 0.2mg/kg per week, and the drug was stopped wit;Control group:Dexamethasone 40mg/d×4d, if the PLT>100×10^9/L or PLT increase =50×10^9/L during the treatment period, dexamethasone was replaced with prednisone 0.5mg~ 1mg/kg orally every day, reduce 0.2mg/kg per week, and quickly reduce the dose within 6~8 weeks.;	Department of Hematology, Anhui Provincial Hospital	NULL	Pending	18	80	Both	Study group:30;Control group:30;	Phase 4	China
2	NCT03771378 (ClinicalTrials.gov)	January 16, 2019	7/12/2018	Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)	Efficacy and Safety of rhTPO and Eltrombopag in the Treatment of Chinese Adults With Primary Immune Thrombocytopenia (ITP):a Multicenter, Double-blind, Randomized, Controlled Trial	Primary Immune Thrombocytopenia	Drug: rhTPO;Drug: eltrombopag	Wuhan Union Hospital, China	The First Affiliated Hospital of Nanchang University;The Third Xiangya Hospital of Central South University;The First People's Hospital of Yuhang District;Xiangyang Central Hospital	Recruiting	18 Years	75 Years	All	96	Phase 4	China
3	NCT04089267 (ClinicalTrials.gov)	December 2016	10/9/2019	Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment of Patients With Primary Immune Thrombocytopenia (ITP)	Multicenter, Randomized, Open, Parallel Controlled Trials Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment of Patients With Primary Immune Thrombocytopenia (ITP)	ITP	Drug: TPO	Shenyang Sunshine Pharmaceutical Co., LTD.	NULL	Completed	18 Years	N/A	All	288	Phase 4	China
4	ChiCTR-INR-16009363	2016-10-13	2016-10-10	'Yu Xue Qi Huo' theory based integrated Traditional Chinese and Western Medicine in the diagnosis and treatment of stratified relapsed and refractory primary immune thrombocytopenia	'Yu Xue Qi Huo' theory based integrated Traditional Chinese and Western Medicine in the diagnosis and treatment of stratified relapsed and refractory primary immune thrombocytopenia	Immune Thrombocytopenia	Study Group A:Yangyin Traditional Chinese Medicine: brew the TCM granules in warm water, 1 pack bid p.o. 1 hour after meal for 6 months continuously;Study Group A:Cyclosporin A capsules: 5mg/kg/d p.o., adjusted according to the serum CsA concentration;Control group B:Traditional Chinese Medicine for control group;Control group B:Cyclosporin A capsules: 5mg/kg/d p.o., adjusted according to the serum CsA concentration ;Study Group C:Yangyin Traditional Chinese Medicine: brew the TCM granules in warm water, 1 pack bid p.o. 1 hour after meal for 6 months continuously ;Study Group C:rhTPO: 1ug/kg/d qd for 14 days continuously, ih, until platelet count reaches 100×10^9/L;Control Group D:Traditional Chinese Medicine for control group;Control Group D:rhTPO: 1ug/kg/d qd for 14 days continuously, ih, until platelet count reaches 100×10^9/L ;	Ruijin Hospital affiliated to Shanghai Jiaotong University School of Medicine	NULL	Recruiting	18	75	Both	Study Group A:55;Study Group A:55;Control group B:55;Control group B:55;Study Group C:45;Study Group C:45;Control Group D:45;Control Group D:45;		China
5	NCT02139501 (ClinicalTrials.gov)	May 2014	13/5/2014	An Investigation of rhTPO With Different Frequencies in the Management of ITP	A Randomized Study to Evaluate the Efficacy and Safety of Different Doses and Frequencies of Recombinant Human Thrombopoietin (rhTPO) in the Management of Primary Immune Thrombocytopenia (ITP)	Immune Thrombocytopenia	Drug: Recombinant Human Thrombopoietin (rhTPO)	Shandong University	The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital	Completed	18 Years	60 Years	Both	48	Phase 3	China
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
6	NCT01734044 (ClinicalTrials.gov)	July 2012	21/11/2012	rhTPO Combining Dexamethasone Versus High-dose Dexamethasone for Initial Treatment of ITP	A Multicentre Investigation of Recombinant Human Thrombopoietin (rhTPO) Combining Dexamethasone Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)	Purpura;Idiopathic Thrombocytopenic Purpura	Drug: recombinant human thrombopoietin (rhTPO); dexamethasone;Drug: Dexamethasone	Shandong University	The Affiliated Hospital of the Chinese Academy of Military Medical Sciences;Anhui Medical University;The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital;Shenzhen Second People's Hospital;China Medical University, China;Zhejiang Provincial Hospital of TCM	Completed	18 Years	75 Years	Both	158	Phase 3	China
7	NCT01734057 (ClinicalTrials.gov)	July 2012	21/11/2012	Recombinant Human Thrombopoietin (rhTPO) Combining Rituximab Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)	A Multicentre Investigation of Recombinant Human Thrombopoietin (rhTPO) Combining Rituximab Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)	Purpura;Idiopathic Thrombocytopenic Purpura	Drug: recombinant human thrombopoietin (rhTPO); rituximab;Drug: Dexamethasone	Shandong University	The Affiliated Hospital of the Chinese Academy of Military Medical Sciences;Anhui Medical University;The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital;Shenzhen Second People's Hospital;China Medical University, China;Zhejiang Provincial Hospital of TCM	Withdrawn	18 Years	75 Years	Both	0	Phase 3	China
8	NCT01525836 (ClinicalTrials.gov)	May 2011	31/1/2012	rhTPO Combining Rituximab Versus Low-dose Rituximab in Management of ITP	Recombinant Human Thrombopoietin in Combination With Rituximab Versus Low-dose Rituximab for the Treatment of Refractory ITP.	Purpura;Idiopathic Thrombocytopenic Purpura	Drug: rituximab; recombinant human thrombopoietin (rhTPO);Drug: Rituximab	Shandong University	The Affiliated Hospital of the Chinese Academy of Military Medical Sciences;The First Affiliated Hospital of Anhui Medical University;The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital;Shenzhen Second People's Hospital;First Hospital of China Medical University	Completed	18 Years	72 Years	Both	123	Phase 3	China
9	NCT01805648 (ClinicalTrials.gov)	February 2011	22/2/2013	Efficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With ITP	A Multicenter, Single-Arm, Open Label Study Evaluating the Efficacy and Safety of Maintenance Treatment With Recombinant Human Thrombopoietin in Thrombocytopenic Subjects With Immune Thrombocytopenia	Thrombocytopenia;Immune Thrombocytopenia;Idiopathic Thrombocytopenic Purpura	Drug: rhTPO	Peking Union Medical College	Shenyang Sunshine Pharmaceutical Co., LTD.	Recruiting	14 Years	70 Years	Both	120	Phase 3	China
10	NCT01506414 (ClinicalTrials.gov)	June 2009	16/11/2011	Recombinant Human Thrombopoietin in Combination With Rituximab in Immune Thrombocytopenia (ITP)	A Multicentre Investigation of Recombinant Human Thrombopoietin (Rh-TPO) Combine With Low-dose Rituximab in Management of Steroid-Resistant/Relapsed Immune Thrombocytopenia (ITP)	Purpura;Idiopathic Thrombocytopenic Purpura	Drug: rhTPO in combination with Rituximab	Ming Hou	Peking Union Medical College Hospital;Chinese Academy of Medical Sciences;First Affiliated Hospital, Sun Yat-Sen University;West China Hospital;Shandong Provincial Hospital;Wuhan Union Hospital, China;Zhejiang University	Completed	16 Years	75 Years	Both	91	Phase 3	China

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

ChiCTR1900027735

2020-01-01

2019-11-24

Clinical study for high-dose dexamethasone combined with versus without human recombinant thrombopoietin (rhTPO) in the treatment of newly diagnosed primary immune thrombocytopenia (ITP)

Adult newly diagnosed immune thrombocytopenia

Study group:Dexamethasone 40mg/d×4d, recombinant human thrombopoietin (rhTPO) 300U/Kg subcutaneous injection, once/d×14d, if PLT>100×109/L or PLT increase =50×109/L during treatment period RhTPO drug was discontinued; rhTPO and dexamethasone were replaced with prednisone 0.5mg~1mg/kg once a day, 0.2mg/kg per week, and the drug was stopped wit;Control group:Dexamethasone 40mg/d×4d, if the PLT>100×10^9/L or PLT increase =50×10^9/L during the treatment period, dexamethasone was replaced with prednisone 0.5mg~ 1mg/kg orally every day, reduce 0.2mg/kg per week, and quickly reduce the dose within 6~8 weeks.;

Department of Hematology, Anhui Provincial Hospital

NULL

Pending

Both

Study group:30;Control group:30;

Phase 4

China

NCT03771378
(ClinicalTrials.gov)

January 16, 2019

7/12/2018

Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)

Efficacy and Safety of rhTPO and Eltrombopag in the Treatment of Chinese Adults With Primary Immune Thrombocytopenia (ITP):a Multicenter, Double-blind, Randomized, Controlled Trial

Primary Immune Thrombocytopenia

Drug: rhTPO;Drug: eltrombopag

Wuhan Union Hospital, China

The First Affiliated Hospital of Nanchang University;The Third Xiangya Hospital of Central South University;The First People's Hospital of Yuhang District;Xiangyang Central Hospital

Recruiting

18 Years

75 Years

All

Phase 4

China

NCT04089267
(ClinicalTrials.gov)

December 2016

10/9/2019

Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment of Patients With Primary Immune Thrombocytopenia (ITP)

Multicenter, Randomized, Open, Parallel Controlled Trials Evaluating the Efficacy and Safety of Different Human Thrombopoietin (rhTPO) Regimens in the Treatment of Patients With Primary Immune Thrombocytopenia (ITP)

ITP

Drug: TPO

Shenyang Sunshine Pharmaceutical Co., LTD.

NULL

Completed

18 Years

N/A

All

288

Phase 4

China

ChiCTR-INR-16009363

2016-10-13

2016-10-10

'Yu Xue Qi Huo' theory based integrated Traditional Chinese and Western Medicine in the diagnosis and treatment of stratified relapsed and refractory primary immune thrombocytopenia

Immune Thrombocytopenia

Study Group A:Yangyin Traditional Chinese Medicine: brew the TCM granules in warm water, 1 pack bid p.o. 1 hour after meal for 6 months continuously;Study Group A:Cyclosporin A capsules: 5mg/kg/d p.o., adjusted according to the serum CsA concentration;Control group B:Traditional Chinese Medicine for control group;Control group B:Cyclosporin A capsules: 5mg/kg/d p.o., adjusted according to the serum CsA concentration ;Study Group C:Yangyin Traditional Chinese Medicine: brew the TCM granules in warm water, 1 pack bid p.o. 1 hour after meal for 6 months continuously ;Study Group C:rhTPO: 1ug/kg/d qd for 14 days continuously, ih, until platelet count reaches 100×10^9/L;Control Group D:Traditional Chinese Medicine for control group;Control Group D:rhTPO: 1ug/kg/d qd for 14 days continuously, ih, until platelet count reaches 100×10^9/L ;

Ruijin Hospital affiliated to Shanghai Jiaotong University School of Medicine

NULL

Recruiting

Both

Study Group A:55;Study Group A:55;Control group B:55;Control group B:55;Study Group C:45;Study Group C:45;Control Group D:45;Control Group D:45;

China

NCT02139501
(ClinicalTrials.gov)

May 2014

13/5/2014

An Investigation of rhTPO With Different Frequencies in the Management of ITP

A Randomized Study to Evaluate the Efficacy and Safety of Different Doses and Frequencies of Recombinant Human Thrombopoietin (rhTPO) in the Management of Primary Immune Thrombocytopenia (ITP)

Immune Thrombocytopenia

Drug: Recombinant Human Thrombopoietin (rhTPO)

Shandong University

The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital

Completed

18 Years

60 Years

Both

Phase 3

China

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

NCT01734044
(ClinicalTrials.gov)

July 2012

21/11/2012

rhTPO Combining Dexamethasone Versus High-dose Dexamethasone for Initial Treatment of ITP

A Multicentre Investigation of Recombinant Human Thrombopoietin (rhTPO) Combining Dexamethasone Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)

Purpura;Idiopathic Thrombocytopenic Purpura

Drug: recombinant human thrombopoietin (rhTPO); dexamethasone;Drug: Dexamethasone

Shandong University

The Affiliated Hospital of the Chinese Academy of Military Medical Sciences;Anhui Medical University;The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital;Shenzhen Second People's Hospital;China Medical University, China;Zhejiang Provincial Hospital of TCM

Completed

18 Years

75 Years

Both

158

Phase 3

China

NCT01734057
(ClinicalTrials.gov)

July 2012

21/11/2012

Recombinant Human Thrombopoietin (rhTPO) Combining Rituximab Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)

A Multicentre Investigation of Recombinant Human Thrombopoietin (rhTPO) Combining Rituximab Versus High-dose Dexamethasone for Initial Treatment of Primary Immune Thrombocytopenia (ITP)

Purpura;Idiopathic Thrombocytopenic Purpura

Drug: recombinant human thrombopoietin (rhTPO); rituximab;Drug: Dexamethasone

Shandong University

Withdrawn

18 Years

75 Years

Both

Phase 3

China

NCT01525836
(ClinicalTrials.gov)

May 2011

31/1/2012

rhTPO Combining Rituximab Versus Low-dose Rituximab in Management of ITP

Recombinant Human Thrombopoietin in Combination With Rituximab Versus Low-dose Rituximab for the Treatment of Refractory ITP.

Purpura;Idiopathic Thrombocytopenic Purpura

Drug: rituximab; recombinant human thrombopoietin (rhTPO);Drug: Rituximab

Shandong University

The Affiliated Hospital of the Chinese Academy of Military Medical Sciences;The First Affiliated Hospital of Anhui Medical University;The First Affiliated Hospital of Dalian Medical University;Shandong Provincial Hospital;Shenzhen Second People's Hospital;First Hospital of China Medical University

Completed

18 Years

72 Years

Both

123

Phase 3

China

NCT01805648
(ClinicalTrials.gov)

February 2011

22/2/2013

Efficacy and Safety Study of Maintenance Treatment With rhTPO in Thrombocytopenic Subjects With ITP

A Multicenter, Single-Arm, Open Label Study Evaluating the Efficacy and Safety of Maintenance Treatment With Recombinant Human Thrombopoietin in Thrombocytopenic Subjects With Immune Thrombocytopenia

Thrombocytopenia;Immune Thrombocytopenia;Idiopathic Thrombocytopenic Purpura

Drug: rhTPO

Peking Union Medical College

Shenyang Sunshine Pharmaceutical Co., LTD.

Recruiting

14 Years

70 Years

Both

120

Phase 3

China

NCT01506414
(ClinicalTrials.gov)

June 2009

16/11/2011

Recombinant Human Thrombopoietin in Combination With Rituximab in Immune Thrombocytopenia (ITP)

A Multicentre Investigation of Recombinant Human Thrombopoietin (Rh-TPO) Combine With Low-dose Rituximab in Management of Steroid-Resistant/Relapsed Immune Thrombocytopenia (ITP)

Purpura;Idiopathic Thrombocytopenic Purpura

Drug: rhTPO in combination with Rituximab

Ming Hou

Peking Union Medical College Hospital;Chinese Academy of Medical Sciences;First Affiliated Hospital, Sun Yat-Sen University;West China Hospital;Shandong Provincial Hospital;Wuhan Union Hospital, China;Zhejiang University

Completed

16 Years

75 Years

Both

Phase 3

China