DDrare: Database of Drug Development for Rare Diseases

Searched query = "Idiopathic thrombocytopenic purpura", "Primary immune thrombocytopenia"
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 = 10-mg dose of E5501 2G tablet; 10-mg dose of E5501 cyclodextrin oral solution; 10-mg dose of E5501 lipid-based oral; 10-mg dose of E5501-P21% powder; AKR-501, YM-477; AMG 531; AMG531; ARGX-113; ATRA; AVATROMBOPAG MALEATE; Alemtuzumab; Alemtuzumab, Rituximab; All-Trans Retinoid Acid(ATRA); Aminophylline; Amoxicillin; Amoxicillin hydrate; Amoxycillin; Anti-CD40L; Anti-CD40LdAb; Anti-D; Atorvastatin; Atorvastatin 10mg; Atorvastatin 20mg; Avatrombopag; Avatrombopag (proposed); Avatrombopag maleate; Avatrombopag tablets; Azathioprine; BMS-986004; BMS-986004 1500 mg IV; BMS-986004 225 mg IV; BMS-986004 675 mg IV; BMS-986004 75 mg IV; BT595; Blinded (Avatrombopoag tablets); Blisibimod; Bortezomib; Caffeic acid tablets; Clarithromycin; Conventional; Corticosteroids (Aprednislon); CsA; Cyclosporin; Cyclosporin A; Cyclosporine; DARATUMUMAB; Danazol; Daratumumab; Decitabine; Dexamethasone; Dexamethasone (1 cycle); Dexamethasone (4 cycles); Dexamethasone Abcur; Dexamethasone and Rituximab; Doptelet; Doptelet 20 mg film-coated tablets; Doxil; E5501; E5501 40 mg 2 x 20-mg tablets, orally, fasted; E5501 40 mg 2 x 20-mg tablets, orally, with food; E5501 40mg 2 x 20-mg tablets, orally, fasted; ELTROMBOPAG; ELTROMBOPAG OLAMINE; Efgartigimod; Efgartigimod alfa; Eltrombopag; Eltrombopag (Revolade); Eltrombopag combining rituximab; Eltrombopag olamine (SB-497115-GR); Eltrombopag olamine, Revolade, Promacta; Eltrombopag oral tablets; FCM; GMA161; Gammaplex; Gammaplex, intravenous immunoglobulin; Glycyrrhetinic Acid; HBM9161 Dose A; HBM9161 Dose B; HUMAN NORMAL IMMUNOGLOBULIN; HUMAN NORMAL IMMUNOGLOBULIN (IV); HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE; HUMAN NORMAL IMMUNOGLOBULIN FOR INTRVENOUS USE; HUMAX-CD38; Hetrombopag; Hetrombopag Olamine; High dose Dexamethasone pulses; Huaiqihuang Granule; Human Immunoglobulin; Human Immunoglobulin G; Human Normal Immunoglobulin; Human Normal Immunoglobulin (iv); Human Normal Immunoglobulin G (IgG > 98% purity); Human immunoglobulin G; Human normal immunoglobulin; Human normal immunoglobulin for intravenous use; I10E; IGIV3I Grifols 10%; IMMUNOGLOBULIN G; INTERLEUKIN 2; IVIG-L; Ig VENA; Ig VENA 100 ml vial; Ig VENA 50 g/l solution for infusion 100 ml vial + infusion set; IgG Next Generation; IgNextGen 10%; Immune Globulin Intravenous (Human), 10% Triple Virally Reduced Solution; Immunglobulin; Immunoglobulin; Inactivated Trivalent Influenza vaccine; Interleukin 2; LIV-GAMMA SN Inj.; Lansoprazole; Lansoprazole, clarithromycin, amoxycillin; MabThera; Mabthera; Medical Standard of Care for ITP; Methylprednisolone; Metronidazole; NPLATE; NPLATE 500 microgramos polvo para solución inyectable; Nanogam; NewGAM; NewGam; Nplate; Null; Octagam 10%; Octagam® 10%; Open Label (Avatrombopag tablets); Other: RP% Measurement by FCM as a Diagnostic Test for ITP; Other: healthy controls; PF-06755347 intravenous; PF-06755347 subcutaneous; PG2; PRTX-100 (Staphylococcal protein A); Palmitate; Placebo; Prednisolone; Prednisone; Privigen®; Promacta; Proteína recombinante estimulante de la megacariopoyesis; R788; REVOLADE; REVOLADE, PROMACTA; RITUXIMAB; ROMIPLOSTIM; ROZANOLIXIZUMAB; Recombinant Human Thrombopoietin; Recombinant Human Thrombopoietin (rhTPO); Recombinant human thrombopoietin (rh-TPO); Recombinant human thrombopoietin (rhTPO); Relovade; Revolade; Revolade 25 mg film-coated tablets; Revolade 50 mg film-coated tablets; RhTPO; RhTPO in combination with Rituximab; Rituximab; Rituximab [MabThera/Rituxan]; Romiplostim; Romiplostin; Rozanolixizumab; SB-497115; SB-497115-GR; SB-497115-GR 12.5mg; SB-497115-GR 25mg; SB-497115-GR 50 mg; SB497115; SM101; SM101 20mg/mL; SM101 5mg/mL; STAPHYLOCOCCAL PROTEIN A; Sirolimus; Standard of care; Steroids; TAK-079; TPO; Takecab tablets + amoxicillin + clarithromycin (first-line eradication) Takecab tablets + amoxicillin + metronidazole (second-line eradication); Thrombopoietin; Triple therapy; Tysabri; UCB7665; Vonoprazan; Water; [UCB7665 ]

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2019-002101-21-NL (EUCTR)	25/05/2020	07/01/2020	A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)	A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia - ADVANCE+	Primary immune thrombocytopenia MedDRA version: 20.1;Level: LLT;Classification code 10050245;Term: Autoimmune thrombocytopenia;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Name: Efgartigimod Product Code: ARGX-113 INN or Proposed INN: Efgartigimod alfa Other descriptive name: ARGX-113	argenx BVBA	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	156	Phase 3	United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Netherlands;Germany;Japan
2	EUCTR2019-002101-21-FR (EUCTR)	22/04/2020	20/02/2020	A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)	A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia - ADVANCE+	Primary immune thrombocytopenia MedDRA version: 20.1;Level: LLT;Classification code 10050245;Term: Autoimmune thrombocytopenia;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Name: Efgartigimod Product Code: ARGX-113 INN or Proposed INN: Efgartigimod alfa Other descriptive name: ARGX-113	argenx BVBA	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	156	Phase 3	United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands;Japan
3	EUCTR2019-002101-21-ES (EUCTR)	09/04/2020	23/01/2020	A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)	A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia - ADVANCE+	Primary immune thrombocytopenia MedDRA version: 20.1;Level: LLT;Classification code 10050245;Term: Autoimmune thrombocytopenia;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Name: Efgartigimod Product Code: ARGX-113 INN or Proposed INN: Efgartigimod alfa Other descriptive name: ARGX-113	argenx BVBA	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	156	Phase 3	United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands;Japan

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

EUCTR2019-002101-21-NL
(EUCTR)

25/05/2020

07/01/2020

A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)

A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients with Primary Immune Thrombocytopenia - ADVANCE+

Primary immune thrombocytopenia
MedDRA version: 20.1;Level: LLT;Classification code 10050245;Term: Autoimmune thrombocytopenia;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Product Name: Efgartigimod
Product Code: ARGX-113
INN or Proposed INN: Efgartigimod alfa
Other descriptive name: ARGX-113

argenx BVBA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

156

Phase 3

United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Netherlands;Germany;Japan

EUCTR2019-002101-21-FR
(EUCTR)

22/04/2020

20/02/2020

Product Name: Efgartigimod
Product Code: ARGX-113
INN or Proposed INN: Efgartigimod alfa
Other descriptive name: ARGX-113

argenx BVBA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

156

Phase 3

United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands;Japan

EUCTR2019-002101-21-ES
(EUCTR)

09/04/2020

23/01/2020

Product Name: Efgartigimod
Product Code: ARGX-113
INN or Proposed INN: Efgartigimod alfa
Other descriptive name: ARGX-113

argenx BVBA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

156

Phase 3

United States;Spain;Ukraine;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Bulgaria;Germany;Netherlands;Japan