DDrare: Database of Drug Development for Rare Diseases

62. 発作性夜間ヘモグロビン尿症
［臨床試験数：202，薬物数：120（DrugBank：25），標的遺伝子数：15，標的パスウェイ数：90］

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
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 = ABP 959; ABP 959 - biosimilar to eculizumab; ABP 959 - proposed biosimilar to eculizumab; ACH-0144471; ACH-0145228; ALN-62643; ALN-CC5; ALXN1102; ALXN1103; ALXN1210; ALXN2040 (ACH-0144471); ALXN2050; APL-2; Anti-C5 antibody; Anti-thymocyte globulin; BCD-148; BCX9930; BCX9930 hydrochloride; Bryostatin 1; C5 Inhibitor; C5 inh MAb, SKY59, RO/CH7092230; C5 inhibitor background therapy; COVERSIN; CROVALIMAB; Campath; Campath, Chemo and/or TBI Allo SCT; Coversin; Crovalimab; Cyclophosphamide; Cyclosporin; Cyclosporin A; Cyclosporin A+Glucocorticoids; Cyclosporine; Cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant; DANICOPAN; Danicopan; Danicopan (ALXN2040); Device: Miltenyi Biotec CliniMACS; Device: Miltenyi CliniMACS; Dextran; ECULIZUMAB; Eculizumab; Elizaria®; Fc- and CDR-modified humanised monoclonal antibody against C5; Filgrastim; Fludarabine; Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Glucocorticoids; H5G1.1-mAb; Human monoclonal antibody directed against complement 5 protein; Iron Dextran; Iron sucrose; L04AA25; LFG316; LNP023; Levamisole; Levamisole+cyclosporin A+Glucocorticoids; Melphalan; Methotrexate; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Myeloablative Preparative Regimen; No; Other: Blood collection for measurement of eculizumab peak concentrations; Other: Laboratory Biomarker Analysis; Other: Standard of care (SOC); Other: laboratory biomarker analysis; Other: pharmacological study; Pegcetacoplan; Pegcetacoplan (APL-2); Phosphate; Procedure: Allogeneic stem cell transplantation; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Stem Cell Transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; RA101495; RA101495 sodium; RAVULIZUMAB; REGN3918; RO7112689; RO7112689/F01; RO7112689/F03-01; RO7112689/F03-03; RVA576; RVA576 (COVERSIN); RVA576 (Coversin); Radiation: Total-Body Irradiation; Radiation: total-body irradiation; Ravulizumab; Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; SB12 (proposed eculizumab biosimilar); SOLIRIS; Sargramostim; Sirolimus; Soliris; Soliris (eculizumab); Soliris 300; Soliris®; Sterile Normal Saline (0.9% NaCl); Sucrose; Synthetic 15-amino-acid macrocyclic peptide; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker; Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker; Systematic chemotherapy and antibodies; Systemic chemotherapy and antibodies; Tacrolimus; ULTOMIRIS; Ultomiris; VARI; ZILUCOPLAN; Zilucoplan; Zilucoplan (RA101495);  LNP023 HYDROCHLORIDE SALT

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	EUCTR2016-002128-10-IT (EUCTR)	19/09/2017	06/02/2018	Studio volto a valutare la sicurezza, l’efficacia, la farmacocinetica e la farmacodinamica di RO7112689 in volontari sani e in pazienti con Emoglobinuria Parossistica Notturna	An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) - An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Code: RO7112689/F01 Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230	F. HOFFMANN - LA ROCHE LTD.	NULL	Not Recruiting	Female: yes Male: yes	49	Phase 1;Phase 2	France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of
2	EUCTR2016-002128-10-FR (EUCTR)	17/08/2017	08/09/2017	A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria	An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230	F. Hoffmann-La Roche Ltd	NULL	Not Recruiting	Female: yes Male: yes	49	Phase 1;Phase 2	France;Hungary;Netherlands;Germany;Japan;Italy;Korea, Republic of
3	EUCTR2016-002128-10-DE (EUCTR)	14/03/2017	24/10/2016	A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria	An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of crovalimab in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 Product Code: RO7112689/F03-01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 Product Code: RO7112689/F03-03 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	49	Phase 1;Phase 2	France;Hungary;Netherlands;Germany;Italy;Japan;Korea, Republic of
4	EUCTR2016-002128-10-HU (EUCTR)	24/01/2017	14/11/2016	A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria	An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	49	Phase 1;Phase 2	France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of
5	EUCTR2016-002128-10-NL (EUCTR)	14/09/2016	05/09/2016	A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria	An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Code: RO7112689/F01 INN or Proposed INN: Not available Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230	F. Hoffmann-La Roche Ltd	NULL	Not Recruiting	Female: no Male: yes	74	Phase 1;Phase 2	France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;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

EUCTR2016-002128-10-IT
(EUCTR)

19/09/2017

06/02/2018

Studio volto a valutare la sicurezza, l’efficacia, la farmacocinetica e la farmacodinamica di RO7112689 in volontari sani e in pazienti con Emoglobinuria Parossistica Notturna

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) - An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO

Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 20.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Code: RO7112689/F01
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230

F. HOFFMANN - LA ROCHE LTD.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of

EUCTR2016-002128-10-FR
(EUCTR)

17/08/2017

08/09/2017

A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 20.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000012950;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230

F. Hoffmann-La Roche Ltd

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

France;Hungary;Netherlands;Germany;Japan;Italy;Korea, Republic of

EUCTR2016-002128-10-DE
(EUCTR)

14/03/2017

24/10/2016

A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of crovalimab in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of crovalimab in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
Product Code: RO7112689/F03-01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230
Product Code: RO7112689/F03-03
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

France;Hungary;Netherlands;Germany;Italy;Japan;Korea, Republic of

EUCTR2016-002128-10-HU
(EUCTR)

24/01/2017

14/11/2016

A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

France;Hungary;Brazil;Netherlands;Germany;New Zealand;Japan;Italy;Korea, Republic of

EUCTR2016-002128-10-NL
(EUCTR)

14/09/2016

05/09/2016

A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria

An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)

Product Code: RO7112689/F01
INN or Proposed INN: Not available
Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230

F. Hoffmann-La Roche Ltd

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;United Kingdom