DDrare: Database of Drug Development for Rare Diseases

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_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2020-000501-93-AT (EUCTR)	27/11/2020	08/09/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects 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 Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;Austria;South Africa;United Kingdom
2	EUCTR2020-000501-93-GB (EUCTR)	29/09/2020	09/07/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects 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 Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;South Africa;United Kingdom
3	NCT04330534 (ClinicalTrials.gov)	March 3, 2020	23/3/2020	First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH	A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria	Paroxysmal Nocturnal Hemoglobinuria	Drug: BCX9930;Drug: Placebo	BioCryst Pharmaceuticals	NULL	Recruiting	18 Years	N/A	All	148	Phase 1;Phase 2	South Africa

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

EUCTR2020-000501-93-AT
(EUCTR)

27/11/2020

08/09/2020

A long term study of BCX9930 in patients with PNH

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects 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 Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride

BioCryst Pharmaceuticals Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

200

Phase 2

European Union;Austria;South Africa;United Kingdom

EUCTR2020-000501-93-GB
(EUCTR)

29/09/2020

09/07/2020

A long term study of BCX9930 in patients with PNH

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride

BioCryst Pharmaceuticals Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

200

Phase 2

European Union;South Africa;United Kingdom

NCT04330534
(ClinicalTrials.gov)

March 3, 2020

23/3/2020

First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH

A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria

Drug: BCX9930;Drug: Placebo

BioCryst Pharmaceuticals

NULL

Recruiting

18 Years

N/A

All

148

Phase 1;Phase 2

South Africa