Clinical trials: Protein Deficiency
Congenital Factor VII Deficiency
A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency
Open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of a single dose of sutacimig monotherapy in participants with congenital FVII deficiency (FVIID).
- Trial name
- NCT07347249
- Testing
- Sutacimig
- Where
- United Kingdom
- Volunteers
- 18 people
- Sponsor
- Hemab ApS
Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer)
An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
This is a Phase Ib/II clinical study. The Phase Ib dose-escalation study aims to evaluate and determine the recommended Phase II dose (RP2D) of TCC1727 in combination with benmelstobart /olaparib /topotecanfor patients with…
- Conditions
- Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer), Gastric (Stomach) Cancer, Endometrial Cancer
- Trial name
- NCT07371663
- Testing
- TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg
- Where
- 3 sites in China
- Volunteers
- 266 people
- Sponsor
- Beijing Tide Pharmaceutical Co., Ltd
Hypogammaglobulinemia, Bacterial Infections
A Study to Evaluate Efficacy, Safety, and PK of XEMBIFY®+Standard Medical Treatment (SMT) Compared to Placebo+SMT to Prevent Infections in Participants With HGG and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma, and Non-Hodgkin Lymphoma
The primary purpose of the study is to evaluate whether biweekly administered XEMBIFY® plus Standard Medical Treatment (SMT) over a one-year period will reduce the rate of major bacterial infections per participant per year in…
- Conditions
- Hypogammaglobulinemia, Bacterial Infections, B-cell Chronic Lymphocytic Leukemia, Multiple Myleoma
- Trial name
- EXCELL (NCT05645107)
- Testing
- Xembify
- Where
- 62 sites in United States, Bosnia and Herzegovina and 6 more
- Volunteers
- 386 people
- Sponsor
- Grifols Therapeutics LLC
Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage
HEMOLEVEN® Expanded Access Program Prevention of Surgical/Postpartum Hemorrhage Severe Inherited Factor XI Deficiency
The objective of the Expanded Access Program is to provide HEMOLEVEN, a replacement coagulation factor XI, to patients with severe inherited factor XI deficiency where, in the opinion of the treating physician, the benefits of…
- Conditions
- Wound; Rupture, Surgery, Cesarean Section, Postpartum Hemorrhage, Surgery
- Trial name
- EAP (NCT01701947)
- Testing
- Hemoleven
- Where
- United States
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Hemophilia A
Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts.
- Trial name
- NCT00157040
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Baxalta now part of Shire
CD55-deficient Protein-losing Enteropathy
Compassionate Use of Pozelimab
Provide compassionate use of pozelimab
- Trial name
- NCT05232110
- Testing
- Pozelimab
- Sponsor
- Regeneron Pharmaceuticals
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
WHIM Syndrome
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not…
- Trial name
- NCT03087370
- Testing
- No intervention
- Sponsor
- X4 Pharmaceuticals
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
Lymphoma, Non-Hodgkin Lymphoma
AZD3470 as Monotherapy or in Combination With Anticancer Agent(s) in Participants With Haematologic Malignancies.
This study is designed to evaluate the safety, tolerability, PK, pharmacodynamics, and preliminary efficacy following oral administration of AZD3470 as a monotherapy, and in combination with other anticancer agents in…
- Conditions
- Lymphoma, Non-Hodgkin Lymphoma, Hodgkin Lymphoma, Peripheral T-cell Lymphoma (PTCL)
- Trial name
- PRIMAVERA (NCT06137144)
- Testing
- AZD3470, Pembrolizumab
- Where
- 38 sites in United States, Australia and 9 more
- Volunteers
- 161 people
- Sponsor
- AstraZeneca
Acute Bacterial Infections, Acute Viral Infections
300 Antibody Diagnostic Test Kit
For the development of a Point of Care IVD test kit for acute phase disease detection against a variety of bacterial and viral infections.
- Trial name
- Antibody 300 (NCT01646411)
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- The Plasma Source
Iron Deficiency Anemia
Ferrous Acetyl-Aspartate Casein Formulation Evaluation Over Ferrous Sulfate in Iron Deficiency Anemia
The scope of this study is to compare the efficacy of the new oral formulation of Fe-ASP to oral ferrous sulfate in patients with iron deficiency anemia (IDA) for the restoration of decreased circulating Hb.
- Trial name
- ACCESS (NCT03524651)
- Testing
- Ferrous Sulfate, Fe-ASP
- Where
- 3 sites in Greece
- Volunteers
- 60 people
- Sponsor
- Uni-Pharma Kleon Tsetis Pharmaceutical Laboratories S.A.
Sarcoma, Kaposi, HIV Infections
SU5416 in Patients With AIDS-Related Kaposi's Sarcoma Who Have Not Responded to Treatment
The purpose of this study is to test the effectiveness of SU5416 in patients with AIDS-related Kaposi's sarcoma (KS).
- Trial name
- NCT00005931
- Testing
- SU5416
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- SUGEN
Hemophilia A
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
- Trial name
- NCT06940830
- Testing
- Efanesoctocog alfa
- Where
- 31 sites in Croatia, Czechia and 6 more
- Volunteers
- 252 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
PNH
Observational of PNH Type Cells in Korean Patients With Bone Marrow Failure Syndrome and Having Hemolytic PNH
Patients who are positive of PNH-type cells are followed up for the percentage of PNH-type cells regularly for examining how it change.
- Trial name
- OPENK (NCT03460301)
- Volunteers
- 233 people
- Sponsor
- Handok Inc.
Congenital Protein C Deficiency
A Study of TAK-662 for Japanese Patients With Congenital Protein C Deficiency
Pharmacokinetic Part: This study is for Japanese participants with congenital protein C deficiency. The main aim of this study is to check how much TAK-662 stays in their blood over time.
- Trial name
- NCT04984889
- Testing
- TAK-662
- Where
- 4 sites in Japan
- Volunteers
- 5 people
- Sponsor
- Takeda
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
HIV Infections
Safety and Effectiveness of L2-7001 (Interleukin-2) in HIV-Positive Patients Receiving Anti-HIV Therapy
The purpose of this study is to see if it is safe and effective to give HIV-positive patients L2-7001 (a type of interleukin-2) plus anti-HIV therapy.
- Trial name
- NCT00002449
- Testing
- Aldesleukin
- Where
- 23 sites in United States
- Volunteers
- 212 people
- Sponsor
- Chiron Corporation
HIV Infections
Evaluating the Safety and Immunogenicity of Polyvalent DNA/gp120 HIV Vaccine in Healthy, HIV-uninfected Adults
The purpose of the study is to evaluate the safety, tolerability, and immunogenicity of polyvalent env (A,B,C,A/E)/gag (C) DNA and gp120 (A,B,C,A/E) protein vaccines (PDPHV201401) co-administered together with or without adjuvant…
- Trial name
- NCT04927585
- Testing
- env (A,B,C,A/E)/gag (C) DNA Vaccine, gp120 (A,B,C,A/E) Protein Vaccine, GLA-SE adjuvant
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Worcester HIV Vaccine