Clinical trials: Deficiency Disease
Insulin-like Growth Factor-1 Deficiency
rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary).
- Trial name
- NCT00572156
- Testing
- NutropinAq® (Somatropin [rDNA origin]), Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- Ipsen
Advanced Solid Tumors
Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug
The purpose of the study is to test how well patients with advanced solid tumors respond to treatment with elimusertib (BAY1895344) in combination with pembrolizumab.
- Trial name
- NCT04095273
- Testing
- Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
- Where
- 16 sites in United States, Germany and 3 more
- Volunteers
- 56 people
- Sponsor
- Bayer
HIV Infection
MK0518 in the Treatment of HIV-Infected Patients Switched From a Protease Inhibitor Regimen (0518-033)(TERMINATED)
The purpose of this study is to investigate the efficacy, safety, and tolerability of an investigational treatment for patients with Human Immunodeficiency Virus (HIV).
- Trial name
- NCT00443729
- Testing
- Comparator: raltegravir, Comparator: lopinavir (+) ritonavir
- Volunteers
- 355 people
- Sponsor
- Merck Sharp & Dohme LLC
HIV Infections
TMC114-TiDP3-C182 - A Study to Compare the Oral Bioavailability of a 800 mg Prototype Tablet Formulation of Darunivar (DRV) to That of the 400 mg Commercial Tablet Formulation in the Presence of Low Dose Ritonavir, Under Fasted and Fed Conditions
The purpose of this study is to compare the drug levels of darunavir obtained after administration of a single administration of the 800 mg tablet (new formulation) to that following administration of two 400 mg commercial…
- Trial name
- NCT01052883
- Testing
- DRV commercial formulation/ DRV new formulation/ rtv 100mg tab
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Tibotec Pharmaceuticals, Ireland
HIV Infections
Double Blind Study of Thymopentin Effects on Patients With HIV-1 Infection
Examine the ability of thymopentin (Timunox) to: Reduce the amount and/or frequency of virus isolation. Stimulate the immune system and alter the clinical findings in patients infected with HIV who do not yet have AIDS.
- Trial name
- NCT00002066
- Testing
- Thymopentin
- Where
- United States
- Sponsor
- Immunobiology Research Institute
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
A Phase I of SS109 in Hemophilia A or and B With Inhibitors
This phase I study aims to evaluate the safety, PK/PD and immunogenicity of SS109 in hemophilia A or and B with inhibitors. Twenty -seven patients are enrolled in study, and divided into five dose cohorts, from 30μg/kg to…
- Conditions
- Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Factor VII Deficiency
- Trial name
- NCT05651061
- Testing
- SS109
- Where
- 10 sites in China
- Volunteers
- 27 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Hereditary Angioedema, Hereditary Angioedema Type I
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks.
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- RAPIDe-2 (NCT05396105)
- Testing
- deucrictibant
- Where
- 60 sites in United States, Argentina and 22 more
- Volunteers
- 150 people
- Sponsor
- Pharvaris Netherlands B.V.
Hypercholesterolemia, Dyslipidaemias
An Exploratory Study To Look At The Effect Of Two Investigational Drugs On Body Fat And Inflammation
The investigational drugs administered in this study activate proteins called PPARs. Data in the scientific literature on PPARs, as well as animal data and early clinical data generated by GSK with these drugs, suggest that…
- Conditions
- Hypercholesterolemia, Dyslipidaemias, Obesity
- Trial name
- NCT00388180
- Testing
- GW501516, GW590735
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Human Immunodeficiency Virus (HIV)
Comparison of Doravirine, Tenofovir, Lamivudine (MK-1439A) and ATRIPLA™ in Treatment-Naive Human Immunodeficiency Virus Type 1 (HIV-1)-Infected Participants (MK-1439A-021)
The purpose of this study is to compare the antiretroviral activity of doravirine, tenofovir, lamivudine (MK-1439A), a single-tablet, once-daily (q.d.) fixed-dose combination (FDC) containing doravirine (MK-1439A) 100 mg +…
- Trial name
- DRIVE-AHEAD (NCT02403674)
- Testing
- Doravirine, Tenofovir, Lamivudine, ATRIPLA™
- Volunteers
- 734 people
- Sponsor
- Merck Sharp & Dohme LLC
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Human Immunodeficiency Virus Infection
Drug Use Investigation of Kaletra Tablets (Once Daily Administration) on Patients With HIV-infection
This study of Kaletra (Lopinavir (LPV)/Ritonavir (RTV)) tablets will be conducted to clarify the following with regard to treatment with this drug: 1.
- Trial name
- NCT01328158
- Where
- 23 sites in Japan
- Volunteers
- 236 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
HIV Infections
Double-Blind, Randomized, Placebo-Controlled Study of Low Dose Oral Interferon Alfa-n3 (Human Leukocyte Derived) in ARC Patients
To conduct a parallel-group, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of an orally administered low dose interferon alfa-n3 as an immunomodulator in the treatment of mild to moderate…
- Trial name
- NCT00002012
- Testing
- Interferon alfa-n3
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Interferon Sciences
Ornithine Transcarbamylase (OTC) Deficiency
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency.
- Trial name
- CAPtivate (NCT02991144)
- Testing
- scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 16 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma
A Clinical Trial to Evaluate Efficacy, Safety, and Pharmacokinetics of Gamunex in Participants With Secondary Antibody Deficiency Associated With Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma
The main goal of this study is to show that people with certain immune problems (from Chronic Lymphocytic Leukemia, Multiple Myeloma, or Non-Hodgkin Lymphoma) get fewer serious infections when they receive Gamunex C through an IV…
- Trial name
- SIGMA (NCT07582432)
- Testing
- Gamunex-C, 10% Injectable Solution
- Where
- 5 sites in United States
- Volunteers
- 49 people
- Sponsor
- Grifols Biologicals, LLC
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard 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
HIV Infections
Non-interventional Study on Use of Darunavir With Ritonavir in Clinical Practice
The purpose of this non-interventional study is a description of use of darunavir in daily practice, in retrospective and perspective manner.
- Trial name
- NCT01375881
- Testing
- darunavir/ritonavir plus background regimen
- Volunteers
- 31 people
- Sponsor
- Janssen-Cilag S.p.A.
Metastatic Castration-Resistant Prostate Cancer
XmAb®20717 (Vudalimab) Alone or in Combination With Chemotherapy or Targeted Therapy in Patients With Metastatic Castration-Resistant Prostate Cancer
This Phase 2 study will investigate the safety and clinical activity of vudalimab (XmAb20717) alone or in combination with standard of care anticancer therapies in patients with metastatic castration-resistant prostate cancer…
- Trial name
- NCT05005728
- Testing
- vudalimab + carboplatin + cabazitaxel, vudalimab + olaparib, vudalimab monotherapy
- Where
- 26 sites in United States
- Volunteers
- 72 people
- Sponsor
- Xencor, Inc.
Hereditary Angioedema
A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)
This study is being conducted to evaluate the efficacy and safety of icatibant compared to placebo in patients experiencing acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT00912093
- Testing
- Icatibant
- Where
- 64 sites in United States, Australia and 7 more
- Volunteers
- 98 people
- Sponsor
- Shire
HIV Infection
Single DermaVir Immunization in HIV-1 Infected Patients on HAART
DermaVir is a plasmid DNA-containing synthetic nanomedicine. It is administered topically with DermaPrep to target Langerhans cells.
- Trial name
- GIHU004 (NCT00712530)
- Testing
- DermaVir, HAART
- Where
- Hungary
- Volunteers
- 9 people
- Sponsor
- Genetic Immunity
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