Clinical trials: Deficiency Disease
Infection, Human Immunodeficiency Virus
A Study to Evaluate the Effect of Boceprevir and Telaprevir on Dolutegravir Pharmacokinetics in Healthy Adult Subjects (ING115697).
Dolutegravir (DTG, GSK1349572) is an integrase inhibitor that is currently in Phase 3 clinical development for the treatment of human immunodeficiency virus (HIV) infection.
- Trial name
- NCT01563328
- Testing
- DTG, BCV, TVR
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Glut1 Deficiency Syndrome, De Vivo Disease
Early Diagnosis of the GLUT1 Deficiency Syndrome With a Blood Based Test
The study aims at validating the diagnostic performances of the METAglut1, a blood in vitro diagnostic test, for the simple and early diagnosis of the Glut1 deficiency syndrome (Glut1DS, or De Vivo disease).
- Conditions
- Glut1 Deficiency Syndrome, De Vivo Disease, Seizures, Movement Disorders
- Trial name
- METAglut1 (NCT03722212)
- Testing
- METAglut1
- Where
- 28 sites in France
- Volunteers
- 636 people
- Sponsor
- METAFORA biosystems
Acute Hereditary Angio Edema
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Prospective, multicenter, randomized, double-blind, parallel group, placebo- controlled, efficacy and safety phase 3 study of an intravenous human plasma- derived C1 esterase inhibitor (C1-INH) concentrate in participants with…
- Trial name
- NCT06361537
- Testing
- OCTA-C1-INH
- Where
- 25 sites in United States, Albania and 11 more
- Volunteers
- 124 people
- Sponsor
- Octapharma
Pyruvate Kinase Deficiency
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
This is an open-label Phase I trial to evaluate the safety of a hematopoietic cell-based gene therapy for patients with Pyruvate Kinase Deficiency (PKD).
- Trial name
- NCT04105166
- Testing
- RP-L301
- Where
- 3 sites in United States, Spain
- Volunteers
- 4 people
- Sponsor
- Rocket Pharmaceuticals Inc.
HIV Infections, Cytopenias
The Safety and Effectiveness of r-HuEPO in Patients With AIDS and Anemia Caused by AIDS and Treatment With AZT
To determine the safety and efficacy of erythropoietin administered subcutaneously to AIDS patients with anemia secondary to their disease and/or concomitant zidovudine (AZT) therapy.
- Trial name
- NCT00002302
- Testing
- Epoetin alfa
- Where
- 2 sites in United States
- Sponsor
- Ortho Pharmaceuticals
Immunity, Vaccine Reaction
Efficacy and Tolerability of ABBC1 in Volunteers Receiving the Influenza or Covid-19 Vaccine
The immune system response needs to be forceful but also balanced for a rapid recovery from infection which avoids harmful overreactions.
- Conditions
- Immunity, Vaccine Reaction, Influenza, Covid19
- Trial name
- NCT04798677
- Testing
- ABBC1 Immunoessential
- Where
- Spain
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- AB Biotek
Intestinal Disease
Study of the Prevalence of Iron Deficiency in Patients With Chronic Inflammatory Bowel Disease
Despite its known prevalence in IBD, a recent study conducted with Prof. Cacoub (unpublished) on the national health insurance database showed that iron deficiency was an under-diagnosed and under-treated co-morbidity.
- Trial name
- NCT04123990
- Testing
- Iron status testing
- Volunteers
- 1,100 people
- Sponsor
- VIFORFRANCE
AIDS Virus, Disorder of Immune Reconstitution
Mesenchymal Stem Cells for Immune Non-responder Patients With HIV Infection
The goal of this clinical trial is to explore the effect of mesenchymal stem cell therapy on immune non-responder patients. The main questions it aims to answer are: 1.
- Trial name
- NCT05872659
- Testing
- normal saline, Mesenchymal stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shandong Qilu Cell Therapy Engineering Technology Co., Ltd
Congenital Antithrombin Deficiency
A Study of KW-3357 in Congenital Antithrombin Deficiency
The aim of this study is to determine the pharmacokinetics, safety and tolerability of KW-3357 in asymptomatic subjects with congenital antithrombin deficiency.
- Trial name
- NCT00938288
- Testing
- KW-3357
- Where
- 8 sites in Germany, Sweden and 1 more
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Anemia
Safety of FCM vs. Standard of Care in Treating Iron Deficiency Anemia in Heavy Uterine Bleeding and Post Partum Patients
The Objective of this study is to study the safety of FCM in patients with anemia caused by Heavy Uterine Bleeding and the Post Partum state.
- Trial name
- NCT00548860
- Testing
- Ferric Carboxymaltose, Standard Medical Care (SMC)
- Where
- United States
- Volunteers
- 2,018 people
- Sponsor
- American Regent, Inc.
Cryptosporidiosis, HIV Infections
Protocol For the Treatment of Cryptosporidiosis in AIDS Patients With Diclazuril (R64,433)
To evaluate the safety and efficacy of diclazuril capsules compared with placebo capsules as treatment of cryptosporidial related diarrhea in AIDS patients.
- Trial name
- NCT00002027
- Testing
- Diclazuril
- Where
- 3 sites in United States
- Volunteers
- 60 people
- Sponsor
- Janssen, LP
HIV
Enfuvirtide/Current Protease Inhibitor Switch to PREZISTA (Darunavir)/Ritonavir + TMC125 in HIV Patients With Enfuvirtide Side Effects.
The purpose of this study is to examine the safety, tolerability, and effectiveness of darunavir/ritonavir combined with TMC125 when current protease inhibitor(s), Non-Nucleoside Reverse Transcriptase Inhibitors (NNRTI(s)) and…
- Trial name
- NCT00460746
- Testing
- TMC125, Darunavir; Ritonavir
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Tibotec, Inc
Chronic Heart Failure, Iron Deficiency
Ferinject® Assessment in Patients With Iron Deficiency and Chronic Heart Failure (FAIR-HF)
This study is designed to evaluate the efficacy of Ferinject® in improving symptoms of CHF in patients with iron deficiency. Analyses will focus both on subjective and objective measures.
- Conditions
- Chronic Heart Failure, Iron Deficiency, Iron Deficiency Anemia, Anaemia
- Trial name
- FAIR-HF (NCT00520780)
- Testing
- Ferinject ® (Ferric carboxymaltose), Normal saline (0.9%)
- Where
- 48 sites in Argentina, Germany and 8 more
- Volunteers
- 456 people
- Sponsor
- Vifor Pharma
Iron Deficiency Anemia
A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with…
- Trial name
- NCT01264679
- Testing
- Ferumoxytol
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
HIV, HIV Infections
Study to Evaluate the Safety and Efficacy of Stribild Versus Ritonavir-Boosted Atazanavir Plus Truvada in Human Immunodeficiency Virus, Type 1 (HIV-1) Infected, Antiretroviral Treatment-Naive Adults
To evaluate the safety and efficacy of Stribild®, a single tablet regimen (STR) containing fixed doses of elvitegravir (EVG)/cobicistat (COBI [GS-9350])/emtricitabine (FTC)/tenofovir disoproxil fumarate (TDF) versus…
- Trial name
- NCT01106586
- Testing
- Stribild, ATV, Ritonavir
- Where
- 146 sites in United States, Australia and 15 more
- Volunteers
- 708 people
- Sponsor
- Gilead Sciences
Fed
Bioavailability Study of Ciprofloxacin 1000 mg ER Tablets of Dr. Reddy's Under Non-Fasting Conditions
The purpose of this study is to compare the relative bioavailability of ciprofloxacin 1000 mg ER tablets of Dr.Reddy's with that of CIPRO® XR 1000 mg of Bayer Pharmaceuticals Corporation in healthy, adult, non-smoking subjects…
- Trial name
- NCT01372358
- Testing
- CIPRO®XR
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Chronic Kidney Disease
Open-label Pharmacokinetic Study of Iron Isomaltoside 1000 (Monofer®) Administered as Single Bolus Injections in Subjects With Stage 5 Chronic Kidney Disease on Dialysis Therapy (CKD-5D)
The purpose of this study is to assess Pharmakokinetic properties of iron isomaltoside 1000 (Monofer®) in doses of 100 mg, 200 mg or 500 mg in subjects with Stage 5 Chronic Kidney Disease on Dialysis Therapy (CKD-5D).
- Trial name
- NCT01469078
- Testing
- Monofer®
- Volunteers
- 18 people
- Sponsor
- Pharmacosmos A/S
Infection, Human Immunodeficiency Virus
Prospective Epidemiological Study Of The Prevalence Of HLA-B*5701
This is a study to determine the prevalence of a genetic marker, HLA-B5701, in the UK population. HLA-B*5701 has been strongly associated with the risk of an allergic reaction to a HIV medicine, Abacavir.
- Trial name
- NCT00453440
- Testing
- Cheek swab & blood test
- Where
- 8 sites in United Kingdom
- Volunteers
- 1,502 people
- Sponsor
- ViiV Healthcare
HIV Infections
Pharmacokinetics and Efficacy of Multiple Dosing of Lipovirtide for Injection in HIV-infected Patients
Primary Objectives 1.Evaluation of safety and tolerability after repeated administration of injectable Lipivirtide in HIV-infected patients not receiving antiretroviral therapy Secondary Objectives 1.
- Trial name
- NCT05349968
- Testing
- Lipovirtide for injection
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Shanxi Kangbao Biological Product Co., Ltd.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- NCT01886378
- Testing
- UX007
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 29 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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