Clinical trials: Deficiency Disease
Cytomegalovirus Retinitis, HIV Infections
The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the Eyes
To evaluate the efficacy and safety of ISIS 2922 in AIDS patients with Cytomegalovirus ( CMV ) retinitis who are unresponsive or intolerant to ganciclovir and/or foscarnet but are otherwise ineligible for ISIS Pharmaceuticals'…
- Trial name
- NCT00002356
- Testing
- Fomivirsen sodium
- Where
- 25 sites in United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
Cytochrome P450 CYP2D6 Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant
Drug & Gene Interaction Risk Analysis With & Without Genetic Testing Among Patients Undergoing MTM
This randomized controlled trial will evaluate whether the use of pharmacogenetic testing through a Medication Therapy Management (MTM) program has a beneficial impact on drug therapy problems.
- Conditions
- Cytochrome P450 CYP2D6 Enzyme Deficiency, Poor Metabolizer Due to Cytochrome P450 CYP2D6 Variant, Ultrarapid Metabolizer Due to Cytochrome P450 CYP2D6 Variant, Extensive Metabolizer Due to Cytochrome P450 CYP2D6 Variant
- Trial name
- NCT02428660
- Testing
- Pharmacogenetic testing, Software-based drug & gene interaction risk analysis, MTM
- Where
- United States
- Volunteers
- 341 people
- Sponsor
- Genelex Corporation
COVID-19, SARS-CoV-2
Study of Self-Amplifying Messenger Ribonucleic Acid (samRNA) Vaccines Against COVID-19 in Healthy Adults and People Living With Human Immunodeficiency Virus (HIV)
The primary objective is to assess the safety and tolerability of samRNA vaccines GRT-R912, GRT-R914, and GRT-R918 when administered as prime and/or boost in healthy adult participants naïve to severe acute respiratory syndrome…
- Trial name
- NCT05435027
- Testing
- GRT-R912, samRNA-Spikebeta-TCE11, GRT-R914, samRNA-Spikebeta-TCE9, GRT-R918, samRNA-SpikeOmicron-N-TCE11
- Where
- 4 sites in South Africa
- Volunteers
- 342 people, healthy volunteers welcome
- Sponsor
- Gritstone bio, Inc.
Gynecomastia, Male Breast Enlargement
UltraShape Device for Non-Invasive Fat Reduction in the Male Breast (Pseudogynecomastia)
Male volunteers who suffer from Pseudogynecomastia and seek noninvasive breast fat reduction will be enrolled into two arms to receive three biweekly UltraShape treatments.
- Trial name
- NCT02711605
- Testing
- UltraShape focused ultrasound device
- Volunteers
- healthy volunteers welcome
- Sponsor
- Syneron Medical
HIV Infection
An Open Label Trial of TMC114/RTV in HIV-1 Infected, Treatment-experienced Subjects.
The primary objective of the TMC114-C215 study is to evaluate the safety and tolerability of TMC114/RTV over time. The secondary objectives are to evaluate the antiviral activity over time and to evaluate the immunological effect…
- Trial name
- NCT00081588
- Testing
- TMC114
- Where
- 64 sites in United States, Argentina and 11 more
- Volunteers
- 555 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
CDG Ib
Study of ORL-1M (D-mannose) in Patients With CDG-Ib
Study of ORL-1M in Patients With CDG-Ib
- Trial name
- NCT03404869
- Testing
- ORL-1M - D-mannose
- Where
- Turkey (Türkiye)
- Volunteers
- 5 people
- Sponsor
- Orpha Labs
Primary Immunodeficiency
CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL
Summary for SCGAM-03: Clinical phase III study to monitor the safety, tolerability and efficacy of subcutaneous human immunoglobulin (Octanorm) in patients with primary immunodeficiency diseases who have completed the SCGAM-01…
- Trial name
- NCT03907241
- Testing
- Octanorm 16.5%
- Where
- 7 sites in United States, Canada
- Volunteers
- 27 people
- Sponsor
- Octapharma
Mycoses, Mycobacterium Avium-Intracellulare Infection
A Randomized Study of Daily and Intermittent Prophylactic Regimens for the Prevention of Disseminated Mycobacterium Avium Complex (MAC) and Fungal Infections in HIV-Infected Patients
PRIMARY: To determine the efficacy of azithromycin and rifabutin alone and in combination for the prevention of disseminated Mycobacterium avium Complex (MAC) infection in HIV-infected patients.
- Conditions
- Mycoses, Mycobacterium Avium-Intracellulare Infection, HIV Infections
- Trial name
- NCT00002122
- Testing
- Azithromycin, Rifabutin, Fluconazole
- Where
- 12 sites in United States
- Volunteers
- 720 people
- Sponsor
- Pfizer
HIV Infections
A Phase I Study of Three Doses of OPC-8212 (Vesnarinone) in HIV-Infected Persons With CD4+ Cell Number > 300 Cells/mm3
To examine the safety and tolerance of three doses of oral vesnarinone in HIV-infected patients with CD4 count > 300 cells/mm3.
- Trial name
- NCT00002129
- Testing
- Vesnarinone
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Otsuka America Pharmaceutical
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
LPL Deficiency
Alipogene Tiparvovec for the Treatment of LPLD Patients
The aim of the study is to provide further confirmatory evidence of clinical benefit in LPLD patients treated with alipogene tiparvovec by assessing both the "clinical response" (as defined by a range of parameters), and "the…
- Trial name
- NCT02904772
- Testing
- alipogene tiparvovec, Prednisolone, Cyclosporins
- Where
- 2 sites in United States, Canada
- Sponsor
- UniQure Biopharma B.V.
Neoplasms
Phase I Dose Escalation Study for VIP152 in Patients With Advanced Cancer
Determine the safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of VIP152 (BAY 1251152) as monotherapy or in combination in patients with solid tumors and aggressive…
- Trial name
- NCT02635672
- Testing
- VIP152 (BAY 1251152), VIP152 (BAY 1251152) 30 mg, Keytruda
- Where
- 16 sites in United States, Chile and 1 more
- Volunteers
- 110 people
- Sponsor
- Vincerx Pharma, Inc.
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
To Determine Bioequivalence Under Fed Conditions
Bioavailability Study of Leflunomide Tablets Under Fed Conditions
To compare the single-dose Bioavailability of Kali and Aventis
- Trial name
- NCT00653003
- Testing
- Leflunomide, ARAVA
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Iron Deficiency Anemia (IDA)
Observational Study on the Use of IV (Intravenous) Iron Therapy Among Subjects With Iron Deficiency Anemia
The primary objective of this study is to compare the total costs to sites and payers of administering IV iron over the 30-day observation period for subjects with IDA who receive Ferric Carboxymaltose (FCM) relative to those who…
- Trial name
- NCT01984554
- Where
- United States
- Volunteers
- 551 people
- Sponsor
- American Regent, Inc.
Iron-Deficiency Anemia, Iron-Deficiency
Real Life Study on Iron Isomaltoside 1000 in the Treatment of ID in CKD, Heart Failure, ObGyn, IBD, Cancer and Elective Surgery (Real-CHOICE).
Real-CHOICE - designed as a prospective, longitudinal, observational, non-interventional study - will investigate the attitude of patients and physicians towards IV (intravenous) iron therapy in general and IIM (iron isomaltoside…
- Trial name
- Real-CHOICE (NCT04318405)
- Testing
- Iron isomaltoside 1000
- Where
- 21 sites in Switzerland
- Volunteers
- 327 people
- Sponsor
- Pierre Fabre Pharma AG
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia…
- Trial name
- NCT04188964
- Testing
- Burosumab
- Where
- 9 sites in Austria, France and 4 more
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Familial Lipoprotein Lipase Deficiency
Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]
LPLD is a rare autosomal recessive disorder, characterized by the presence of marked chylomicronemia and hence hypertriglyceridemia.
- Trial name
- NCT01109498
- Testing
- Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil, cyclosporine
- Where
- Canada
- Volunteers
- 14 people
- Sponsor
- Amsterdam Molecular Therapeutics
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Comparison of a Blood Clotting Drug (Recombinant Factor XIII) Produced by Two Different Manufacturers in Healthy Male Subjects
This trial is conducted in Europe. The aim of this clinical trial is to compare the metabolism of a blood-clotting drug (recombinant Factor XIII)) produced by two different manufacturers (Novo Nordisk and Avecia) in healthy male…
- Conditions
- Congenital Bleeding Disorder, Congenital FXIII Deficiency, Healthy
- Trial name
- NCT01082406
- Testing
- catridecacog, recombinant factor XIII
- Where
- United Kingdom
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
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