Clinical trials: Deficiency Disease
Alpha-Mannosidosis
Expanded Access to Velmanase Alfa
Individual patient expanded access requests may be considered for patients who have no other treatment options.
- Trial name
- NCT04959240
- Testing
- Velmanase Alfa
- Sponsor
- Chiesi Farmaceutici S.p.A.
HIV Infections
A Randomised Trial to Evaluate the Antiviral Efficacy and Safety of Treatment With 500 mg Tipranavir (TPV) Plus 100 mg or 200 mg Ritonavir (RTV) p.o. BID in Comparison to 400 mg Lopinavir (LPV) Plus 100 mg RTV p.o. BID in Combination With Standard Background Regimen in ARV Therapy naïve Patients.
Evaluation of safety and efficacy of Tipranavir (TPV) boosted with Ritonavir (RTV) versus an active control arm (Lopinavir / RTV) in antiretroviral (ARV) therapy naïve HIV-1 infected patients
- Trial name
- NCT00144105
- Testing
- TPV500mg/RTV200mgBID, TPV500mg/RTV100mgBID, LPV400mg/RTV100mgBID
- Where
- 81 sites in Argentina, Australia and 13 more
- Volunteers
- 562 people
- Sponsor
- Boehringer Ingelheim
Alpha-Mannosidosis
Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis
Randomized, double-blind, placebo-controlled, parallel group study where subjects will receive velmanase alfa or placebo for 24 weeks.
- Trial name
- SHAMAN (NCT04031066)
- Testing
- Velmanase Alfa
- Sponsor
- Chiesi Farmaceutici S.p.A.
HIV Infections, Cytopenias
Treatment Program for Anemia in AIDS Patients
The objective of the Eprex (erythropoietin) Treatment Program is to provide erythropoietin for the treatment of anemia in AIDS patients.
- Trial name
- NCT00002022
- Testing
- Epoetin alfa
- Where
- United States
- Sponsor
- R W Johnson Pharmaceutical Research Institute
Familial Chylomicronemia Syndrome
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT06360237
- Testing
- Olezarsen
- Where
- United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
Arginase I Deficiency, Hyperargininemia
A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D.
- Trial name
- NCT05676853
- Testing
- Pegzilarginase
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Aeglea Biotherapeutics
HIV Infections
A Study of Retrovir and Epivir Alone or in Combination With 141W94 in HIV-Infected Patients
The purpose of this study is to see if it is safe and effective to add 141W94 to an anti-HIV regimen that includes retrovir plus epivir.
- Trial name
- NCT00002195
- Testing
- Amprenavir, Lamivudine, Zidovudine
- Where
- 9 sites in United States
- Volunteers
- 290 people
- Sponsor
- Glaxo Wellcome
HIV Infections
Induction-Maintenance With Atazanavir in HIV Naïve Patients (The INDUMA Study)
The purpose of this study is to compare the proportion of subjects with HIV-1 RNA viral load < 50 c/mL through Week 48 of the Maintenance Phase among HIV-infected subjects with an initial undetectable viral load following an…
- Trial name
- INDUMA (NCT00207142)
- Testing
- Atazanavir + 2 NRTIs, Atazanavir + Ritonavir + 2 NRTIs
- Where
- 27 sites in Estonia, France and 8 more
- Volunteers
- 252 people
- Sponsor
- Bristol-Myers Squibb
Infection, Human Immunodeficiency Virus
Relative Bio-availability Study of Dolutegravir and Lamivudine Fixed Dose Combinations
Dolutegravir (DTG) and lamivudine (3TC) are indicated in combination with other antiretroviral agents for the treatment of human immunodeficiency virus type 1 (HIV-1) infection.
- Trial name
- NCT02738931
- Testing
- Dolutegravir 50 mg tablet, Lamivudine 300 mg tablet, Dolutegravir/Lamivudine 50 mg/300 mg Tablet (Product Code AA)
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Cytomegalovirus Retinitis, HIV Infections
An Open Study of Foscarnet Treatment First Episode CMV-Retinitis in AIDS Patients
To evaluate the safety and efficacy of foscarnet induction therapy for treatment of AIDS patients experiencing their first episode of cytomegalovirus (CMV) retinitis.
- Trial name
- NCT00002039
- Testing
- Foscarnet sodium
- Where
- United States
- Sponsor
- Astra USA
Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.
- Trial name
- NCT00410566
- Testing
- rhASM
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
Healthy
Bioequivalence Study of Terbinafine Hydrochloride Tablets, 250 mg of Dr. Reddy's Under Fed Conditions
The purpose of this study is to compare the rate and extent of absorption of terbinafine hydrochloride 250 mg tablets versus Lamisil® 250 mg tablets administered as 1 x 250 mg tablet under fed conditions.
- Trial name
- NCT01286701
- Testing
- Terbinafine Hydrochloride Tablets, 250 mg
- Where
- Canada
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
HIV Infections
A Randomized, Double-Blind Study of MKC-442 Combined With Viracept in Patients Who Are Epivir + Retrovir Experienced and Are Protease Inhibitor- and Non-Nucleoside Reverse Transcriptase Inhibitor-Naive
To compare the proportion of patients whose plasma HIV-1 RNA level falls and remains below the limit of quantification by the Roche Amplicor Monitor (400 copies/ml)[AS PER AMENDMENT 8/4/98: 50 copies/ml] between weeks 0 and 24.
- Trial name
- NCT00002215
- Testing
- Emivirine, Nelfinavir mesylate
- Where
- United States
- Sponsor
- Triangle Pharmaceuticals
HIV Infections
Bioavailability Study of 10 Milligram (mg) and 5 mg Tablets Versus Conventional Tablets of Dolutegravir
The aim of this cross-over study is to compare the relative bioavailability and pharmacokinetic parameters of both 10 mg conventional tablets and 5 mg dispersible tablets of dolutegravir (DTG) with that of 25 mg or 50 mg…
- Trial name
- NCT03095638
- Testing
- Conventional dolutegravir 10 mg tablet, Conventional dolutegravir 50 mg tablet, Dispersible dolutegravir 5 mg tablet
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
Primary Immune Deficiency Disorders (PIDD)
Open Label, Phase III Study of NABI-IGIV 10% [Immune Globulin Intravenous(Human), 10%] In Subjects With Primary Immune Deficiency Disorders (PIDD)
The purpose of this study is to determine if NABI-IGIV (10%) [Immune Globulin Intravenous (Human), 10%] is safe and effective in preventing serious bacterial infections (SBIs) in the treatment of patients with primary immune…
- Trial name
- NCT00538915
- Testing
- Nabi-IGIV 10% [Immune Globulin Intravenous (Human). 10%]
- Where
- 17 sites in United States
- Volunteers
- 63 people
- Sponsor
- ADMA Biologics, Inc.
HIV Infections
Safety of and Immune Response to an HIV-1 Subtype C Vaccine (AVX101) in HIV Uninfected Adults
The purpose of this study is to evaluate the safety of and immune response to an alphavirus replicon, HIV-1 subtype C gag vaccine, AVX101, in HIV uninfected adults in the United States, South Africa, and Botswana.
- Trial name
- NCT00097838
- Testing
- AVX101
- Where
- 8 sites in United States, Botswana and 1 more
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- AlphaVax, Inc.
Infection, Human Immunodeficiency Virus
Study to Compare the Safety and Anti-HIV Effect of GSK1265744 Versus Placebo in HIV-1 Infected Adults (ITZ112929)
The purpose of this randomized, double-blinded study is to test the safety of GSK1265744 and how well it works on reducing the amount of HIV in the blood.
- Trial name
- NCT00920426
- Testing
- GSK1265744 30mg, GSK1265744 5mg
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- ViiV Healthcare
Hypophosphatasia
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01163149
- Testing
- asfotase alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 19 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
HIV Infections
Safety and Effectiveness of 3 Anti-HIV Treatments in Patients Who Have Failed Previous Treatments Containing Nelfinavir
The purpose of this study is to see if 3 anti-HIV drug combinations are safe and effective in patients who have failed previous anti-HIV treatments using nelfinavir (NFV).
- Trial name
- NCT00006326
- Testing
- Indinavir sulfate, Ritonavir, Delavirdine mesylate
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Agouron Pharmaceuticals
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.