Clinical trials: Deficiency Disease
Human Immunodeficiency Virus Type 1 (HIV-1) Infection
A Clinical Study of Islatravir and Ulonivirine for People With HIV-1 Who Have Not Been Treated Before (MK-8591B-062)
Researchers are looking for new ways to treat HIV-1 (Human Immunodeficiency Virus Type 1). The usual (standard) treatment for HIV-1 is antiretroviral therapy (ART), which includes taking medicines to lower the amount of HIV-1 in…
- Trial name
- NCT07266831
- Testing
- ISL, ULO, BIC/FTC/TAF
- Where
- 55 sites in United States, Argentina and 7 more
- Volunteers
- 570 people
- Sponsor
- Merck Sharp & Dohme LLC
Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism
Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Homozygous familial Hypercholesterolemia from blood
- Conditions
- Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism, Corneal Arcus
- Trial name
- BioHoFH (NCT03198897)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Phosphomannomutase 2 Deficiency, PMM2-CDG
A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG
This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults.
- Trial name
- NCT06892288
- Testing
- GLM101 (Part A, Double-blind), GLM101 (Part B, Open-label)
- Where
- 15 sites in United States, Belgium and 8 more
- Volunteers
- 50 people
- Sponsor
- Glycomine, Inc.
HIV Infections
Switching HIV-1 Infected Subjects From a Highly Active Anti-Retroviral Treatment (HAART) Regimen Dosed Twice Daily or More Frequently to a Once-Daily Regimen
The purpose of this study is to demonstrate that virologically controlled HIV-infected individuals can successfully switch from a standard-of-care regimen dosed twice-daily or more frequently to a simpler once-daily (QD) regimen…
- Trial name
- NCT00135369
- Testing
- stavudine extended-release, lamivudine, efavirenz
- Where
- 47 sites in United States, Puerto Rico
- Volunteers
- 300 people
- Sponsor
- Bristol-Myers Squibb
HIV, AIDS
TMC278-TiDP6-C130: Pharmacokinetics, Safety and Tolerability of TMC278 in Subjects With Mildly or Moderately Impaired Hepatic Function.
The purpose of this study is to investigate the pharmacokinetics (how the drug is absorbed into the bloodstream, distributed in the body and eliminated from the body) after a single dose and after repeated administration of…
- Conditions
- HIV, AIDS, Hepatic Impairment
- Trial name
- NCT00736905
- Testing
- TMC278
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Tibotec Pharmaceuticals, Ireland
HIV Infections
Safety and Effectiveness of Giving Indinavir, Ritonavir, Stavudine, and Lamivudine to HIV-Infected Patients Who Have Never Received Anti-HIV Drugs
The purpose of this study is to see if it is safe to give indinavir (IDV) and ritonavir (RTV) in combination with stavudine (d4T) and lamivudine (3TC) to HIV-positive patients who have never received anti-HIV therapy.
- Trial name
- NCT00002451
- Testing
- Indinavir sulfate, Ritonavir, Lamivudine
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Merck Sharp & Dohme LLC
Vitamin D Deficiency
A Study to Assess the Effectiveness of an Investigational Drug in Elderly Subjects With Low Vitamin D Levels (0822A-009)
This study will assess the effectiveness of an investigational drug in elderly subjects with low vitamin D levels.
- Trial name
- NCT00242476
- Testing
- MK0822A
- Volunteers
- 216 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Trial name
- NCT06274788
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Where
- 10 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fresenius Kabi
HIV Infections
Proof of Concept Treatment Study of Orally Administered VH4004280 or VH4011499 in HIV-1 Infected Adults
The primary purpose of the study is to evaluate the antiviral activity of orally administered VH4004280 and VH4011499 monotherapy over 10 days in human immunodeficiency virus (HIV-1) infected Treatment-Naïve (TN) participants.
- Trial name
- CINNAMON (NCT06039579)
- Testing
- VH4004280, VH4011499, Antiretroviral therapy
- Where
- 20 sites in United States, Argentina and 7 more
- Volunteers
- 44 people
- Sponsor
- ViiV Healthcare
Complete DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Safety-Efficacy
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection.
- Conditions
- Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT01220531
- Testing
- Cultured Thymus Tissue
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Growth Hormone Disorder, Adult Growth Hormone Deficiency
Investigation of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Multiple Doses of Somapacitan in Subjects With Various Degrees of Impaired Renal Function Compared to Subjects With Normal Renal Function
The trial is conducted in Europe. The aim of the trial is to investigate the steady state exposure of somapacitan in subjects with various degrees of renal impairment (mild, moderate, severe renal impairment, requiring…
- Conditions
- Growth Hormone Disorder, Adult Growth Hormone Deficiency, Growth Hormone Deficiency in Children
- Trial name
- NCT03186495
- Testing
- Somapacitan
- Where
- Germany
- Volunteers
- 44 people
- Sponsor
- Novo Nordisk A/S
HIV Positive
Evaluation of a Blood Test to Measure Immune Function in HIV Positive People Compared With HIV Negative People
The health of the immune system in HIV infected people is currently determined from a blood test measuring the number of cluster of differentiation 4 (CD4) T lymphocytes. These cells play a critical role in an immune response.
- Trial name
- QFM (NCT01904201)
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- QIAGEN Gaithersburg, Inc
HIV Infection, Hepatitis C Infection
Evaluation of HCV Care and Treatment for HIV-HCV Co-infected Patients in Decentralised Areas in Vietnam
With the advent of direct acting antiviral (DAA) treatment in 2013, HCV elimination has become feasible. Still, achieving HCV elimination in resource-limited countries appears to be arduous as several challenges need to be…
- Trial name
- MOVIDA-Hep2 (NCT05506475)
- Testing
- Blood samples
- Where
- 2 sites in Vietnam
- Volunteers
- 400 people
- Sponsor
- Institut Pasteur
HIV Infection
Abacavir/Lamivudine Versus Emtricitabine/Tenofovir Both In Combination With Lopinavir/Ritonavir For The Treatment Of HIV
This study was designed to test the safety and effectiveness of EPZICOM(abacavir/lamivudine) and TRUVADA (emtricitabine/tenofovir) for the treatment of HIV infection when both are used in combination with KALETRA…
- Trial name
- HEAT (NCT00244712)
- Testing
- emtricitabine/tenofovir, abacavir/lamivudine
- Where
- 74 sites in United States, Puerto Rico
- Volunteers
- 688 people
- Sponsor
- GlaxoSmithKline
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Healthy Subjects, Hepatic Impairment
GSK1349572 Hepatic Impairment Study
GSK1349572 is an integrase inhibitor that is currently in clinical development for the treatment of human immunodeficiency virus (HIV) infection.
- Conditions
- Healthy Subjects, Hepatic Impairment, Infection, Human Immunodeficiency Virus, HIV Infections
- Trial name
- NCT01231529
- Testing
- GSK1349572
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Growth Hormone Deficiency
Safety Study of Syntropin (Human Growth Hormone) for the Treatment of Growth Hormone Deficiency
The purpose of this study is to evaluate the pharmacokinetics of Syntropin (a human growth hormone) and to determine the serum concentration of IGF-1 after Syntropin injection.
- Trial name
- NCT00489294
- Testing
- Syntropin
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Phage Pharmaceuticals, Inc.
Pompe Disease, Glycogen Storage Disease Type II
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset…
- Trial name
- NCT00701129
- Testing
- Alglucosidase Alfa, Methotrexate, Rituximab
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
Hereditary Angioedema, Angioneurotic Edema
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening.
- Conditions
- Hereditary Angioedema, Angioneurotic Edema, Genetic Disorders
- Trial name
- NCT00262301
- Testing
- recombinant human C1 inhibitor
- Where
- 2 sites in Netherlands, Romania
- Volunteers
- 75 people
- Sponsor
- Pharming Technologies B.V.
Pmm2-CDG, Phosphomannomutase 2 Deficiency
24-Week Study to Assess the PD, Safety, Tolerability, and PK of GLM101 in Participants With PMM2-CDG
This is a Phase 2, randomized, open-label, 24-week treatment study to evaluate the potential pharmacodynamic (PD) activity, safety, tolerability, and pharmacokinetics (PK) of GLM101 in adult, adolescent, and pediatric, patients…
- Trial name
- NCT05549219
- Testing
- GLM101
- Where
- 4 sites in United States, Spain and 1 more
- Volunteers
- 27 people
- Sponsor
- Glycomine, Inc.
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