Clinical trials: Deficiency Disease
Hypertriglyceridemia, Obesity
Setmelanotide in a Single Patient With Partial Lipodystrophy
The objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent…
- Trial name
- NCT03262610
- Testing
- Setmelanotide
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Iron Deficiency Anaemia, Iron Deficiency Anemia
Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease
Evaluation of safety and efficacy of iron isomaltoside/ferric derisomaltose compared with iron sucrose, in subjects with both non-dialysis-dependent chronic kidney disease (NDD-CKD) and iron deficiency anaemia (IDA).
- Conditions
- Iron Deficiency Anaemia, Iron Deficiency Anemia, Chronic Kidney Disease
- Trial name
- NCT02940860
- Testing
- Iron isomaltoside/ferric derisomaltose, Iron sucrose
- Where
- 141 sites in United States
- Volunteers
- 1,538 people
- Sponsor
- Pharmacosmos A/S
Growth Hormone Deficiency
Evolution Of Growth Rate In Children With Growth Retardation Due to Glucocorticosteroid Therapy And Treated By Genotonorm
To estimate the evolution of height and growth rate over 5 years of growth hormone (GH) treatment To estimate the prognostic factors of growth rate with GH treatment To confirm the good clinical and biological safety of GH…
- Trial name
- NCT00163189
- Testing
- Somatropin
- Where
- 19 sites in France
- Volunteers
- 98 people
- Sponsor
- Pfizer
HIV Infections
A Phase I/II Clinical Study of Nystatin I.V. (Intravenous) in Patients With HIV Infection.
To evaluate the clinical toxicity, safety, and maximum tolerated dose (MTD) of intravenous nystatin in patients with HIV infection. To evaluate the potential anti-HIV activity and clinical pharmacology of intravenous nystatin.
- Trial name
- NCT00002097
- Testing
- Nystatin
- Where
- United States
- Sponsor
- Argus Pharmaceuticals
Primary Immune Deficiency Disorder
A Study About Low Blood Pressure in Patients With Primary Immunodeficiency Disease Treated With Immune Globulin Products
This is a safety study to evaluate the risk of low blood pressure in subjects with Primary Immune Deficiency disorder (PID) treated with Bivigam™ or another commercial product under real world conditions.
- Trial name
- NCT03037359
- Testing
- Bivigam, Other
- Where
- 17 sites in United States
- Volunteers
- 200 people
- Sponsor
- ADMA Biologics, Inc.
Coronary Artery Disease
Serial OCT Evaluation of Tissue Coverage in Patients Submitted to Inspiron Drug Eluting Stent Implantation
Prospective, multicentric, single-arm study to evaluate fast and effective tissue repair in patients undergone percutaneous coronary intervention with drug eluting stent Inspiron.
- Trial name
- REPAIR (NCT03269461)
- Testing
- Angiography and Optical Coherence Tomography evaluations
- Where
- 5 sites in Brazil
- Volunteers
- 60 people
- Sponsor
- Scitech Produtos Medicos SA
Dry Eye Syndromes, Keratoconjunctivitis Sicca
Six Month Study to Evaluate the Safety and Effectiveness of the Intranasal Lacrimal Neurostimulator
In this study, the safety and effectiveness of the Oculeve Intranasal Lacrimal Neurostimulator after 180 days of use in participants with aqueous tear deficiency will be evaluated.
- Trial name
- NCT02526290
- Testing
- Intranasal Lacrimal Neurostimulator (Oculeve)
- Where
- 3 sites in United States
- Volunteers
- 97 people
- Sponsor
- Oculeve, Inc.
HIV Infection
Safety, Tolerability and Immunogenicity Induced by the THV01 Treatment in Patients Infected With HIV-1 Clade B and Treated With Highly Active Antiretroviral Therapy (HAART).
The objectives of this Phase I/II trial is to evaluate the safety, tolerability and immunogenicity of THV01 compared to placebo in HIV-1 infected patients on HAART (highly active antiretroviral therapies).
- Trial name
- NCT02054286
- Testing
- THV01-1, THV01-2
- Where
- 11 sites in Belgium, France
- Volunteers
- 38 people
- Sponsor
- Theravectys S.A.
Iron Deficiency Anemia
Iron Indices and Intravenous Ferumoxytol: Time to Steady State
The purpose of this study is to evaluate the time point of transferrin saturation (TSAT) and ferritin stabilization after a thirteen-treatment period following a ferumoxytol load, as well as to determine the point at which serum…
- Trial name
- NCT01148745
- Testing
- ferumoxytol
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Dialysis Clinic, Inc.
HIV Infections
Safety and Effectiveness of TRIZIVIR (Abacavir/Lamivudine/Zidovudine) With Efavirenz in HIV-Infected Patients Who Have Never Taken Anti-HIV Drugs
The purpose of this study is to determine the effect of treatment with Trizivir (TZV) plus efavirenz (EFV) or TZV alone on viral load (level of HIV in the blood).
- Trial name
- NCT00011895
- Testing
- Abacavir sulfate, Lamivudine and Zidovudine, Efavirenz
- Where
- 42 sites in United States
- Volunteers
- 400 people
- Sponsor
- GlaxoSmithKline
Acquired Immune Deficiency Syndrome (AIDS), HIV Infections
Study of Cobicistat-Boosted Atazanavir (ATV/co), Cobicistat-Boosted Darunavir (DRV/co) and Emtricitabine/Tenofovir Alafenamide (F/TAF) in Children With HIV
The goal of this clinical study is to learn more about the safety and dosing of study drugs, cobicistat-boosted Atazanavir (ATV/co), cobicistat-boosted darunavir (DRV/co) and emtricitabine/tenofovir alafenamide (F/TAF), in…
- Trial name
- NCT02016924
- Testing
- ATV, DRV, Cobicistat
- Where
- 23 sites in United States, Argentina and 5 more
- Volunteers
- 133 people
- Sponsor
- Gilead Sciences
Lysosomal Acid Lipase Deficiency, Wolman Disease
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
- Trial name
- NCT01358370
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Human Immunodeficiency Virus Infection
Treatment Perception of QD (Once a Day) Dosed Kaletra (Tablets)
Both twice-a-day (BID) and once-a-day (QD) dosing are approved in Europe for the protease inhibitor lopinavir/ritonavir (LPV/r; Kaletra®).
- Trial name
- QD-Kapital (NCT01383005)
- Where
- 20 sites in Spain
- Volunteers
- 97 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Cytomegalovirus Infections, HIV Infections
A Multiple Dose Crossover Pharmacokinetics Study to Evaluate the Effects of Food on the Absorption of Oral Ganciclovir
This study is designed to determine the influence of food on the absorption and relative bioavailability of oral ganciclovir by comparing the absorption of oral ganciclovir in a fed and fasting state at steady state plasma levels.
- Trial name
- NCT00002251
- Testing
- Ganciclovir
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Hoffmann-La Roche
HIV Infections
A Study of Indinavir Sulfate Given Together With Stavudine to HIV-Positive Patients
The purpose of this study is to see if it is safe and effective to give indinavir sulfate plus stavudine to HIV-infected patients who have already been treated with zidovudine.
- Trial name
- NCT00002354
- Testing
- Indinavir sulfate, Stavudine
- Where
- United States
- Volunteers
- 540 people
- Sponsor
- Merck Sharp & Dohme LLC
Growth Hormone Deficiency
Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
This is an open label randomized 24 week crossover trial assessing the treatment burden of a weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone injection regimen (Genotropin).
- Trial name
- NCT03831880
- Testing
- Genotropin, somatrogon
- Where
- 32 sites in United States, Bulgaria and 3 more
- Volunteers
- 87 people
- Sponsor
- Pfizer
Cryptosporidiosis, HIV Infections
Azithromycin in the Treatment of Cryptosporidiosis in Patients With Acquired Immune Deficiency Syndrome (AIDS): A Randomized, Multi-Center, Placebo-Controlled, Double-Blind Study
To evaluate the safety and efficacy of azithromycin in the treatment of intestinal cryptosporidial infection in AIDS patients.
- Trial name
- NCT00002082
- Testing
- Azithromycin
- Where
- 4 sites in United States
- Sponsor
- Pfizer
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC
Advanced Solid Tumor
Study of CX-5461 in Patients With Solid Tumours and BRCA1/2, PALB2 or Homologous Recombination Deficiency (HRD) Mutation
This is an open-label, multi-center, phase 1b study designed to determine a tolerable dose of CX-5461 administered by IV infusion on Day 1 and Day 8 of a 28-day cycle in patients with selected solid tumours and associated…
- Trial name
- NCT04890613
- Testing
- CX-5461
- Where
- 8 sites in United States, Canada
- Volunteers
- 52 people
- Sponsor
- Senhwa Biosciences, Inc.
Primary Immunodeficiency Diseases (PID)
Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens and treatment administration…
- Trial name
- NCT02593188
- Testing
- HYQVIA
- Where
- 30 sites in United States
- Volunteers
- 264 people
- Sponsor
- Baxalta now part of Shire
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