Clinical trials: Deficiency Disease
Growth Hormone Deficiency, Growth Retardation
Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) In Children With Short Stature Secondary
To assess the effect of long-term treatment by Genotonorm on linear growth
- Trial name
- NCT00174278
- Testing
- Somatropin
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Pfizer
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
HIV Infections
Study to Explore Safety And Tolerability of Fosamprenavir With or Without Ritonavir in Combination With TRIZIVIR or COMBIVIR
Antiretroviral Therapy (ART) naive subjects will be enrolled in this clinical research study to test the safety and tolerability of fosamprenavir with or without ritonavir in combination TRIZIVIR and COMBIVIR.
- Trial name
- NCT00043888
- Testing
- fosamprenavir, COMBIVIR, ritonavir
- Where
- 18 sites in United States, France and 1 more
- Volunteers
- 60 people
- Sponsor
- GlaxoSmithKline
Pneumonia, Pneumocystis Carinii, HIV Infections
A Controlled Trial Comparing the Efficacy of Aerosolized Pentamidine and Parenteral / Oral Trimethoprim - Sulfamethoxazole in the Treatment of Pneumocystis Carinii Pneumonia in AIDS
This is a randomized double-blinded controlled study comparing aerosolized pentamidine with trimethoprim plus sulfamethoxazole (TMP / PurposeX).
- Trial name
- NCT00002056
- Testing
- Pentamidine isethionate
- Where
- United States
- Sponsor
- LyphoMed
Iron-deficiency
Study of the Prevalence of Iron Deficiency in Elderly Patient in Hospital Environment
Iron deficiency is defined by insufficient tissue iron stores and anemia is the ultimate stage of iron deficiency. Anemia should never be neglected in an elderly subject because it is associated with an increase in mortality, but…
- Trial name
- CARENFERPA (NCT05514951)
- Testing
- Blood sampling
- Where
- 16 sites in France
- Volunteers
- 888 people
- Sponsor
- Vifor Pharma
HIV Infection
Study of Adherence Effects and Clinical Outcomes of Kaletra Based HIV Antiviral Therapy
The purpose of this study is to evaluate the effects of adherence on HIV antiviral response to treatment regimens containing Kaletra or other HIV protease inhibitors.
- Trial name
- NCT00234962
- Testing
- Lopinavir/Ritonavir
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Abbott
Growth Hormone Deficiency With Pituitary Anomalies
Validation of Macimorelin as a Test for Adult Growth Hormone Deficiency
The Macimorelin Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, 2-way crossover Trial.
- Trial name
- NCT02558829
- Testing
- Macimorelin, Insulin
- Where
- 27 sites in United States, Austria and 7 more
- Volunteers
- 157 people, healthy volunteers welcome
- Sponsor
- AEterna Zentaris
Growth Hormone Deficiency
A Long-Term Safety Study of Somavaratan in Japanese Children With Growth Hormone Deficiency
This study is a multi-center, open-label safety study assessing long-term somavaratan administration.
- Trial name
- NCT03145831
- Testing
- Somavaratan
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Versartis Inc.
HIV Infections
An Open-Label, Crossover Study to Assess the Effect of Food on the Oral Bioavailability and Pharmacokinetic Profile of 3'-Deoxy-3'-Fluorothymidine (FLT) in Asymptomatic Human Immunodeficiency Virus (HIV)-Infected Subjects
To characterize the pharmacokinetics of orally administered FLT (in a liquid formulation) after single doses in both the fed and fasting states; to assess the effect of food on the oral bioavailability of FLT
- Trial name
- NCT00002260
- Testing
- Alovudine
- Where
- United States
- Sponsor
- Lederle Laboratories
IgA Nephropathy, Lupus Nephritis (LN)
A Phase 2 Study to Evaluate the Safety, PD, PK, and Clinical Activity of ADX-097 in Participants With IgAN, LN or C3G
A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Clinical Activity of ADX-097 Administered Subcutaneously in Male and Female Participants Aged 16 Years or Older with Immunoglobulin A Nephropathy…
- Conditions
- IgA Nephropathy, Lupus Nephritis (LN), C3 (Complement Component 3) Glomerulopathy
- Trial name
- NCT06419205
- Testing
- ADX-097
- Where
- 4 sites in United States, Brazil
- Volunteers
- 30 people
- Sponsor
- Akebia Therapeutics
Anemia, Iron-Deficiency, Kidney Failure, Chronic
Effect of Ferrlecit® Versus Oral Iron on Iron Deficient Chronic Kidney Disease (CKD) Patients Receiving Erythropoietic Agents
This study compares the effect of Ferrlecit® (a form of intravenous iron) to ferrous sulfate (a form of oral iron) in treating anemia and iron deficiency in chronic kidney disease patients who are receiving erythropoietic agents…
- Trial name
- NCT00224042
- Testing
- Sodium Ferric Gluconate complex in sucrose, Ferrous sulfate tablets
- Where
- 24 sites in United States, Puerto Rico
- Volunteers
- 52 people
- Sponsor
- Watson Pharmaceuticals
HIV Infections
Study of Itraconazole in Patients With Advanced HIV Infection
To demonstrate a dosage regimen for intravenous itraconazole that produces a plasma concentration range comparable to that obtained after currently used oral dosages of itraconazole oral solution; and to obtain preliminary safety…
- Trial name
- NCT00002370
- Testing
- Itraconazole
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Janssen, LP
Infection, Human Immunodeficiency Virus
Relative Bioavailability Study With BMS-955176
The purpose of this study is to select the best dose level (amount of drug given) and best formulation of the study drug (BMS-955176) to develop further.
- Trial name
- NCT02095886
- Testing
- BMS-955176
- Where
- United Kingdom
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Iron Deficiency Anemia
A Single Ascending Study of IOP Injection in Patients With Iron Deficient Anemia
The purpose is to evaluate the efficacy and safety of IOP Injection (MPB-1514) for the treatment of iron-deficient anemia (IDA).
- Trial name
- NCT03485053
- Testing
- IOP Injection / MPB-1514
- Where
- 8 sites in United States
- Volunteers
- 49 people
- Sponsor
- MegaPro Biomedical Co. Ltd.
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
Tetrahydrobiopterin Deficiency
Retrospective Observational Safety Effectiveness With Kuvan in hpA
A retrospective study to collect the effectiveness and safety data of the past treatment with KUVAN in Chinese patients with HPA caused by BH4 deficiency.
- Trial name
- ROSEKA (NCT03864029)
- Testing
- KUVAN
- Where
- China
- Volunteers
- 26 people
- Sponsor
- BioMarin Pharmaceutical
Hereditary Angioedema
An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates
This is an open-label, single sequence study to evaluate the effect of BCX7353 on hepatic and intestinal cytochrome P450 enzymes using probe substrate drugs in healthy subjects.
- Trial name
- NCT02819102
- Testing
- BCX7353 and probes
- Where
- United Kingdom
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
HIV Infections
Phase II/III Study of the Tolerance and Efficacy of Combined Use of Didanosine (2',3'-Dideoxyinosine; ddI) and Lentinan in HIV-Positive Patients
To determine the tolerance and side effects of a combination of lentinan and didanosine (ddI) in comparison with ddI alone. To determine whether the combination of lentinan and ddI produces a significant immunorestorative effect…
- Trial name
- NCT00002099
- Testing
- Lentinan, Didanosine
- Where
- United States
- Sponsor
- AJI Pharma USA
Infections, Human Immunodeficiency Virus and Tuberculosis
A Phase 1, Open Label, Placebo-Controlled Study to Evaluate the Effect of GSK1349572 on Iohexol and Para-Aminohippurate Clearance in Healthy Subjects
The current study is designed to confirm the mechanism behind the increase in serum creatinine observed during GSK1349572 therapy; specifically, the study will determine whether GSK1349572 has any effect on glomerular filtration…
- Trial name
- NCT01214993
- Testing
- GSK1349572, Iohexol Injection, Para-aminohippurate infusion
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
MBL-Deficient, Neutropenia
Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.
- Trial name
- NCT00138736
- Testing
- Mannan Binding Lectin (MBL), Mannan Binding Lectin
- Where
- 2 sites in Netherlands
- Volunteers
- 12 people
- Sponsor
- Prothya Biosolutions
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