Clinical trials: Deficiency Disease
Growth Hormone Deficiency
Molecular Basis of the Growth Axis in Short Stature
To examine the clinical genetic and biochemical characteristics of children with growth hormone deficiency.
- Trial name
- NCT00453245
- Where
- Israel
- Sponsor
- HaEmek Medical Center, Israel
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Identification of Undiagnosed Lysosomal Acid Lipase Deficiency
Partners HealthCare maintains a Patient Data Registry (PDR) with information from all patient encounters at Partners HealthCare facilities.
- Trial name
- NCT01716728
- Testing
- enzyme analysis
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Massachusetts General Hospital
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
Candidiasis, Esophageal, HIV Infections
Evaluation of Amphotericin B in the Treatment of Biopsy Proven Candida Esophagitis in Immunocompromised Patients
To determine the appropriate duration of amphotericin B therapy for Candida esophagitis. To compare the effectiveness of two different amphotericin B doses in the treatment of biopsy-proven Candida esophagitis.
- Trial name
- NCT00002041
- Testing
- Amphotericin B
- Where
- United States
- Sponsor
- Bristol-Myers Squibb
Primary Immunodeficiency (PID)
Subcutaneous Ig NextGen 16% in PID Patients
This study aims to assess the safety, tolerability, efficacy and pharmacokinetics of Ig NextGen 16% in people with antibody deficiency currently being treated with IntragamP.
- Trial name
- NCT00391131
- Testing
- IgNextGen 16%
- Where
- 12 sites in Australia, New Zealand
- Volunteers
- 35 people
- Sponsor
- CSL Limited
Lymphoma, Non-Hodgkin, HIV Infections
Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma
To estimate the response rate, overall and disease-free survival, toxicities, factors associated with outcome, and effect on quality of life in patients with AIDS-related primary CNS lymphoma treated with CHOD (cyclophosphamide…
- Trial name
- NCT00000801
- Testing
- Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride
- Where
- 7 sites in United States
- Volunteers
- 33 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV Infections
Lopinavir/Ritonavir Monotherapy in Children
The purpose of this study is to evaluate the efficacy (clinical, immunological, virological outcome), pharmacokinetics and safety of lopinavir/ritonavir (LPV/r) monotherapy maintenance in Thai children after viral load…
- Trial name
- NCT00626301
- Testing
- LPV/r
- Where
- 3 sites in Thailand
- Volunteers
- 40 people
- Sponsor
- The HIV Netherlands Australia Thailand Research Collaboration
Chronic Granulomatous Disease
Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease
This study will investigate the safety and effectiveness of a modified stem cell transplant procedure for treating chronic granulomatous disease (CGD) in patients with active infection.
- Trial name
- NCT00001765
- Testing
- Nexell Isolex with T-cell Depletion, Baxter isolex 300i
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
LRBA Deficiency, CTLA4 Haploinsufficiency
New Biomarkers for Diagnosis and Follow-up of Patients With LRBA or CTLA4 Deficiencies
Primary immune deficiencies (PID) are a group of chronic diseases characterized by recurrent infections. Apart from recurrent infections, in some of PIDs autoimmunity, allergy or malignancy could be accompanied to the diseases.
- Trial name
- NCT04377867
- Testing
- Abatacept Injection [Orencia]
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Marmara University
GLILD in a Population of Children and Young Adults
GLILD Diagnosed in Children and Young Adults With Common Variable Immunodeficiency
8 to 22% of patients with common variable immunodeficiency (CVID) will develop Granulomatous Lymphocytic Interstitial Lung Disease (GLILD), which has emerged as a major cause of mortality.
- Trial name
- pGLILD (NCT03648567)
- Where
- 6 sites in France
- Volunteers
- 24 people
- Sponsor
- Central Hospital, Nancy, France
Lung Cancer, Malignant Mesothelioma
Alanosine in Treating Patients With Cancer
RATIONALE: Drugs used in chemotherapy such as alanosine use different ways to stop tumor cells from dividing so they stop growing or die.
- Conditions
- Lung Cancer, Malignant Mesothelioma, Pancreatic Cancer, Sarcoma
- Trial name
- NCT00062283
- Testing
- L-alanosine
- Where
- 12 sites in United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
HIV, AIDS
EuroSIDA As An External Comparator To MOTIVATE Trials
Human Immunodeficiency Virus (HIV) infected patients who meet the entry criteria for the maraviroc pivotal trials (Maraviroc versus Optimized Therapy in Viremic Antiretroviral Treatment-Experienced Patients, MOTIVATE) will be…
- Trial name
- NCT01329783
- Testing
- maraviroc
- Volunteers
- 1,181 people
- Sponsor
- ViiV Healthcare
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
Sarcoma, Kaposi, HIV Infections
A Phase I/II Study of Recombinant Interleukin-4 in AIDS and Kaposi's Sarcoma
To determine the safety and tolerance of interleukin-4 (IL-4) in patients with AIDS-related Kaposi's sarcoma. To determine the effects of IL-4 on tumor growth in patients with AIDS-related Kaposi's sarcoma.
- Trial name
- NCT00000769
- Testing
- Interleukin-4
- Where
- 3 sites in United States
- Volunteers
- 48 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV Positive
Oligonucleotide Ligation Assay (OLA) Resistance Study
The investigators propose to gauge improvements in the rate of durable suppression of viral replication by ART when OLA is used to guide clinical decisions at the PEPFAR Coptic Hope Center in Kenya, and to determine the…
- Trial name
- OLA (NCT01898754)
- Testing
- Pre-ART Oligonucleotide Assay (OLA)
- Where
- 2 sites in Kenya
- Volunteers
- 991 people
- Sponsor
- University of Washington
Leukocyte Adhesion Deficiency Syndrome
Interferon Gamma to Treat Leukocyte Adhesion Deficiency Type I
This study will evaluate the safety and effectiveness of the drug, interferon gamma, in treating leukocyte adhesion deficiency type I (LAD I).
- Trial name
- NCT00001905
- Testing
- Interferon gamma
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Vitamin D Deficiency
Vitamin D Deficiency Still Growing
Vitamin D deficiency has become one of the most prevalent health problems in modern society. However, there has been no study that has reported the trend of vitamin D status in Asia.
- Trial name
- NCT03304795
- Volunteers
- 39,759 people, healthy volunteers welcome
- Sponsor
- DongGuk University
Iron Deficiency Anemia
Short-term Intravenous Iron Isomaltose Anhydride for IDA
This prospective, randomized, controlled study aims to evaluate the impact of short-term intravenous iron isomaltose anhydride on postoperative recovery and the requirement for allogeneic red blood cells (RBC) transfusion in…
- Trial name
- NCT03915327
- Testing
- Intravenous iron isomaltose anhydride
- Volunteers
- 1,600 people
- Sponsor
- West China Hospital
Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children
GB06 Phase III Trial for Pediatric Growth Hormone Deficiency
This study aims to evaluate the efficacy and safety of GB06 (a biosimilar of Norditropin®FlexProTM from Novo Nordisk) for the treatment of growth disorders caused by growth hormone deficiency (GHD) in children.
- Conditions
- Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children, Growth Hormone Deficiency, Pediatric
- Trial name
- NCT07553351
- Testing
- GB06, Norditropin®FlexProTM
- Where
- China
- Volunteers
- 192 people
- Sponsor
- Kexing Biopharm Co., Ltd.
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