Clinical trials: Deficiency Disease
Iron Deficiency Anemia Secondary to IBD or Gastric Bypass
Treatment Response of Injectafer vs Oral Iron to Baseline Hepcidin Levels in Patients With Iron Deficiency Anemia (IDA) Secondary to Inflammatory Bowel Disease (IBD) or Gastric Bypass
The primary objective of this study is to evaluate the treatment response of Injectafer vs. oral iron to baseline hepcidin levels to determine if any of these select IBD or Gastric Bypass patients may demonstrate to be…
- Trial name
- NCT02086968
- Testing
- Injectafer, Ferrous Sulfate tablets
- Volunteers
- 198 people
- Sponsor
- American Regent, Inc.
Lung Disease, Pulmonary Disease
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
- Conditions
- Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency
- Trial name
- AATD (NCT06622668)
- Testing
- Biological NTLA-3001
- Where
- New Zealand
- Sponsor
- Intellia Therapeutics
Diabetic Kidney Disease
Efficacy and Safety of QiShen YiQi Dripping Pills in the Treatment of Diabetic Kidney Disease (Syndrome of Qi Deficiency With Blood Stasis)
This study will evaluate the efficacy and Safety of QiShen YiQi Dripping Pills in the treatment of Diabetic Kidney Disease (syndrome of Qi deficiency with blood stasis)
- Trial name
- NCT06006689
- Testing
- Low dose Qishen Yiqi Dripping Pills 3 bags, High dose Qishen Yiqi Dripping Pills 3 bags
- Where
- 16 sites in China
- Volunteers
- 228 people
- Sponsor
- Tasly Pharmaceutical Group Co., Ltd
Acquired Immunodeficiency Syndrome, HIV Infections
D/C/F/TAF Versus COBI-boosted DRV Plus FTC/TDF in HIV-1 Infected, Antiretroviral Treatment Naive Adults
This study is to evaluate the safety and efficacy darunavir/cobicistat/emtricitabine/tenofovir alafenamide (D/C/F/TAF) fixed dose combination (FDC) tablet versus darunavir (DRV)+cobicistat (COBI)+emtricitabine (FTC)/tenofovir…
- Trial name
- NCT01565850
- Testing
- D/C/F/TAF, DRV, COBI
- Where
- 44 sites in United States, Puerto Rico
- Volunteers
- 153 people
- Sponsor
- Gilead Sciences
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Investigating Efficacy and Safety of Once-weekly NNC0195-0092 Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment…
- Trial name
- NCT02616562
- Testing
- somapacitan, Norditropin® FlexPro® pen
- Where
- 89 sites in United States, Austria and 12 more
- Volunteers
- 76 people
- Sponsor
- Novo Nordisk A/S
Infection, Human Immunodeficiency Virus
Safety and Tolerability Study to Evaluate Lower Dose of GSK2248761 in Antiretroviral Treatment-Naive HIV-1 Infected Adults.
GSK has in-licensed a novel NNRTI-class candidate (GSK2248761, IDX12899) for the treatment of subjects with HIV-1 infection from Idenix Pharmaceuticals.
- Trial name
- NCT00945282
- Testing
- GSK2248761, Lopinavir/ritonavir, HAART
- Where
- Argentina
- Volunteers
- 8 people
- Sponsor
- ViiV Healthcare
HIV Infections
A Safety Study of Dapivirine (TMC120) Vaginal Microbicide Gel in South Africa
Approximately 42 HIV-negative women, aged >18 and < 50, will be enrolled in this study. Each volunteer will have a 2:1 chance of receiving Dapivirine Gel-002 versus placebo.
- Trial name
- NCT00304668
- Testing
- dapivirine (TMC120) vaginal gel
- Volunteers
- healthy volunteers welcome
- Sponsor
- International Partnership for Microbicides, Inc.
Infection, Human Immunodeficiency Virus I
A Trial Comparing GSK1349572 50mg Once Daily to Raltegravir 400mg Twice Daily
The purpose of this trial is to assess the non-inferior antiviral activity of GSK1349572 50 mg once daily versus RAL 400mg twice daily over 48 weeks; non-inferiority will also be tested at Week 96.
- Trial name
- SPRING-2 (NCT01227824)
- Testing
- GSK1349572 (dolutegravir), raltegravir, ABC/3TC
- Where
- 101 sites in United States, Australia and 7 more
- Volunteers
- 828 people
- Sponsor
- ViiV Healthcare
Lung Cancer
Exploration of Predictive Markers of Neoadjuvant Immunotherapy in Non-Small Cell Lung Cancer
Neoadjuvant immunotherapy has become the standard perioperative treatment in lung cancer, but its effective predictive biomarkers are lacking.
- Trial name
- NCT05965557
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Geneplus-Beijing Co. Ltd.
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset)
Pompe Pregnancy Sub-Registry
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is…
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00567073
- Where
- 35 sites in United States, Australia and 7 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Alpha-1 Antitrypsin Deficiency
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels [6.0 ×…
- Trial name
- NCT01054339
- Testing
- rAAV1-CB-hAAT
- Where
- 4 sites in United States, Ireland
- Volunteers
- 9 people
- Sponsor
- Beacon Therapeutics
Mucopolysaccharidosis II
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
- Trial name
- NCT00069641
- Testing
- Iduronate-2-sulfatase enzyme replacement therapy
- Where
- 9 sites in United States, Brazil and 2 more
- Volunteers
- 96 people
- Sponsor
- Shire
Infection, Human Immunodeficiency Virus
Single and Repeated Dose Escalation Study of GSK2838232
GSK2838232 is a Human Immunodeficiency Virus (HIV) maturation inhibitor being developed for the treatment of HIV in combination with other antiretroviral therapy (ART).
- Trial name
- NCT02795754
- Testing
- GSK2838232 PIB, GSK2838232 IR1, GSK2838232 IR2
- Where
- United States
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Pompe Disease, Glycogen Storage Disease Type II
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Pompe disease is caused by a deficiency of a critical enzyme in the body called acid alpha glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures…
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II, Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- NCT00025896
- Testing
- recombinant human acid alpha-glucosidase (rhGAA)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Genzyme, a Sanofi Company
Adult Growth Hormone Deficiency
A Study to Follow Patients With Adult Growth Hormone Deficiency (AGHD) Treated With Sogroya® for Long Term Safety Information
In this study, the general long-term safety and effectiveness of Sogroya (somapacitan) in adults with growth hormone deficiency (AGHD) being treated per normal clinical practice is looked into.
- Trial name
- NCT05718570
- Testing
- Somapacitan
- Where
- 55 sites in United States, France and 4 more
- Volunteers
- 400 people
- Sponsor
- Novo Nordisk A/S
Colorectal Cancer
Evaluation of Co-formulated Pembrolizumab/Quavonlimab (MK-1308A) Versus Other Treatments in Participants With Microsatellite Instability-High (MSI-H) or Mismatch Repair Deficient (dMMR) Stage IV Colorectal Cancer (CRC) (MK-1308A-008/KEYSTEP-008).
The purpose of this study is to assess the efficacy and safety of co-formulated pembrolizumab/quavonlimab versus other treatments in participants with MSI-H or dMMR Metastatic Stage IV Colorectal Cancer.
- Trial name
- NCT04895722
- Testing
- Pembrolizumab, Pembrolizumab/Quavonlimab, Pembrolizumab/Favezelimab
- Where
- 109 sites in United States, Belgium and 20 more
- Volunteers
- 302 people
- Sponsor
- Merck Sharp & Dohme LLC
Human Immunodeficiency Virus (HIV) Infection
Pharmacokinetics of Islatravir in Participants With Severe Renal Impairment (MK-8591-026)
This study will evaluate the general tolerability and pharmacokinetics (PK) of a single 60 mg dose of MK-8591 (Islatravir) in participants with severe renal insufficiency, compared to participants in good health.
- Trial name
- NCT04303156
- Testing
- Islatravir
- Where
- 2 sites in United States, Germany
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Factor XIII Deficiency
A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency
Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding.
- Trial name
- NCT00883090
- Testing
- FXIII Concentrate (Human)
- Where
- 6 sites in United States, Spain
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Hereditary Angioedema
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks
This phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)
- Trial name
- NCT04739059
- Testing
- CSL312
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 171 people
- Sponsor
- CSL Behring
HIV Infections, Peripheral Nervous System Diseases
Study of NGX-4010 for Treatment of Painful HIV-Associated Neuropathy
The purpose of the study is to determine if an investigational drug, NGX-4010 (high-concentration capsaicin patch), is safe, tolerable and effective in treating painful HIV-associated neuropathy.
- Conditions
- HIV Infections, Peripheral Nervous System Diseases, Pain
- Trial name
- NCT00085761
- Testing
- Capsaicin Dermal Patch
- Where
- 2 sites in United States
- Sponsor
- NeurogesX
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