Clinical trials: Deficiency Disease
HIV Infection, HIV Infections
Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients
This research study is being carried out to study a new way to possibly treat HIV. This agent is called a "Zinc Finger Nuclease" or ZFN for short. ZFNs are proteins that can delete another protein named CCR5.
- Trial name
- NCT01044654
- Testing
- SB-728-T
- Where
- 9 sites in United States
- Volunteers
- 19 people
- Sponsor
- Sangamo Therapeutics
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
HIV Infections
Phase 3 Safety and Effectiveness Trial of Dapivirine Vaginal Ring for Prevention of HIV-1 in Women
This is a double-blind, randomised, placebo-controlled study to assess the safety and efficacy of a silicone elastomer vaginal matrix ring.
- Trial name
- ASPIRE (NCT01617096)
- Testing
- Dapivirine Vaginal Ring
- Where
- 7 sites in Malawi, South Africa and 2 more
- Volunteers
- 2,629 people, healthy volunteers welcome
- Sponsor
- International Partnership for Microbicides, Inc.
Human Immunodeficiency Virus
Prospective Observational Epidemiologic Study of Maraviroc's Safety
The study will assess if use of maraviroc along with an optimized background regimen of antiretroviral drugs in usual clinical practice is as safe as using only an optimized regimen of antiretroviral drugs.
- Trial name
- POEM (NCT00665561)
- Testing
- Maraviroc along with an optimized background antiretroviral drug regimen, Optimized background antiretroviral drug regimen without maraviroc
- Where
- 269 sites in United States, Belgium and 9 more
- Volunteers
- 2,500 people
- Sponsor
- ViiV Healthcare
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also…
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Trial name
- NCT06805695
- Testing
- No Intervention
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 13 people
- Sponsor
- iECURE, Inc.
Chronic Kidney Disease stage3, Chronic Kidney Disease stage4
Phase 3 Safety and Efficacy Study of CTAP101 Extended-release Capsules in Children With Secondary Hyperparathyroidism
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled study in children with stage 3-4 chronic kidney disease (CKD), secondary hyperparathyroidism (SHPT) and vitamin D insufficiency.
- Conditions
- Chronic Kidney Disease stage3, Chronic Kidney Disease stage4, Vitamin d Deficiency, Secondary Hyperparathyroidism
- Trial name
- NCT05543928
- Testing
- CTAP101
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- OPKO Health, Inc.
HIV/AIDS, Dried Blood Spot
Monitoring Of Viral Load In Decentralised Area in Vietnam
As of today, HIV-infected patients followed in decentralized area have little or even no access to viral load monitoring because laboratories able to perform this biological measurement are only in large cities, and because…
- Conditions
- HIV/AIDS, Dried Blood Spot, Viral Load
- Trial name
- MOVIDA-2 (NCT03249493)
- Testing
- Blood sample on DBS
- Where
- Vietnam
- Volunteers
- 584 people
- Sponsor
- Institut Pasteur
Thrombocytosis, Iron-Deficiency Anemia
Ferinject® in Patient With Thrombocytosis Secondary to Inflammatory Bowel Disease (IBD)
The aim of this study is to show the benefits for patients, with a high platelet count, iron deficiency and IBD, receiving intravenous iron therapy.
- Trial name
- NCT00882414
- Testing
- FERINJECT® (Ferric carboxymaltose)
- Where
- Austria
- Volunteers
- 26 people
- Sponsor
- Vifor Pharma
HIV Infections
Phase I Study of Alferon N Injection in Persons With Asymptomatic Human Immunodeficiency Virus (HIV) Infection
To determine the safety and tolerance of subcutaneous injections of natural interferon alpha (IFN) in asymptomatic HIV-positive persons and record its effects on the HIV virus in these individuals.
- Trial name
- NCT00002078
- Testing
- Interferon alfa-n3
- Where
- 2 sites in United States
- Sponsor
- Purdue Frederick
Alpha-1 Antitrypsin Deficiency
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose…
- Trial name
- NCT06405633
- Testing
- WVE-006
- Where
- 9 sites in Australia, Canada and 4 more
- Volunteers
- 48 people
- Sponsor
- Wave Life Sciences USA, Inc.
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
HIV Infections
Clinical Performance of Reveal G4 Rapid HIV-1 Antibody Test
To determine the efficacy of an improved rapid diagnostic test using venous whole blood and fingerstick whole blood. The clinical performance of Reveal G4 Rapid HIV-1 Antibody Test will be determined by comparing the results with…
- Trial name
- NCT02190578
- Testing
- Reveal G4
- Where
- 4 sites in United States
- Volunteers
- 1,649 people, healthy volunteers welcome
- Sponsor
- MedMira Laboratories Inc.
Hereditary Angioedema, HAE
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.
- Trial name
- HAErmony-1 (NCT05121376)
- Testing
- Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 44 people
- Sponsor
- BioMarin Pharmaceutical
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acquired Immunodeficiency Syndrome, Healthy Volunteers
Effects of Crofelemer on the Gut Microbiome in Healthy Volunteers and in HIV+ Patients With Non-Infectious Diarrhea
This study is intended to evaluate: 1. Any changes in the gut microbiome from baseline compared to end of study in both healthy (HIV-negative) subjects and HIV+ patients with or without chronic diarrhea, following one month of…
- Conditions
- Acquired Immunodeficiency Syndrome, Healthy Volunteers, HIV/AIDS, HIV Diarrhea
- Trial name
- NCT04192487
- Testing
- Crofelemer delayed-release tablets 125mg
- Where
- 3 sites in United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Napo Pharmaceuticals, Inc.
Malnutrition, Cancer
Impact of a New Plant-based High-energy Oral Nutritional Supplement on Nutritional Outcomes in Malnourished Patients
This study aims to compare nutritional outcomes in terms of percentage of weight gain between a new planted-based high-energy ONS and a standard high-energy ONS with animal protein (cow's mil protein) in patients at malnutrition…
- Conditions
- Malnutrition, Cancer, Inflammatory Bowel Diseases, Chronic Obstructive Pulmonary Disease (COPD)
- Trial name
- NCT06055543
- Testing
- Fortimel
- Where
- 6 sites in Spain
- Volunteers
- 140 people
- Sponsor
- Danone Specialized Nutrition
HIV Infections
The Safety and Effectiveness of 524W91
To assess the safety and pharmacokinetics of single oral doses of 524W91 administered in HIV-infected patients. To determine the effects of food on bioavailability of 524W91.
- Trial name
- NCT00002335
- Testing
- Emtricitabine
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Glaxo Wellcome
HIV Infections
A Study to Evaluate the Safety and Effectiveness of a New Non-Nucleoside Reverse Transcriptase Inhibitor (NNRTI), (+)-Calanolide A, in HIV-Positive Patients Who Have Never Received Anti-HIV Treatment
The purpose of this study is to test the safety and effectiveness of a new non-nucleoside reverse transcriptase inhibitor (NNRTI), (+)-calanolide A, in HIV-positive patients who have never received anti-HIV treatment.
- Trial name
- NCT00002243
- Testing
- Calanolide A
- Where
- 9 sites in United States
- Volunteers
- 32 people
- Sponsor
- Sarawak MediChem Pharmaceuticals
Hereditary Angioedema
A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)
This study is being conducted to evaluate the efficacy and safety of icatibant compared to placebo in patients experiencing acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT00912093
- Testing
- Icatibant
- Where
- 64 sites in United States, Australia and 7 more
- Volunteers
- 98 people
- Sponsor
- Shire
HIV Infections
Safety Study of Tipranavir Co-administered With Low-dose Ritonavir (TPV/r) in Patients With Advanced HIV-1 Infection and Limited Treatment Options
This study evaluated the safety and tolerability of tipranavir to improve treatment options for HIV type 1-infected patients who have been previously treated and whose treatment is no longer effective.
- Trial name
- NCT00144287
- Testing
- Tipranavir/Ritonavir
- Where
- 53 sites in Germany
- Volunteers
- 255 people
- Sponsor
- Boehringer Ingelheim
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