Clinical trials: Deficiency Disease
Advanced Solid Tumors Cancer, MSI-H Cancer
A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.
The goal of this clinical trial is to learn if NDI-219216 is safe for patients, and if NDI-219216 might be a possible treatment for advanced solid tumors in the later phases of the study.
- Trial name
- NCT06898450
- Testing
- NDI-219216
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 134 people
- Sponsor
- Nimbus Wadjet, Inc.
Growth Hormone Deficiency
A Multi-Dose Study to Assess Tolerability, Safety and Pharmacology of hGH-ViaDerm™ System in Adults With GH-Deficiency
This study aims to examine the safety, tolerability and pharmacokinetics of transdermal delivery of human Growth Hormone (hGH or somatropin) using the ViaDerm device in adult patients with Growth Hormone Deficiency Syndrome.
- Trial name
- NCT00455260
- Testing
- hGH-ViaDerm™ System (hGH or somatropin), hGH-ViaDerm™ System
- Volunteers
- 60 people
- Sponsor
- Teva Neuroscience, Inc.
Hereditary Angioedema
A Study of STAR-0215 in Healthy Adult Participants
This is a first-in-human, single-center, randomized, double-blind, placebo-controlled, single ascending dose study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of a single…
- Trial name
- NCT05477160
- Testing
- STAR-0215 (SC), STAR-0215 (IV)
- Where
- United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Astria Therapeutics, Inc.
HIV Infections
Effects of Tipranavir/Ritonavir on the Pharmacokinetic Characteristics of Triple Drug Nucleoside and Non-nucleoside Reverse Transcriptase Inhibitor Therapy in HIV-1-infected Subjects
Primary: Sequentially determine the effects of three dose combinations of tipranavir (TPV) / ritonavir (RTV) (administered b.i.d.), TPV 1250 mg/RTV 100 mg vs. TPV 750 mg/RTV 100 mg vs.
- Trial name
- NCT02251223
- Testing
- Tipranavir low dose, Tipranavir medium dose, Tipranavir high dose
- Volunteers
- 208 people
- Sponsor
- Boehringer Ingelheim
HIV Infections
Efficacy and Safety Study of Miconazole Lauriad to Treat Oropharyngeal Candidiasis in HIV Patients
The purpose of this study is to evaluate the clinical cure of miconazole Lauriad 50 mg (1x50mg) Bioadhesive buccal tablets compared with clotrimazole troches (5x10mg) after 14 days of treatment (at the test of cure visit, at Day…
- Trial name
- NCT00390780
- Testing
- miconazole Lauriad, Clotrimazole
- Where
- 27 sites in United States, Canada
- Volunteers
- 578 people
- Sponsor
- Valerio Therapeutics
Mitochondrial Myopathies, Mitochondrial Pathology
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases
- Trial name
- NuPower (NCT05162768)
- Testing
- Elamipretide
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 102 people
- Sponsor
- Stealth BioTherapeutics Inc.
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
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Safety and Pharmacokinetics of Recombinant Factor XIII in Healthy Volunteers
This trial was conducted in Europe. The aim of this trial was to investigate safety and pharmacokinetics of escalating single doses of catridecacog (recombinant factor XIII, rFXIII) in healthy volunteers.
- Conditions
- Congenital Bleeding Disorder, Congenital FXIII Deficiency, Healthy
- Trial name
- NCT01847989
- Testing
- catridecacog
- Where
- United Kingdom
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
HIV Infections
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a New Formulation of Cabotegravir Long-Acting Administered Intramuscularly in a 4-month Dosing Interval (Q4M)
This study will evaluate the pharmacokinetics (PK), safety, and tolerability of a new formulation of Cabotegravir (CAB) dosed every 4-months (Q4M) for pre-exposure prophylaxis (PrEP) in participants at risk of HIV-1 acquisition.
- Trial name
- NCT06741397
- Testing
- CAB LA, New formulation of CAB LA
- Where
- 27 sites in United States, Puerto Rico
- Volunteers
- 229 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Infection, Human Immunodeficiency Virus
Single Dose Escalation Study of GSK2838232 in Healthy Subjects
GSK2838232 is a novel human immune virus (HIV) maturation inhibitor being developed for the treatment of chronic HIV infection. This study is the first administration of GSK2838232 in humans to establish the initial safety…
- Trial name
- NCT01802918
- Testing
- GSK2838232, Ritonavir
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
HSIL, High-Grade Squamous Intraepithelial Lesions, Human Papilloma Virus Infection
Study of Topical ABI-1968 in Subjects With Precancerous Anal Lesions Resulting From Human Papillomavirus (HPV) Infection
This study evaluates the use of ABI-1968, a topical cream, in the treatment of anal precancerous lesions in adults with and without human immunodeficiency virus (HIV) infection
- Conditions
- HSIL, High-Grade Squamous Intraepithelial Lesions, Human Papilloma Virus Infection, HIV Infection, Anal Cancer
- Trial name
- NCT03677960
- Testing
- Topical ABI-1968 cream
- Where
- 2 sites in United States, Australia
- Volunteers
- 6 people
- Sponsor
- Antiva Biosciences
Infection, Human Immunodeficiency Virus, HIV Infections
Study Of An Investigational Regimen Including FDA Approved HIV Drugs In HIV-Infected Pediatric Subjects
This is a 48-week study to evaluate the safety, tolerability, pharmacokinetics, and antiviral activity of an investigational regimen including FDA approved HIV drugs in HIV-infected pediatric subjects, ages 4 weeks to < 2 years…
- Trial name
- NCT00071760
- Testing
- GW433908, ritonavir
- Where
- 15 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- ViiV Healthcare
Primary Immunodeficiency Diseases (PID)
A Study of TAK-664 in People With Primary Immunodeficiency Diseases Who Have Not Yet Been Treated With Immunoglobulins
Primary immunodeficiency disease (or PIDD) is a group of conditions in which the immune system does not work properly. Some people with PIDD do not make enough antibodies.
- Trial name
- NCT07794735
- Testing
- TAK-664
- Volunteers
- 9 people
- Sponsor
- Takeda
HIV Infections
Investigation of the Potential Pharmacokinetic Interaction Between Nevirapine (VIRAMUNE) and Ethinyl Estradiol/Norethindrone in HIV-1 Infected Women
Study to determine the effects of nevirapine treatment on the pharmacokinetics of ethinyl estradiol (EE)/norethindrone (NET).
- Trial name
- NCT02182791
- Testing
- Nevirapine, EE/NET
- Volunteers
- 14 people
- Sponsor
- Boehringer Ingelheim
HIV Infections
Regimen Switch to Dolutegravir/Lamivudine Fixed Dose Combination From Current Antiretroviral Regimen in HIV-1 Infected and Virologically Suppressed Adults (SALSA)
The aim of this study is to determine if virologically suppressed Human Immunodeficiency Virus (HIV) Type 1 infected adults on a current antiretroviral regimen (CAR) (including 2 nucleoside reverse transcriptase inhibitors…
- Trial name
- NCT04021290
- Testing
- DTG/3TC FDC, CAR
- Where
- 119 sites in United States, Argentina and 15 more
- Volunteers
- 493 people
- Sponsor
- ViiV Healthcare
Cancer, Hematologic Diseases
A Study of Safety, Tolerability, and Pk of rhMBL in Pediatric Hematology/Oncology Pts With Fever and Neutropenia
The goal of this clinical research study is to find the dose of EZN-2232 that can be given to MBL deficient pediatric cancer patients undergoing chemotherapy.
- Conditions
- Cancer, Hematologic Diseases, Fever, Neutropenia
- Trial name
- NCT00520325
- Testing
- Intravenous recombinant human mannose binding lectin
- Sponsor
- Enzon Pharmaceuticals, Inc.
Hepatitis C Virus Infection, Human Immunodeficiency Virus Infection
A Multipart, Open-label Study to Evaluate the Safety and Efficacy of ABT-450/r/ABT-267 With and Without ABT-333 Coadministered With and Without Ribavirin in Adult With Genotype 1 or 4 Hepatitis C Virus (HCV) Infection and Human Immunodeficiency Virus, Type 1 Coinfection
The primary objectives of this study are to assess the safety of ABT-450/r/ABT-267 with and without ABT-333 coadministered with and without ribavirin (RBV) for 12 and 24 weeks in HCV GT1- or 4-infected participants with HIV-1…
- Conditions
- Hepatitis C Virus Infection, Human Immunodeficiency Virus Infection, Chronic Hepatitis C, Compensated Cirrhosis and Non-cirrhotics
- Trial name
- TURQUOISE-I (NCT01939197)
- Testing
- ABT-450/r/ABT-267, ABT-333, ribavirin
- Volunteers
- 318 people
- Sponsor
- AbbVie
Human Immunodeficiency Virus (HIV)
Human Immunodeficiency Virus (HIV) Postexposure Prophylaxis (PEP) With Darunavir/Ritonavir (DRV/r)
The primary purpose of this study is to assess the rate of early discontinuation from randomized Human Immunodeficiency Virus (HIV) Postexposure Prophylaxis (PEP) for any reason other than confirmation of the negative HIV…
- Trial name
- PEPDar (NCT01516970)
- Testing
- Darunavir/Ritonavir (DRV/r), Lopinavir in fixed combination with Ritonavir, Zidovudine
- Where
- 17 sites in Germany
- Volunteers
- 312 people
- Sponsor
- Janssen-Cilag G.m.b.H
Heart Failure
Efficacy and Safety of Jia Shen Tablets in Chronic Heart Failure
Evaluate the efficacy and safety of Jia Shen Tablets in patients with Chronic Heart Failure.
- Trial name
- NCT06669169
- Testing
- Low dose Jia Shen Tablet, High dose Jia Shen Tablet
- Where
- China
- Volunteers
- 288 people
- Sponsor
- Tasly Pharmaceutical Group Co., Ltd
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