Clinical trials: Deficiency Disease
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00483379
- Testing
- alglucosidase alfa
- Where
- 11 sites in United States, Australia and 1 more
- Volunteers
- 13 people
- Sponsor
- Genzyme, a Sanofi Company
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI
This study is designed to provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were…
- Trial name
- NCT02859727
- Testing
- CDZ173
- Where
- 8 sites in United States, Belarus and 5 more
- Volunteers
- 37 people
- Sponsor
- Pharming Technologies B.V.
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
HIV Infections, Smallpox
Safety Study of MVA Smallpox Vaccine in HIV-positive Subjects Who Are Vaccinia Naive
The purpose of this study is to assess the safety and immunogenicity of two MVA smallpox vaccine injections in healthy adults that are 18-35 years of age with HIV infection
- Trial name
- NCT00282581
- Testing
- MVA Smallpox Vaccine
- Where
- 6 sites in United States
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
HIV Infections
Single Tablet Regimen (STR) Simplification Study for HIV-1 Infected Patients
To compare the effectiveness (efficacy, safety \& tolerability) of a Single Tablet Regimen of efavirenz/emtricitabine/tenofovir DF to subjects continuing on unmodified HAART as measured by the proportion of patients who maintain…
- Trial name
- NCT00365612
- Testing
- Efavirenz (EFV), Emtricitabine (FTC), Tenofovir DF (TDF)
- Where
- 56 sites in United States, Puerto Rico
- Volunteers
- 300 people
- Sponsor
- Gilead Sciences
HIV Infections
Italian Prospective Data Collection for Better Patient Characterization in a Highly Treatment Experienced Population
Data collection for better patient characterization in a highly treatment experienced population. Studying the clinical practice for the treatment of HIV multiexperienced patients in the real life.
- Trial name
- NCT00614302
- Testing
- TPV
- Where
- 21 sites in Italy
- Volunteers
- 428 people
- Sponsor
- Boehringer Ingelheim
HIV Infections
Study Using CABENUVA™ for the Treatment of Human Immunodeficiency Virus (HIV)-1, Administered in Infusion Centers (IC) or Alternate Sites of Administration (ASA) in the United States (U.S.)
GLACIER (Giving Long Acting CABENUVA in an Infusion center/ASA) is an interventional study examining the administration of CABENUVA (Cabotegravir long acting [LA] plus Rilpivirine LA) intramuscular (IM) in infusion centers/ASAs…
- Trial name
- GLACIER (NCT04982445)
- Testing
- CABENUVA
- Where
- 15 sites in United States
- Volunteers
- 44 people
- Sponsor
- ViiV Healthcare
HIV Infections
Phase I Study of the Safety, Tolerance, and Pharmacokinetics of 9-[2-(Bispivaloyloxymethyl)Phosphonylmethoxyethyl]Adenine (Bis-POM PMEA; Adefovir Dipivoxil) in HIV-Infected Patients
To study the safety, tolerance, and pharmacokinetics of a single dose of bis-POM PMEA (adefovir dipivoxil) when administered by the oral route in patients with HIV infection.
- Trial name
- NCT00002128
- Testing
- Adefovir dipivoxil
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Gilead Sciences
HIV Infections
A Phase 4 Cross-Sectional Study of Bone Mineral Density in HIV-1 Infected Subjects
A Phase 4 study is to characterize the profile of low bone mineral density (BMD) in ≥ 50 year old male subjects and post-menopausal female subjects on any tenofovir disoproxil fumarate (TDF)-based regimen
- Trial name
- NCT01850212
- Where
- 73 sites in Austria, Belgium and 11 more
- Volunteers
- 476 people
- Sponsor
- Gilead Sciences
HIV Infections
A Study to Investigate the Antiviral Effect, Safety, Tolerability and Pharmacokinetics of VH3739937 in in Treatment-Naive Adults Living With HIV-1
The primary purpose of this study is to assess the antiviral activity of VH3739937 in Human Immunodeficiency Virus Type-1 (HIV-1) infected treatment naive (TN) participants during monotherapy.
- Trial name
- NCT06061081
- Testing
- VH3739937
- Where
- 18 sites in United States, Argentina and 4 more
- Volunteers
- 21 people
- Sponsor
- ViiV Healthcare
Growth Hormone Disorder, Growth Hormone Deficiency in Children
An Observational Study on Treatment Compliance by Children Treated With Growth Hormone
This study is conducted in Europe. This observational study aims at evaluating compliance with growth hormone treatment in children and identifying factors influencing compliance.
- Trial name
- IMPACT (NCT01068639)
- Testing
- No treatment given
- Sponsor
- Novo Nordisk A/S
NSCLC (Advanced Non-small Cell Lung Cancer), NSCLC (Non-small Cell Lung Cancer)
A Multicenter Randomized Open-Label Trial Evaluating ctDNA-Guided Interruption Versus Standard of Care Immune-Checkpoint Inhibitor (ICI) Therapy In Patients With Advanced / Metastatic Solid Tumors.
This is a multicenter, open-label, randomized (1:1) trial designed to evaluate whether ctDNA-guided interruption of immune-checkpoint inhibitor (ICI) therapy provides comparable survival to standard of care (SoC) continuous ICI…
- Conditions
- NSCLC (Advanced Non-small Cell Lung Cancer), NSCLC (Non-small Cell Lung Cancer), NSCLC (Non-small Cell Lung Carcinoma), NSCLC
- Trial name
- SIGNAL-IO 301 (NCT07689812)
- Testing
- Signatera Genome ultra-sensitive ctDNA blood test
- Volunteers
- 920 people
- Sponsor
- Natera, Inc.
HIV Infections
A Phase I/II Clinical Study of WF 10 IV Solution ( TCDO ) in Patients With HIV Infection
To evaluate the clinical toxicity, safety, and MTD of WF 10 ( TCDO ) intravenous solution administered to patients with HIV infection. To evaluate the potential anti-HIV activity of TCDO.
- Trial name
- NCT00002119
- Testing
- WF10
- Where
- United States
- Sponsor
- Oxo Chemie GmbH
Hyperlipoproteinemia Type I
Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies
LPL (Lipoprotein Lipase) is an enzyme which plays an important role in the elimination of triglycerides (fat) and the clearance of dietary fat particles known as chylomicrons (CM) in the blood.
- Trial name
- NCT01447901
- Where
- Canada
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Amsterdam Molecular Therapeutics
Cancer, Metastatic, Cancer
SYNERGY-AI: Artificial Intelligence Based Precision Oncology Clinical Trial Matching and Registry
International registry for cancer patients evaluating the feasibility and clinical utility of an Artificial Intelligence-based precision oncology clinical trial matching tool, powered by a virtual tumor boards (VTB) program, and…
- Conditions
- Cancer, Metastatic, Cancer, Cancer of Pancreas, Cancer of Liver
- Trial name
- NCT03452774
- Testing
- Clinical Trial Matching
- Where
- 68 sites in United States, Puerto Rico
- Volunteers
- 50,000 people
- Sponsor
- Massive Bio, Inc.
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
HIV Infections, Psoriasis
Treatment of Psoriasis Using Acitretin in HIV-Positive Patients
To determine the efficacy of acitretin in the treatment of psoriasis in HIV/AIDS patients. Etretinate, a retinoid, has proven successful in the treatment of HIV-infected patients with psoriasis, but it has an elimination…
- Trial name
- NCT00002143
- Testing
- Acitretin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
Advanced Solid Tumor, Ovarian Cancer
NUV-868 as Monotherapy and in Combination With Olaparib or Enzalutamide in Adult Patients With Advanced Solid Tumors
NUV-868-01 is a first-in human, open- label, Phase 1/2 dose escalation and expansion study in patients with advanced solid tumors.
- Conditions
- Advanced Solid Tumor, Ovarian Cancer, Ovary Cancer, Cancer of Ovary
- Trial name
- NCT05252390
- Testing
- NUV-868, Olaparib, Enzalutamide
- Where
- 22 sites in United States, Australia
- Volunteers
- 82 people
- Sponsor
- Nuvation Bio Inc.
HTLV-I Infections, HTLV-II Infections
Dual Algorithm Post Market Clinical Study
The purpose of this study is to assess the validity and reproducibility of the MP Diagnostics HTLV Blot 2.4 in blood specimens testing repeat reactive (RR) on the first FDA licensed screening assay (Abbott Prism) and non-reactive…
- Conditions
- HTLV-I Infections, HTLV-II Infections, Human T-lymphotropic Virus 1, Human T-lymphotropic Virus 2
- Trial name
- NCT03146013
- Testing
- MP Diagnostics HTLV Blot 2.4
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- MP Biomedicals, LLC
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