Clinical trials: Deficiency Disease
Cockayne Syndrome
Pharmacokinetics and Safety Study of Single and Multiple Oral Doses Prodarsan™ in Patients With Cockayne Syndrome
This study is to compare the exposure of orally administered Prodarsan to the intravenous dosed Osmitrol (10% solution) in Cockayne Syndrome (CS) patients.
- Trial name
- NCT01142154
- Testing
- Prodarsan
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- DNage B.V.
Limb Girdle Muscular Dystrophy
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Advanced Cancer, Advanced Solid Tumor
A Study of Oral 7HP349 (Alintegimod) in Combination With Ipilimumab Followed by Nivolumab Monotherapy
This study is an open-label Phase Ib (Part A) dose escalation followed by a blinded, randomized, multi cohort Phase 2a (Part B) comparison of combination vs. reference regimens.
- Conditions
- Advanced Cancer, Advanced Solid Tumor, Melanoma, Metastasis
- Trial name
- NCT06362369
- Testing
- Alintegimod, Ipilimumab, Nivolumab
- Where
- 5 sites in United States
- Volunteers
- 126 people
- Sponsor
- 7 Hills Pharma, LLC
HIV Infections, Peripheral Neuropathy
Study of Pregabalin Versus Placebo in the Treatment of Nerve Pain Associated With HIV Neuropathy
Study to determine if pregabalin is more effective than placebo in treating subjects with nerve pain associated with HIV neuropathy.
- Trial name
- NCT00232141
- Testing
- pregabalin
- Where
- 44 sites in United States, Puerto Rico
- Volunteers
- 302 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
IgA Nephropathy, Lupus Nephritis (LN)
A Phase 2 Study to Evaluate the Safety, PD, PK, and Clinical Activity of ADX-097 in Participants With IgAN, LN or C3G
A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Clinical Activity of ADX-097 Administered Subcutaneously in Male and Female Participants Aged 16 Years or Older with Immunoglobulin A Nephropathy…
- Conditions
- IgA Nephropathy, Lupus Nephritis (LN), C3 (Complement Component 3) Glomerulopathy
- Trial name
- NCT06419205
- Testing
- ADX-097
- Where
- 4 sites in United States, Brazil
- Volunteers
- 30 people
- Sponsor
- Akebia Therapeutics
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
Growth Hormone Deficiency
VRS-317 in Adult Subjects With Growth Hormone Deficiency
The purpose of this research study is to determine the safety and tolerability of up to five doses of VRS-317 in Adult Growth Hormone Deficient patients.
- Trial name
- NCT01359488
- Testing
- VRS-317
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Versartis Inc.
Immunologic Deficiency Syndrome
Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency
The purpose of this study is to evaluate the safety, efficacy and pharmacokinetics of Immune Globulin Intravenous (Human) IVIG-SN™ in subjects with primary immunodeficiency diseases.
- Trial name
- NCT01406470
- Testing
- Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™
- Where
- 11 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Green Cross Corporation
Malignant Lymphoma of Lymph Nodes of Inguinal Region, Malignant Lymphoma of Lymph Nodes of Axillary
Optical Detection of Peripheral Nerve Bundles During Surgery
Investigation of application possibilities of optical spectroscopy within the field of surgical resection procedures to spare nerve tissue.
- Conditions
- Malignant Lymphoma of Lymph Nodes of Inguinal Region, Malignant Lymphoma of Lymph Nodes of Axillary, Malignant Lymphoma of Lymph Nodes of the Cervix, Carcinoma of Parotid Gland
- Trial name
- NerveSpect (NCT01731379)
- Testing
- Surgical resection
- Where
- Netherlands
- Volunteers
- 35 people
- Sponsor
- Philips Healthcare
HIV Infections
Safety of EVG+RTV Administered With Other Antiretroviral Agents for the Treatment of HIV-1 Infection
The main objective of this study is to observe the long-term safety of elvitegravir (EVG) boosted with ritonavir (RTV) in combination with other antiretroviral (ARV) agents in participants who have completed a prior EVG+RTV…
- Trial name
- NCT00445146
- Testing
- EVG, RTV, ARV regimen
- Where
- 48 sites in United States, Puerto Rico
- Volunteers
- 192 people
- Sponsor
- Gilead Sciences
Human Immunodeficiency Virus
Quality of Life of HIV-infected Participants Switched to Raltegravir Versus Other Antiretroviral Regimens (MK-0518-266)
This is a multi-center, longitudinal observational study of adult human immunodeficiency virus (HIV) participants at academic and community-based practices in the United States who are switching from first-line to second-line…
- Trial name
- NCT01814722
- Volunteers
- 63 people
- Sponsor
- Merck Sharp & Dohme LLC
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences
HIV Infections
Study to Evaluate Pharmacokinetic (PK), Safety and Tolerability of Cabotegravir (CAB) 400 Milligrams Per Milliliter (mg/mL) Formulation in Healthy Adult Participants
This is an active control, randomized study to investigate the safety, tolerability and PK of repeat dose administration of long-acting CAB 400 mg/mL formulation intramuscular (IM) (gluteus medius and vastus lateralis) and…
- Trial name
- NCT04484337
- Testing
- Cabotegravir sodium (Oral Lead In), Cabotegravir 400 mg/mL, Cabotegravir 200 mg/mL
- Where
- 5 sites in United States, New Zealand
- Volunteers
- 138 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Alpha 1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Trial name
- NCT04167345
- Testing
- VX-814
- Where
- 28 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Human Immunodeficiency Virus
A Safety Study of Brentuximab Vedotin in Participants With HIV
This study will test brentuximab vedotin to see if it is safe for people with human immunodeficiency virus (HIV) who have low CD4+ and have received antiretroviral therapy (ART) treatment.
- Trial name
- NCT05244473
- Testing
- brentuximab vedotin, ART
- Where
- 2 sites in United States
- Sponsor
- Seagen Inc.
Symptomatic Neurogenic Orthostatic Hypotension
Sustained Effect of Droxidopa in Symptomatic Neurogenic Orthostatic Hypotension
To evaluate the time to treatment intervention in patients with Parkinson's Disease (PD), Multiple System Atrophy (MSA), Pure Autonomic Failure (PAF), Non-Diabetic Autonomic Neuropathy (NDAN) or Dopamine Beta Hydroxylase (DBH)…
- Trial name
- RESTORE (NCT02586623)
- Testing
- Droxidopa capsules
- Where
- 168 sites in United States
- Volunteers
- 453 people
- Sponsor
- H. Lundbeck A/S
Growth Hormone Deficiency
Crossover Study to Assess the Safety and Pharmacokinetic of Pegylated Somatropin(PEG Somatropin) in GHD Children
The purpose of the phase 1 study is to assess the safety and pharmacokinetics of PEG somatropin, which administered once per week, compared with the daily used somatropin, and to evaluate the safety and possibility to replace…
- Trial name
- Phase 1 (NCT01613573)
- Testing
- somatropin AQ, pegylated somatropin
- Volunteers
- 12 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Primary Immunodeficiency
Pharmacokinetics and Safety of IVIG Nanogam 100 mg/ml
Intravenous immunoglobulin (IVIG) is used for treatment of a heterogeneous group of immune related disorders both as immune-replacement and immune-modulating therapy.
- Trial name
- Nanogam (NCT01985373)
- Testing
- Intravenous immunoglobulin infusion
- Where
- 4 sites in Netherlands
- Volunteers
- 23 people
- Sponsor
- Prothya Biosolutions
HIV Infection
A Prospective, Observational Study of Pregnancy Outcomes Among Women Exposed to Truvada for PrEP Indication
This is a prospective, observational study to describe pregnancy outcomes among HIV-1 uninfected pregnant women who are taking emtricitabine (FTC)/tenofovir disoproxil fumarate (TDF) (Truvada®) for a pre-exposure prophylaxis…
- Trial name
- NCT01865786
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- Gilead Sciences
HIV Infections, Hepatitis B Infections
Rapid Diagnostics for HIV and Hepatitis
The purpose of this study is to determine the efficacy of two rapid diagnostic tests in plasma, venipuncture whole blood, and fingerstick whole blood.
- Conditions
- HIV Infections, Hepatitis B Infections, Hepatitis C Infections
- Trial name
- NCT02190305
- Testing
- Multiplo HBc/HIV/HCV and Reveal HBsAg
- Where
- 4 sites in United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- MedMira Laboratories Inc.
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