Clinical trials: Deficiency Disease
HIV Infection, HIV Infections
Isentress Re-examination Study (MK-0518-115)
This survey is conducted for preparing application materials for re-examination under the Pharmaceutical Affairs Laws and its Enforcement Regulation, its aim is to reconfirm the clinical usefulness of Isentress through collecting…
- Trial name
- NCT01042808
- Volunteers
- 996 people
- Sponsor
- Merck Sharp & Dohme LLC
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.
Factor VII Deficiency, Glanzmann Thrombasthenia
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Conditions
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- Trial name
- NCT04548791
- Testing
- Coagulation Factor VIIa variant
- Where
- 20 sites in United States, India and 3 more
- Volunteers
- 19 people
- Sponsor
- Catalyst Biosciences
Ornithine Transcarbamylase Deficiency
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g.
- Trial name
- NCT04442347
- Testing
- ARCT-810
- Where
- 8 sites in United States
- Volunteers
- 16 people
- Sponsor
- Arcturus Therapeutics, Inc.
HIV-infection/Aids
Phase III Clinical Study of Azvudine in Hiv-infected Treatment Naive Patients
Azvudine,(FNC)， new nuclear nucleoside reverse transcriptase inhibitors, FNC make itself a better candidate to be co-formulated in other anti-HIV therapies, thus to improve patient's compliance, approved by state drug…
- Trial name
- NCT04303598
- Testing
- FNC, 3TC, TDF
- Where
- 12 sites in China
- Volunteers
- 720 people
- Sponsor
- Henan Genuine Biotech Co., Ltd.
Infection, Human Immunodeficiency Virus, HIV Infections
A Study of GSK1349572 Versus Raltegravir (RAL) With Investigator Selected Background Regimen in Antiretroviral-Experienced, Integrase Inhibitor-Naive Adults
ING111762 is a 48 week, randomized, double-blind, active-controlled, multicenter, parallel group, non-inferiority study. The study will be conducted in at least 688 HIV-1 infected antiretroviral experienced, integrase-naïve…
- Trial name
- SAILING (NCT01231516)
- Testing
- GSK1349572, Raltegravir
- Where
- 180 sites in United States, Argentina and 18 more
- Volunteers
- 724 people
- Sponsor
- ViiV Healthcare
Hereditary Angioedema
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
- Trial name
- KONFIDENT-KID (NCT06467084)
- Testing
- KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
HIV Infections
A Pilot Study of Atazanavir/Ritonavir/Efavirenz as a Nucleoside Sparing Regimen
The purpose of this study is to evaluate the safety and efficacy of a novel nucleoside sparing regimen containing atazanavir, ritonavir and efavirenz, using two different doses of atazanavir.
- Trial name
- NCT00135343
- Testing
- Atazanvir/ritonavir + efavirenz
- Where
- 15 sites in United States
- Volunteers
- 60 people
- Sponsor
- Bristol-Myers Squibb
Mycobacterium Avium-intracellular Infection, HIV Infections
The Safety and Effectiveness of Clarithromycin in the Prevention of Mycobacterium Avium Complex (MAC) Infection in HIV-Infected Patients
To determine whether clarithromycin is safe and effective in preventing disseminated Mycobacterium avium Complex in HIV-infected patients with CD4 counts <= 100 cells/mm3.
- Trial name
- NCT00002336
- Testing
- Clarithromycin
- Where
- 22 sites in United States
- Sponsor
- Abbott
Hereditary Angioedema, HAE
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in…
- Trial name
- APeX-J (NCT03873116)
- Testing
- BCX7353 capsules
- Where
- 10 sites in Japan
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals
Anaemia in Chronic Kidney Disease
A Non-interventional Study of Diafer in Subjects With CKD on Haemodialysis for Treatment of Iron Deficiency
The purpose of the study is to monitor initiated Diafer® therapy administered according to hospital practice and the product labeling in routine clinical practice in haemodialysis patients with Chronic Kidney Disease.
- Trial name
- DINO (NCT02301026)
- Testing
- 5% Iron Isomaltoside 1000
- Where
- 2 sites in Sweden, United Kingdom
- Volunteers
- 209 people
- Sponsor
- Pharmacosmos A/S
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
This is an open-label, single-arm, multicenter Phase 1/2 study evaluating the safety and efficacy of gene therapy by transplantation of Prime Edited autologous CD34+ stem cells modified ex vivo (PM359) in participants with…
- Trial name
- NCT06559176
- Testing
- PM359
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 12 people
- Sponsor
- Prime Medicine, Inc.
HIV Infections
Investigation of V520 in a HIV Vaccine Dose Refinement Study (V520-027)(TERMINATED)
This study will test the safety and immunogenicity of an investigational Human Immunodeficiency Virus (HIV) vaccine. Immunogenicity will be measured by evaluating the immune response to several different dose levels.
- Trial name
- NCT00350623
- Testing
- MRKAd5 HIV-1 gag/pol/nef 4 x 10^9 Ad5 vg/dose (V520), MRKAd5 HIV-1 gag/pol/nef 8 x 10^9 Ad5 vg/dose (V520), MRKAd5 HIV-1 gag/pol/nef 1.5 x 10^10 Ad5 vg/dose (V520)
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Secondary Hyperparathyroidism
Open-label Study to Assess the Long-term Safety and Efficacy of Etelcalcetide (Also Known as AMG 416 or KAI-4169) in Patients With Secondary Hyperparathyroidism
The purpose of this study is to evaluate the long term safety and efficacy of thrice weekly intravenous (IV) administration of etelcalcetide in the treatment of secondary hyperparathyroidism (SHPT) in patients receiving…
- Trial name
- NCT01576146
- Testing
- Etelcalcetide
- Volunteers
- 30 people
- Sponsor
- KAI Pharmaceuticals
HIV Infections
Trial of an Alternative Dosing Regimen of Oral Retrovir in Patients With AIDS or Advanced ARC
To evaluate the safety and tolerance, in patients with severe clinical manifestations of HIV infection, of zidovudine (AZT) administered daily for 48 weeks as a low dose every 4 hours or a higher dose every 12 hours.
- Trial name
- NCT00002020
- Testing
- Zidovudine
- Where
- 12 sites in United States, Puerto Rico
- Sponsor
- Glaxo Wellcome
Human Immunodeficiency Virus - Type 1
A Study to Compare Effectiveness and Safety of Darunavir/Ritonavir (DRV/Rtv) 800mg/100mg Once Daily Versus DRV/Rtv 600mg/100mg Twice Daily in Early Treatment-Experienced HIV-1 Infected Patients (ODIN)
The purpose of this study is to test if being treated with darunavir/ritonavir (DRV/rtv) 800/100 mg daily is as effective as being treated with DRV/rtv 600/100 mg twice daily, in early antiretroviral (ARV)-experienced patients…
- Trial name
- ODIN (NCT00524368)
- Testing
- Darunavir (DRV), Ritonavir (rtv)
- Where
- 85 sites in United States, Argentina and 17 more
- Volunteers
- 590 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
Cradle Cap, Seborrheic Dermatitis
TRIDERM D.S. Crema Normalizzante for Neonatal Hyperseborrhea (Cradle Cap) and Associated Cutaneous Manifestations of Mild-to-Moderate Seborrheic Dermatitis in Children Aged 0 to 3 Years
This clinical study aims to evaluate the effects of using the cosmetic product TRIDERM D.S. Crema Normalizzante in children aged 0 to 3 years with neonatal hyperseborrhea ("cradle cap"), potentially associated with manifestations…
- Trial name
- NCT07860788
- Testing
- TRIDERM D.S. Crema Normalizzante
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- ICIM International S.r.l.
COVID-19, SARS-CoV-2
Study of Self-Amplifying Messenger Ribonucleic Acid (samRNA) Vaccines Against COVID-19 in Healthy Adults and People Living With Human Immunodeficiency Virus (HIV)
The primary objective is to assess the safety and tolerability of samRNA vaccines GRT-R912, GRT-R914, and GRT-R918 when administered as prime and/or boost in healthy adult participants naïve to severe acute respiratory syndrome…
- Trial name
- NCT05435027
- Testing
- GRT-R912, samRNA-Spikebeta-TCE11, GRT-R914, samRNA-Spikebeta-TCE9, GRT-R918, samRNA-SpikeOmicron-N-TCE11
- Where
- 4 sites in South Africa
- Volunteers
- 342 people, healthy volunteers welcome
- Sponsor
- Gritstone bio, Inc.
Hypogammaglobulinemia
Kinetics, Efficacy and Safety of IVIG-L in Hypogammaglobulinemia Patients
The kinetics, efficacy and safety of a liquid intravenous immunoglobulin product, IVIG-L, were studied in patients with hypogammaglobulinemia, who are regularly treated with intravenous immunoglobulin substitution therapy.
- Trial name
- NCT00138697
- Testing
- IVIG-L
- Where
- 6 sites in Netherlands
- Volunteers
- 15 people
- Sponsor
- Prothya Biosolutions
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