Clinical trials: Deficiency Disease
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
HIV Infections
A Pharmacokinetic Study of Dapivirine Vaginal Rings in Belgium
IPM 018 is a double-blind, randomized, placebo-controlled study conducted at one site in Belgium among 24 healthy, HIV-negative women to evaluate dapivirine release for 28 days from matrix and reservoir intravaginal rings, each…
- Trial name
- NCT00469768
- Testing
- dapivirine reservoir intravaginal ring, dapivirine matrix intravaginal ring
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- International Partnership for Microbicides, Inc.
Xeroderma Pigmentosum
A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V
The CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin.
- Trial name
- NCT05370235
- Testing
- Afamelanotide
- Where
- 2 sites in Belgium, Spain
- Volunteers
- 6 people
- Sponsor
- Clinuvel Europe Limited
HIV/AIDS
Safety, Tolerability and Immunogenicity of MVA.HTI and ChAdOx1.HTI With Vesatolimod in HIV-1 Positive Patients
AELIX-003 study aims to investigate the safety, tolerability, immunogenicity and efficacy of a regimen containing AELIX Therapeutics' HTI T-cell vaccines and Gilead´s Toll-Like Receptor 7 (TLR7) agonist vesatolimod in…
- Trial name
- AELIX-003 (NCT04364035)
- Testing
- ChAdOx1.HTI, MVA.HTI, GS-9620
- Where
- 9 sites in Spain
- Volunteers
- 50 people
- Sponsor
- Aelix Therapeutics
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Observational Prospective Study on Patients Treated With Norditropin®
This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Adult Growth Hormone Deficiency, Genetic Disorder
- Trial name
- NCT00960128
- Testing
- somatropin
- Where
- 23 sites in Argentina, Belgium and 21 more
- Volunteers
- 21,249 people
- Sponsor
- Novo Nordisk A/S
Hereditary Angioedema (HAE)
A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China
The Chinese health authority has approved lanadelumab to prevent Hereditary Angioedema (HAE) attacks in persons of 12 years and older. It has also approved icatibant to treat acute HAE attacks in persons 2 years and older.
- Trial name
- NCT06346899
- Testing
- No intervention
- Where
- 13 sites in China
- Volunteers
- 115 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorder
Study of Octanorm Subcutaneous IG in Patients With PID
This is an open-label phase III study with a 12-week wash-in/wash-out period followed by a 12-month efficacy period. The main goals of the study are to assess the efficacy of octanorm in preventing serious bacterial infections…
- Trial name
- NCT01888484
- Testing
- octanorm 16.5%
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 75 people
- Sponsor
- Octapharma
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II)
Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
To describe severe late onset patients with pompe disease receiving Myozyme®
- Conditions
- Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
- Trial name
- NCT00731081
- Where
- France
- Volunteers
- 8 people
- Sponsor
- Genzyme, a Sanofi Company
HIV Infections
A Phase I/II Safety and Immunogenicity Trial of UBI Microparticulate Monovalent (HIV-1 MN) Branched Peptide Vaccine in HIV-1 Seronegative Human Subjects
To evaluate the safety and immunogenicity of a combination of microparticulate monovalent HIV-1 MN synthetic branched peptide candidate vaccine for oral administration and monovalent HIV-1 MN synthetic branched peptide vaccine in…
- Trial name
- NCT00002428
- Testing
- HIV-1 Peptide Vaccine, Microparticulate Monovalent, rgp120/HIV-1MN Monovalent Octameric V3 Peptide Vaccine
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- United Biomedical
Anemia, Iron Deficiency Anemia
Aptus at Home: Co-design of a Home-based Monitoring System for Anaemia
Co-design of a home-based anaemia monitoring system with participants who have previous diagnosis of anaemia.
- Trial name
- NCT03835364
- Testing
- Blood measurement
- Where
- United Kingdom
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Entia Ltd
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Pituitary Disorders, Adult Growth Hormone Deficiency
Treatment of Adults With Growth Hormone Deficiency
The purpose of this study is to evaluate efficacy and safety profile of a new weekly administered growth hormone preparation compared with placebo in adults with growth hormone deficiency.
- Trial name
- NCT00294619
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- LG Life Sciences
HIV Infections
Non-interventional Observational Study With Viramune® in HIV to Evaluate Gender Specific Data
The question that prompted this study is the extent to which results from clinical trials can be translated into everyday practice (external validity).
- Trial name
- NCT01134939
- Where
- 34 sites in Germany
- Volunteers
- 265 people
- Sponsor
- Boehringer Ingelheim
Hereditary Angioedema
Patient Registry to Evaluate the Real-world Safety of Ruconest®
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
- Trial name
- NCT03697187
- Testing
- rhC1INH
- Where
- United States
- Volunteers
- 152 people
- Sponsor
- Pharming Technologies B.V.
Human Immunodeficiency Virus (HIV) Infection
Pharmacokinetics, Safety And Toleration Of Maraviroc Administered To Subjects With Various Degrees Of Renal Impaired And Normal Renal Function
The purpose of this study is to assess whether a dosing adjustment is needed in patients with renal impairment.
- Trial name
- NCT00717067
- Testing
- Maraviroc, Ritonavir, Saquinavir
- Where
- 2 sites in Germany
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
HIV Infections
Phase 2b, Open-label, Multicenter, Rollover Study to Assess Antiviral Activity and Safety of Long-acting (LA) Cabotegravir (CAB) Plus LA Rilpivirine (RPV), Administered Every 2 Months (Q2M), in Human Immunodeficiency Virus (HIV)-Positive Participants From the LATTE Study
This study (POLAR), is designed to assess the antiviral activity and safety of CAB LA plus RPV LA, administered Q2M, in approximately 100 adult HIV-1 infected, antiretroviral therapy (ART) experienced participants.
- Trial name
- NCT03639311
- Testing
- CAB LA, RPV LA, RPV
- Where
- 30 sites in United States, Canada
- Volunteers
- 97 people
- Sponsor
- ViiV Healthcare
HIV Infection
Safety and Efficacy Study of AGS-004 During Analytical Treatment Interruption
The purpose of this study is to determine the safety and effectiveness of AGS-004, an immune therapy, for HIV-infected individuals.
- Trial name
- NCT01069809
- Testing
- AGS-004
- Where
- 9 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Argos Therapeutics
Dry Eye Syndrome
SYSTANE® BALANCE Symptoms (OSDI) & Inflammatory Biomarkers
The purpose of this study is to evaluate the ability of SYSTANE® BALANCE and SYSTANE® Gel to improve comfort in subjects with dry eyes.
- Trial name
- NCT01733732
- Testing
- SYSTANE® BALANCE Lubricant Eye Drops, SYSTANE® Gel
- Volunteers
- 54 people
- Sponsor
- Alcon Research
Hereditary Angioedema (HAE)
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Takhzyro is an approved treatment for hereditary angioedema (HAE) in South Korea. HAE is a rare condition which causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07445087
- Testing
- No intervention
- Where
- 3 sites in South Korea
- Volunteers
- 35 people
- Sponsor
- Takeda
HIV Infections
To Assess the Relative Bioavailability (BA) of TRIUMEQ® and Dolutegravir and Lamivudine (DTG/3TC) Pediatric Dispersible Tablet Formulations in Healthy Volunteers
This is a 2-part, single-dose, open label, randomized 3-way cross-over study to compare BA of pediatric study drugs TRIUMEQ and (DTG/3TC) in healthy volunteers under fasted conditions. Study will be conducted in 2-parts.
- Trial name
- NCT03441984
- Testing
- Treatment A, Treatment B, Treatment C
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare