Clinical trials: Deficiency Disease
HIV Infections
Tipranavir Expanded Access Program (EAP) in PI-experienced Patients With HIV-1 Infection
This study was designed to provide early access to and evaluate the safety of TPV/r in PI-experienced patients with HIV-1 infection.
- Trial name
- NCT00097799
- Testing
- Tipranavir, Ritonavir
- Where
- 115 sites in United States
- Sponsor
- Boehringer Ingelheim
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
- Trial name
- NCT02193867
- Testing
- Sebelipase Alfa
- Where
- 5 sites in United States, Finland and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Intractable Epilepsy, Glucose Transporter Type 1 Deficiency Syndrome
Evaluation of Keyo in Children With Epilepsy
To evaluate the tolerance, acceptability and compliance of Keyo in 20 subjects aged 3 years and over, with intractable epilepsy or Glut-1 DS on a KD.
- Conditions
- Intractable Epilepsy, Glucose Transporter Type 1 Deficiency Syndrome, Ketogenic Dieting
- Trial name
- Keyo (NCT02915211)
- Testing
- Keyo
- Where
- 2 sites in United Kingdom
- Volunteers
- 17 people
- Sponsor
- Vitaflo International, Ltd
Infection, Human Immunodeficiency Virus
Relative Oral Bioavailability Study of Different Fixed Dose Combinations of Dolutegravir and Rilpivirine in Healthy Subjects
Treatment of human immunodeficiency virus (HIV) infection requires daily oral administration of a combination of antiretroviral drugs to reduce the patient's HIV levels.
- Trial name
- NCT02373930
- Testing
- DTG 50 mg, RPV 25 mg, DTG/RPV 50 mg/25 mg: Product Code AS
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Hepatitis C, Chronic, Hepacivirus
Efficacy and Safety of PegIntron Plus Ribavirin for Treatment of Chronic Hepatitis C in HIV-Infected Subjects (Study P04469)(TERMINATED)
In this study, adult Indonesian subjects with human immunodeficiency virus (HIV) coinfected with chronic hepatitis C (CHC) will be given peginterferon alfa-2b (PEG-IFN) plus ribavirin (RBV) combination therapy.
- Conditions
- Hepatitis C, Chronic, Hepacivirus, HIV Infections
- Trial name
- NCT00687544
- Testing
- Peginterferon alfa-2b (SCH 054031), Ribavirin (SCH 018908)
- Volunteers
- 11 people
- Sponsor
- Merck Sharp & Dohme LLC
Growth Hormone Disorder, Adult Growth Hormone Deficiency
French National Registry of Adults With Growth Hormone Deficiency Treated With Somatropin
This study is conducted in Europe. The aim of this study is to extract data from the French National Registry holding information about patients having initiated growth hormone therapy with the objective to describe patients…
- Trial name
- NCT01580605
- Testing
- somatropin
- Where
- France
- Volunteers
- 331 people
- Sponsor
- Novo Nordisk A/S
Metastatic Colorectal Cancer (CRC)
A Study of DES-1357 in People With Deficient Mismatch Repair (dMMR)/Microsatellite Instability-High (MSI-H) Metastatic Colorectal Cancer (mCRC)
This study is conducted sequentially in 3 parts. The primary objective for Part 1 is to determine the maximum tolerated dose of DES-1357 in participants with confirmed dMMR/MSI-H colorectal cancer (CRC).
- Trial name
- NCT07807800
- Testing
- DES-1357
- Volunteers
- 88 people
- Sponsor
- D. E. Shaw Research, LLC
Infection, Human Immunodeficiency Virus
A Study to Evaluate the Effects of Calcium Carbonate and Ferrous Fumarate on Pharmacokinetics of Dolutegravir in Healthy Adult Subjects
Human immune virus (HIV) infected subjects may take mineral supplements in combination with their antiretroviral medications. Calcium and Iron supplementations are commonly used and both of these have the potential to interact…
- Trial name
- NCT01762995
- Testing
- Dolutegravir 50 mg, Calcium Carbonate 1200 mg, Ferrous Fumarate 324 mg
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
Iron-deficiency
Cross-over Study to Evaluate the Rate and the Extent of Iron Absorption of an ODF Iron Supplement vs an Iron Supplement in Capsules
Open, monocentric, comparative, cross-over study to evaluate the rate and the extent of iron absorption after a single oral dose of (A) IBSA Iron orodispersible film vs (B) SiderAL® FORTE capsules in healthy women aged 18 to 55…
- Trial name
- NCT05660200
- Testing
- IBSA Iron ODF, SiderAL® FORTE
- Where
- Italy
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- IBSA Farmaceutici Italia Srl
Ornithine Transcarbamylase Deficiency
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
The objective of the study is to characterize 24-hour plasma ammonia levels, characterize urea production rates in healthy normal subjects.
- Trial name
- NCT04269122
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Ultragenyx Pharmaceutical Inc
HIV Infections
First-Time-in-Human (FTIH) Study to Evaluate the Safety, Tolerability and Pharmacokinetics (PK) of VH4004280 in Healthy Participants
This FTIH study aims to evaluate the safety, tolerability and PK of the novel investigational Human immunodeficiency virus (HIV)-1 capsid inhibitor VH4004280 in healthy adults.
- Trial name
- NCT05163522
- Testing
- VH4004280, Midazolam
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
HIV Infections
A Study of Ritonavir (ABT-538) When Used With Nelfinavir in HIV-Infected Patients
The purpose of this study is to see if it is safe and effective to give ritonavir plus nelfinavir to HIV-infected patients. This study will also see how ritonavir and nelfinavir are absorbed by the body and how they affect the…
- Trial name
- NCT00002201
- Testing
- Ritonavir, Nelfinavir mesylate
- Where
- United States
- Sponsor
- Abbott
HIV Infections
Study Evaluating Vaccine in Adults With HIV
The main purpose of this study is to learn whether the study vaccine and the adjuvants(drugs that are used to help immune response) have an acceptable safety profile in treating individuals with HIV.
- Trial name
- NCT00195312
- Testing
- HIV-1 gag DNA (formulated with bupivacaine), IL-15 DNA (formulated with bupivacaine), IL-12 DNA (formulated with bupivacaine)
- Where
- 13 sites in United States, Germany and 1 more
- Volunteers
- 91 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Methylmalonic Acidemia, Propionic Acidemia
"The MaP Study": Mapping the Patient Journey in MMA and PA
Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to…
- Trial name
- NCT03484767
- Where
- 17 sites in United States, France and 2 more
- Volunteers
- 97 people
- Sponsor
- ModernaTX, Inc.
Postmenopausal Period, Osteoporosis, Postmenopausal
Angeliq Regulatory Post Marketing Surveillance
Angeliq regulatory Post-Marketing Surveillance (PMS) is to get data about safety and efficacy in real practice for the indication approved by Korea Food and Drug Administration (KFDA).
- Trial name
- Angeliq rPMS (NCT01064453)
- Testing
- E2/DRSP (Angeliq, BAY86-4891)
- Where
- South Korea
- Volunteers
- 4,078 people
- Sponsor
- Bayer
Anemia
Safety and Tolerability of Ferric Carboxymaltose (FCM) Versus Standard of Care in Treating Iron Deficiency Anemia
The objective of this study is to evaluate the safety of FCM in patients with anemia who are not dialysis dependent.
- Trial name
- NCT00703937
- Testing
- Ferric Carboxymaltose (FCM), Standard Medical Care (SMC) for the treatment of IDA
- Where
- United States
- Volunteers
- 708 people
- Sponsor
- American Regent, Inc.
Primary Immunodeficiency Diseases (PID)
Efficacy, Safety and Pharmacokinetic of Virchow IVIG in PID Patients
The study duration for individual patients will be up to 55 weeks for the 4-weekly and 3- weekly schedule of the treatment which includes 3 weeks for screening, 48 weeks for Study Medication administration and 4 weeks for…
- Trial name
- VBPL-IVIGP3 (NCT07017036)
- Testing
- IVIG-VImmune, IVIG-Immuglo
- Where
- 2 sites in India
- Volunteers
- 44 people
- Sponsor
- Virchow Group
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Glycogen Storage Disease, Type 14
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Study of ORL-1G in Patients With Glycogen Storage Disease Type 14
- Trial name
- NCT03404856
- Testing
- ORL-1G - D-galactose
- Where
- Turkey (Türkiye)
- Volunteers
- 5 people
- Sponsor
- Orpha Labs
Infection, Human Immunodeficiency Virus
PENTA Fosamprenavir Study
Twice daily fosamprenavir, in combination with low dose ritonavir (FPV/RTV BID), is indicated for the treatment of HIV-infected adults, adolescents and children of 6 years of age and above for use in combination with other…
- Trial name
- NCT01077635
- Testing
- Exposure to licensed dose of FPV (+/- 20% of 18mg/kg BID + RTV)
- Volunteers
- 1 people
- Sponsor
- ViiV Healthcare
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