Clinical trials: Newborn Diseases
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol
Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction
The study is to assess the long-term safety, tolerability, and efficacy after 48 and 72 weeks with monthly (Q4W [<31 days]) dosing of subcutaneous (SC) LIB003 300 mg administered in patients with CVD or at high risk for CVD…
- Conditions
- Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
- Trial name
- LIBerate-OLE (NCT04798430)
- Testing
- lerodalcibep
- Where
- 12 sites in United States, India and 4 more
- Volunteers
- 2,000 people
- Sponsor
- LIB Therapeutics LLC
Osteoarthritis, Post-traumatic Arthritis
Postmarketing Study to Determine Performance of the SIGMA HP® PARTIAL KNEE SYSTEM
This is a postmarketing study to assess the overall performance and survivorship of the SIGMA HP® PARTIAL KNEE SYSTEM.
- Conditions
- Osteoarthritis, Post-traumatic Arthritis, Gout, Pseudo-gout
- Trial name
- NCT01529099
- Testing
- SIGMA HP PARTIAL KNEE
- Where
- 3 sites in Italy, Switzerland
- Volunteers
- 64 people
- Sponsor
- DePuy International
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Asthma
A Study to Evaluate the Safety, Tolerability and Absorption to the Blood After Administration of Single and Multiple Doses of AZD8154 in Healthy Participants.
This study is a Phase 1, first in human (FiH) study, consisting of 3 parts (Part 1, Part 2, and Part 3) in healthy male and female participants of non-childbearing potential, performed at a single study center.
- Trial name
- NCT03436316
- Testing
- AZD8154
- Where
- Germany
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Lactose Intolerance
Efficacy, Safety, and Tolerability Study of RP-G28 in Subjects With Lactose Intolerance
RP-G28 is being investigated for treatment of moderate to severe lactose intolerance and its potential to improve the tolerance of lactose (dairy products).
- Trial name
- NCT02673749
- Testing
- RP-G28
- Where
- United States
- Volunteers
- 377 people
- Sponsor
- Ritter Pharmaceuticals, Inc.
Hereditary Angioedema (HAE)
Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers.
- Trial name
- NCT02663687
- Testing
- Recombinant human C1 esterase inhibitor, SHP623
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Shire
Noonan Syndrome
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested…
- Trial name
- NCT00351221
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Insmed Incorporated
Congenital Bleeding Disorder, Haemophilia A
Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients
The study has descriptive purposes, with aim of assessing how turoctocog alfa is used in the everyday practice and to provide a baseline for the management of haemophilia A and does not involve any change in the clinical…
- Trial name
- NCT06574984
- Testing
- Turoctocog alfa
- Where
- 7 sites in Iraq
- Volunteers
- 329 people
- Sponsor
- Novo Nordisk A/S
Fabry Disease
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3.
- Trial name
- NCT01853852
- Testing
- GR181413A/AT1001 solution, GR181413A/AT1001 capsule, Potable water
- Where
- Australia
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Turner Syndrome
A Study to Evaluate Subjects With Turner Syndrome Treated With Growth Hormone
This study is a multicenter, open-label, observational, postmarketing surveillance study of Genentech growth hormone (GH) products in the treatment of girls with Turner syndrome in the United States and Canada.
- Trial name
- NCT00097552
- Volunteers
- 1,696 people
- Sponsor
- Genentech, Inc.
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.
Long QT Syndrome
Effect of Oral GS-6615 on Dofetilide-Induced QT Prolongation, Safety, and Tolerability in Healthy Adults
This study will evaluate the effect of oral GS-6615 on QTc interval in healthy adults with dofetilide-induced QTc prolongation.
- Trial name
- NCT02365532
- Testing
- GS-6615, Dofetilide
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Atopic Dermatitis (AD), Atopic Dermatitis (Eczema)
Clinical Trial to Evaluate the Efficacy and Safety of an Ozonated Oil in Atopic Dermatitis
This clinical trial is designed to evaluate the efficacy, safety, and tolerability of a topical ozonated vegetable oil formulation in paediatric patients with mild-to-moderate atopic dermatitis.
- Trial name
- NCT07744516
- Testing
- ozonated oil, non-ozonated oil
- Where
- 3 sites in Spain
- Volunteers
- 50 people
- Sponsor
- Laboratorios Ozoaqua
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
The purpose of this study is: To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects treated previously with a single 7-day course of QLT091001 in Study RET IRD 01 To…
- Trial name
- NCT01521793
- Testing
- QLT091001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 27 people
- Sponsor
- QLT Inc.
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Alzheimer's Disease, Multiple Sclerosis
A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery
The purpose of this research project is to collect and store blood samples and clinical data. Researchers can then use the stored samples in future studies.
- Conditions
- Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease
- Trial name
- NCT01592552
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Dystrophic Epidermolysis Bullosa
A Follow-up Study to Evaluate the Efficacy and Safety of ALLO-ASC-DFU in ALLO-ASC-EB-101 Clinical Trial
This is an open-label follow up study to evaluate the safety for the subjects with ALLO-ASC-DFU treatment in phase 1/2 clinical trial(ALLO-ASC-EB-101) for 24 months.
- Trial name
- NCT03183934
- Testing
- ALLO-ASC-DFU
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Anterogen Co., Ltd.
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
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