Clinical trials: Congenital Anomalies
Cranioplasty, Craniofacial Abnormalities
Stryker PEEK Customized Implant: A Prospective Post Market Follow Up to Evaluate Safety, Performance and Effectiveness
The purpose of this study is to confirm the safety, performance and effectiveness of Stryker's PEEK Customized Implants when used for the augmentation and/or restoration of bony and/or soft tissue deformities in the cranial and…
- Conditions
- Cranioplasty, Craniofacial Abnormalities, Craniofacial Injuries
- Trial name
- NCT05362370
- Testing
- Stryker's PEEK Customized Implant
- Where
- 13 sites in United States, Austria and 4 more
- Volunteers
- 99 people
- Sponsor
- Stryker Craniomaxillofacial
Phenylketonuria (PKU)
A Study of MZE782 in Adults With PKU
The purpose of this study is to evaluate the safety, tolerability, and efficacy of MZE782 in adults with Phenylketonuria (PKU).
- Trial name
- CIPheR (NCT07694440)
- Testing
- MZE782
- Where
- 9 sites in United States, France and 1 more
- Volunteers
- 38 people
- Sponsor
- Maze Therapeutics
Hemophilia B
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B.
- Trial name
- PUPs B-LONG (NCT02234310)
- Testing
- rFIXFc
- Where
- 27 sites in United States, Australia and 9 more
- Volunteers
- 33 people
- Sponsor
- Bioverativ, a Sanofi company
GM1 Gangliosidosis, GM2 Gangliosidosis
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2).
- Conditions
- GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease
- Trial name
- RETRIEVE (NCT04470713)
- Where
- 17 sites in United States, Belgium and 8 more
- Volunteers
- 226 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
MPS IIIB (Sanfilippo B Syndrome)
Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
- Trial name
- NCT02090179
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myotonic Dystrophy Type 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- ACHIEVE (NCT05481879)
- Testing
- DYNE-101
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 127 people
- Sponsor
- Dyne Therapeutics
Nystagmus, Congenital, Nystagmus, Acquired
Open-lable Extension Study on Safety and Efficacy of Neramexane to Treat Congenital and Acquired Nystagmus
The purpose of this study is to investigate the long-term safety, tolerability and efficacy of neramexane mesylate in the treatment of congenital idiopathic nystagmus (CIN).
- Conditions
- Nystagmus, Congenital, Nystagmus, Acquired, Multiple Sclerosis
- Trial name
- NCT00799942
- Testing
- Neramexane mesylate
- Where
- United Kingdom
- Volunteers
- 48 people
- Sponsor
- Merz Pharmaceuticals GmbH
Chronic Cutaneous Ulcer, Sickle Cell Disease
Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment
Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).
- Trial name
- D-SCOUT (NCT04058197)
- Testing
- Deferoxamine Product
- Where
- 4 sites in United States
- Sponsor
- TauTona Group
Anemia, Sickle Cell
A Study of the Absorption, Metabolism, and Excretion of GBT440 in Healthy Male Subjects
This study will provide information regarding the metabolic pathway of GBT440, the need for evaluation of potential drug-drug interactions, and the need for studies in special populations.
- Trial name
- NCT02497924
- Testing
- GBT440
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Global Blood Therapeutics
Gout, Coronary Artery Disease
Uric Acid Study in Healthy Male Volunteers
This study is being conducted to examine the effect of AZD6140 on the levels of certain chemicals in subjects' blood and urine.
- Trial name
- NCT00738842
- Testing
- AZD6140
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Gout
DECT Study in Allopurinol-Treated Gout Patients
The purpose of this study is to determine presence and volume of monosodium urate (MSU) crystal deposits as determined by dual-energy computed tomography (DECT) imaging in gout subjects treated with allopurinol with and without…
- Trial name
- NCT02393560
- Testing
- DECT scan
- Where
- 10 sites in United States, New Zealand
- Volunteers
- 223 people
- Sponsor
- Ardea Biosciences, Inc.
Alpha-Mannosidosis
Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years
- Trial name
- rhLaman-08 (NCT02998879)
- Testing
- Velmanase Alfa (e.g. Lamazym)
- Where
- 6 sites in Austria, Denmark and 3 more
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Prader-Willi Syndrome
Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating the safety and efficacy of DCCR treatment.
- Trial name
- NCT03714373
- Testing
- DCCR
- Where
- 28 sites in United States, United Kingdom
- Volunteers
- 115 people
- Sponsor
- Soleno Therapeutics, Inc.
Hypercholesterolemia, Familial Hypercholesterolemia
Long Term Post Marketing Specified Drug Use Result Survey for Evolocumab in Japan
The purpose of this post-marketing survey is to obtain real-world information on the safety and effectiveness of evolocumab in Japan.
- Trial name
- NCT02808403
- Testing
- No intervention
- Where
- 138 sites in Japan
- Volunteers
- 6,000 people
- Sponsor
- Amgen
Cystic Fibrosis
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
- Trial name
- NCT04362761
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 29 sites in Australia, Belgium and 2 more
- Volunteers
- 172 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Spinal Deformity, Spinal Degenerative Disorder
The Ailliance Post-Market Clinical Study
The purpose of this clinical study is to collect performance and safety data for post-market Medtronic devices indicated for cranial and/or spinal indication(s). Subjects are enrolled and followed postoperatively to 24 months.
- Conditions
- Spinal Deformity, Spinal Degenerative Disorder, Spinal Fusion Failure, Spinal Trauma
- Trial name
- NCT05856370
- Testing
- Follow-up schedule: pre-operative baseline up to 24-months post-procedure
- Where
- 29 sites in United States, France and 4 more
- Volunteers
- 1,000 people
- Sponsor
- Medtronic Spinal and Biologics
Hemophilia A
BAX 855 PK-guided Dosing
1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%) 2.
- Trial name
- PROPEL (NCT02585960)
- Testing
- PEGylated Recombinant Factor VIII
- Where
- 83 sites in United States, Australia and 20 more
- Volunteers
- 135 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
- Trial name
- NCT06634420
- Testing
- NTLA-2002, Normal Saline IV Administration
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- Intellia Therapeutics
Sickle Cell Disease
Absorption, Metabolism, and Excretion of a Single Dose of Ferriprox® in Patients With Sickle Cell Disease
The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease.
- Trial name
- NCT01835496
- Testing
- single 1500 mg dose of Ferriprox
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- ApoPharma
Lower Eyelid Steatoblepharon (Excess Eyelid Fat)
Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe steatoblepharon (excess eyelid fat).
- Trial name
- NCT02230761
- Testing
- XOPH5 Ointment
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Topokine Therapeutics, Inc.
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