Clinical trials: Congenital Anomaly
Congenital Hemangioma, Hemangioendothelioma
A Study to Evaluate How Safe and Effective is the Mixture of Lipiodol® Ultra Fluid and Glue When Used for Embolization Procedures
This was a multicenter phase IV Post-Marketing Study. The study is designed to investigate the safety of Lipiodol® Ultra Fluid in association with surgical glues used according to each site medical practice of vascular…
- Conditions
- Congenital Hemangioma, Hemangioendothelioma, Angiosarcoma, Arteriovenous Malformations
- Trial name
- NCT02625389
- Testing
- Lipiodol Ultra Fluid with surgical glues
- Where
- 16 sites in India
- Volunteers
- 132 people
- Sponsor
- Guerbet
Primary Hyperoxaluria Type 1
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Trial name
- NCT02795325
- Testing
- DCR-PH1
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 41 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Mucopolysaccharidosis I, Hurlers Syndrome
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT00912925
- Testing
- rhIDU (recombinant human-Alpha-L-Iduronidase)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Gouty Arthritis (GA)
A Bioequivalence Study of Two Different Dosage Form of Genakumab
The purpose of this study is to investigate whether Genakumab Injection is bioequivalent to Genakumab for Injection.
- Trial name
- NCT06451848
- Testing
- Genakumab Injection (T, Solution for injection), Genakumab for Injection (R, Powder for solution for injection)
- Where
- China
- Volunteers
- 178 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Pediatric Oncology
A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy
Dose escalation phase of the study : To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in…
- Trial name
- NCT02085148
- Testing
- Regorafenib (BAY73-4506), Vincristine (Cellcristin®), Irinotecan (Irinotecan Cell pharm®)
- Where
- 12 sites in France, Italy and 2 more
- Volunteers
- 62 people
- Sponsor
- Bayer
Duchenne Muscular Dystrophy
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
- Trial name
- NCT02752048
- Testing
- TAS-205
- Where
- 11 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants.
- Trial name
- PROTECT-ALT (NCT06684314)
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Taiwan
- Volunteers
- 79 people
- Sponsor
- Sanofi
Down Syndrome, Edwards Syndrome
Whole Blood Specimen Collection From Pregnant Subjects
To obtain whole blood specimens from pregnant subjects to be used for research and development and clinical validation studies of prenatal assays.
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Klinefelter Syndrome
- Trial name
- NCT02430584
- Testing
- Observational - no intervention
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Progenity, Inc.
Missing Teeth
Post Market Clinical FoIIow up (PMCF) Medical Device (MD) of Z-Systems Monotype Zirconia Dental Product: Z5m Dental Implants Registry
Commercially pure (CP) titanium is the gold standard to produce dental implants because it has a huge volume of scientific publications over a period of more than 50 years, it is cheap and simple to produce (i.e., making dental…
- Trial name
- NCT06007365
- Testing
- Z5m Dental Implants
- Where
- Switzerland
- Volunteers
- 70 people
- Sponsor
- Z-Systems
Pompe Disease
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
- Trial name
- NCT02675465
- Testing
- ATB200, AT2221
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 29 people
- Sponsor
- Amicus Therapeutics
Dermatitis, Atopic, Eczema
Skin Effects of a Topical Amino Acid Moisturizing Cream and Desonide in Atopic Dermatitis
The purpose of this study is to compare the effects of an amino acid moisturizing cream and desonide cream in reducing skin symptoms associated with AD/eczema in subjects with mild to moderate atopic dermatitis/eczema.
- Trial name
- NCT02286700
- Testing
- Desonide Cream, Amino Acid Moisturizing Cream
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- NeoStrata Company, Inc.
Congenital Adrenal Hyperplasia
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen.
- Trial name
- NCT04457336
- Where
- 65 sites in United States, Australia and 18 more
- Volunteers
- 96 people
- Sponsor
- Spruce Biosciences
Genetic Disorder, Noonan Syndrome
Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658
This trial is conducted in Europe. The aim of this trial is to obtain the PTPN11 mutation status and investigate the impact of the PTPN11 mutation status on the effect of somatropin (Norditropin®) by use of data obtained in the…
- Trial name
- NCT01529944
- Testing
- somatropin
- Where
- Sweden
- Volunteers
- 22 people
- Sponsor
- Novo Nordisk A/S
Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic
AV-23-001 AVAVA MIRIA Pilot Study
This non-significant risk study is intended to demonstrate the use of the MIRIA Skin Treatment System can show improvement in dermatologic conditions currently indicated for treatment with a laser (such as, but not limited to…
- Conditions
- Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic, Pigmented Lesions, Skin Laxity
- Trial name
- NCT06557434
- Testing
- MIRIA
- Where
- 2 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- AVAVA, Inc.
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Choroideremia
Natural Disease Progression in Participants With Choroideremia
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants.
- Trial name
- NCT04795206
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1,178 people
- Sponsor
- Biogen
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
Duchenne Muscular Dystrophy
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
This is an open label, long-term extension study in approximately 24 male DMD subjects consisting of children (ages 4-12, inclusive) and adolescents (ages 13-16, inclusive) who participated in the MP-104-CL-005 PK study.
- Trial name
- NCT02295748
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
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