Clinical trials: Newborn Diseases
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Epidermolysis Bullosa
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
The purpose of this study is to evaluate the efficacy and safety of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.
- Trial name
- NCT01749306
- Testing
- ABH001, Control wound treatment
- Where
- 14 sites in United States, Austria and 6 more
- Volunteers
- 1 people
- Sponsor
- Shire Regenerative Medicine, Inc.
Huntington's Disease
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
- Trial name
- NCT03664804
- Testing
- No Study Drug was Administered in this Study
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Atopic Dermatitis, Atopic Dermatitis Eczema
The Efficacy of NTU 101 Lactic Acid Bacteria Powder in the Adjuvant Improvement of Atopic Dermatitis Clinical Trial
This study is a randomized, double-blind, placebo-controlled, parallel-controlled trial (14 weeks in total), divided into three periods (screening, treatment, and discontinuation follow-up)
- Trial name
- NCT05719649
- Testing
- Lactic acid bacteria NTU 101
- Volunteers
- 72 people
- Sponsor
- SunWay Biotech Co., LTD.
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Lower Urinary Tract Symptoms, Bladder Outlet Obstruction
A Study of Mirabegron (YM178) in Men With Lower Urinary Tract Symptoms (LUTS) and Bladder Outlet Obstruction (BOO)
This study examined the safety, tolerability, and efficacy of mirabegron (YM178) compared to placebo.
- Trial name
- NCT00410514
- Testing
- Mirabegron
- Where
- 27 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Astellas Pharma Inc
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A.
- Trial name
- NCT04418414
- Testing
- Gene therapy, Biological
- Volunteers
- 7 people
- Sponsor
- Expression Therapeutics, LLC
Skin Laxity
Rollover Study to Evaluate Histological Results of Radiofrequency Device Treatments on the Flanks
The purpose of this study is to evaluate histological results of the treatments with the TempSure Firm handpiece on the flanks performed in the CYN20-FIRM-LIPO study.
- Trial name
- NCT04881149
- Testing
- TempSure treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Cynosure, Inc.
Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies
The objective of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of S241656 and to determine the recommended dose for expansion (RDE) of S241656 in participants with relapsed/refractory (R/R) acute…
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT07719348
- Testing
- S241656, Posaconazole
- Volunteers
- 64 people
- Sponsor
- Servier
Cervical Disc Degenerative Disorder, Cervical Fusion
Post-market Observational Clinical Study With the CE-marked Implantable Class IIb Medical Device "ITC CERVICAL"
The goal of this observational post-market study is to evaluate the clinical performance and safety of the ITC CERVICAL device, a CE-marked implantable cervical interbody fusion cage, in adult patients undergoing anterior…
- Trial name
- NCT07556978
- Where
- 2 sites in Italy
- Volunteers
- 32 people
- Sponsor
- MT Ortho s.r.l.
Becker Muscular Dystrophy
A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
The ARCH study was an open-label, single-center, Phase 1b study of sevasemtem (EDG-5506) to assess the safety and pharmacokinetics (PK) of sevasemten in adults with Becker muscular dystrophy (BMD).
- Trial name
- ARCH (NCT05160415)
- Testing
- Sevasemten
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Edgewise Therapeutics, Inc.
Arteriovenous Fistula
Post Approval Study Investigating Lutonix Drug Coated Balloon for Treatment of Dysfunctional Arteriovenous Fistulae
This prospective, global, multicenter, single arm post-approval study is designed to investigate the clinical use and safety of the Lutonix® 035 AV Drug Coated Balloon (DCB) PTA Catheter in subjects presenting with clinical and…
- Trial name
- AVPAS (NCT03506308)
- Testing
- LUTONIX 035 Drug Coated Balloon PTA Catheter
- Where
- 26 sites in United States, Canada
- Volunteers
- 213 people
- Sponsor
- C. R. Bard
Non-infectious Uveitis
Safety and Efficacy of AIN457 in Noninfectious Uveitis
This study was performed to evaluate the efficacy and safety of AIN457 for patients with active uveitis that requires systemic immunosuppression.
- Trial name
- NCT00685399
- Testing
- AIN457, AIN 457
- Where
- 23 sites in United States, Germany
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals
Huntington Disease
Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease
This is the first clinical study of VY-HTT01, a gene therapy for early-stage Huntington's Disease (HD) patients. The primary goal of this trial is to evaluate the safety and tolerability of VY-HTT01.
- Trial name
- NCT04885114
- Testing
- Intraparenchymal rAAV1 - (mi)RNA HTT
- Sponsor
- Voyager Therapeutics
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital…
- Trial name
- ZEBRHA 2 (NCT07416604)
- Testing
- NXT007, Emicizumab
- Where
- 63 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Cystic Fibrosis
A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic Fibrosis
The main objective of the study is to evaluate the pharmacokinetics of brensocatib in participants with cystic fibrosis following once daily oral administration of study drug and to evaluate the safety of brensocatib compared to…
- Trial name
- NCT05090904
- Testing
- Brensocatib
- Where
- 14 sites in United States
- Volunteers
- 29 people
- Sponsor
- Insmed Incorporated
Severe Hemophilia A
Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale
The primary objective of the study is to compare the pharmacokinetic (PK) of recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) manufactured at the current scale of 2000 L (2K) to the PK of rFVIIIFc manufactured at…
- Trial name
- Elevate (NCT02502149)
- Testing
- rFVIIIFc
- Where
- 15 sites in United States, Australia and 1 more
- Volunteers
- 24 people
- Sponsor
- Bioverativ Therapeutics Inc.
Atopic Dermatitis
Safety, Tolerability, and Preliminary Efficacy of Soquelitinib in Participants With Moderate to Severe AD
Safety, tolerability, and preliminary efficacy of soquelitinib in participants with moderate to severe AD
- Trial name
- NCT06345404
- Testing
- Soquelitinib
- Where
- 16 sites in United States
- Volunteers
- 82 people
- Sponsor
- Corvus Pharmaceuticals, Inc.
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