Clinical trials: Genetic Disease
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Sanfilippo Syndrome B
Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome
This is an open-label, phase I/II study of intra-cerebral administration of adenovirus-associated viral vector containing the human NAGLU cDNA to children suffering from Sanfilippo type B syndrome.
- Trial name
- NCT03300453
- Testing
- rAAV2/5-hNAGLU
- Where
- France
- Volunteers
- 4 people
- Sponsor
- UniQure Biopharma B.V.
Mucopolysaccharidosis VI
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs).
- Trial name
- iMProveS (NCT03370653)
- Testing
- Odiparcil
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 20 people
- Sponsor
- Inventiva Pharma
Hemophilia
Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients
This study will assess the efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor
- Trial name
- NCT06922045
- Testing
- STSP-0601 for Injection
- Where
- 18 sites in China
- Volunteers
- 40 people
- Sponsor
- Jiangsu BioJeTay Biotechnology Co., Ltd.
Sickle Cell Anemia
Sickle Cell Disease: A Retrospective Chart Review
This study is a retrospective chart review of sickle cell patients and will include patients whom have received blood transfusions and those whom have not.
- Trial name
- NCT01441375
- Where
- 2 sites in United States
- Volunteers
- 261 people
- Sponsor
- Novartis Pharmaceuticals
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
An Observational Study in Children With CLN2 Batten Disease
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
- Trial name
- NCT04462692
- Sponsor
- REGENXBIO Inc.
Atopic Dermatitis, Atopic Dermatitis Eczema
Bioavailability and Tolerability of Fexofenadine Hydrochloride Topical Lotion 1%
The investigational lotion is envisaged as an short- and long term "Ease \& Prevent" monotherapy for adults and children with mild to moderate eczema.
- Conditions
- Atopic Dermatitis, Atopic Dermatitis Eczema, Atopic Rash, Rash
- Trial name
- ATOFEX-1 (NCT05461456)
- Testing
- Fexofenadine Hydrochloride Topical Lotion 1% (OP2101)
- Where
- Bangladesh
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- OrchestratePharma GmbH
Primary Immunodeficiency (PID)
Subcutaneous Ig NextGen 16% in PID Patients
This study aims to assess the safety, tolerability, efficacy and pharmacokinetics of Ig NextGen 16% in people with antibody deficiency currently being treated with IntragamP.
- Trial name
- NCT00391131
- Testing
- IgNextGen 16%
- Where
- 12 sites in Australia, New Zealand
- Volunteers
- 35 people
- Sponsor
- CSL Limited
Asthma, COPD
An Evaluation of 9MW1911 Injection in Healthy Subjects
This study is a randomized, double-blind, dose-escalating phase I clinical study to evaluate the safety, tolerability and pharmacokinetics of 9MW1911 injection in healthy subjects.
- Conditions
- Asthma, COPD, Atopic Dermatitis
- Trial name
- NCT05803902
- Testing
- Experimental drug 9MW1911
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- RESCUE (NCT02652767)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 39 people
- Sponsor
- GenSight Biologics
Hyperuricemia, Gout
Phase IIa Study of ALLN-346 (Engineered Urate Oxidase) in Subjects With Hyperuricemia, Gout and Chronic Kidney Disease
The purpose of this Phase IIa study is to evaluate the safety, tolerability and pharmacodynamics of ALLN-346 in subjects with hyperuricemia and gout, and with mild to moderate chronic kidney disease.
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Trial name
- NCT04987294
- Testing
- ALLN-346
- Where
- 17 sites in United States
- Volunteers
- 19 people
- Sponsor
- Allena Pharmaceuticals
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Dermatitis, Atopic
A Study of JNJ-39758979 in Adult Japanese Patients With Moderate Atopic Dermatitis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of JNJ-39758979 in adult Japanese patients with moderate, active atopic dermatitis.
- Trial name
- NCT01497119
- Testing
- JNJ-39758979, 300 mg, JNJ-39758979, 100 mg
- Where
- 13 sites in Japan
- Volunteers
- 88 people
- Sponsor
- Janssen Pharmaceutical K.K.
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Rectal Cancer, Ulcerative Colitis
Bioabsorbable Staple Line Reinforcement in Colorectal,Coloanal and Ileoanal Anastomoses
The primary purpose of this prospective, randomized multicenter center study is to evaluate and compare the outcomes of colorectal, coloanal and ileoanal anastomoses reinforced with a bioabsorbable staple line reinforcement…
- Conditions
- Rectal Cancer, Ulcerative Colitis, Familial Adenomatous Polyposis, Diverticulitis
- Trial name
- NCT00663819
- Testing
- GORE SEAMGUARD® Bioabsorbable Staple Line Reinforcement, Staple line without reinforcement
- Where
- 16 sites in United States
- Volunteers
- 258 people
- Sponsor
- W.L.Gore & Associates
Acute Gout
Canakinumab in the Treatment of Acute Gout Flares and Prevention of New Flares in Patients Unable to Use Non-steroidal Anti-inflammatory Drugs (NSAIDs) and/or Colchicines Including a 12 Week Extension and a 1 Year Open-label Extension Study.
The purpose of this study was to demonstrate that canakinumab given upon acute gout flares relieves the signs and symptoms and prevents recurrence of gout flares in patients with frequent flares of gout for whom non-steroidal…
- Trial name
- β-RELIEVED-II (NCT01080131)
- Testing
- Canakinumab 150 mg, Triamcinolone acetonide 40 mg
- Where
- 108 sites in United States, Canada and 3 more
- Volunteers
- 226 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis, Eczema
A Study to Assess the Long-Term Safety and Efficacy of Lebrikizumab (LY3650150) in Participants 6 Months to <18 Years of Age With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to assess the long-term safety and efficacy of lebrikizumab in participants 6 Months to <18 years of age with moderate-to-severe atopic dermatitis.
- Trial name
- ADorable-2 (NCT05735483)
- Testing
- Lebrikizumab
- Where
- 94 sites in United States, Argentina and 11 more
- Volunteers
- 338 people
- Sponsor
- Eli Lilly and Company
Dermatitis, Atopic
Phase IIa Study of WBI-1001 Cream for Atopic Dermatitis
WBI-1001 is a synthetic,new, non-steroid, small molecule being developed as a candidate drug for the topical, cream treatment of inflammatory skin diseases.
- Trial name
- WBI-1001-201 (NCT00837551)
- Testing
- WBI-1001
- Where
- Canada
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Welichem Biotech Inc.
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product 1) and Nitisinone 10 mg Tablets 'Baked' for 6 months @ 40°C/75% RH (Test Product 2) are bioequivalent to the reference product Orfadin 10 mg…
- Trial name
- NCT02750345
- Testing
- Nitisinone, Nitisinone Baked Tablet, Orfadin
- Where
- South Africa
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Alpha-Mannosidosis
Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamzede i.v. treatment of subjects with alpha-Mannosidosis.
- Trial name
- rhLAMAN-07 (NCT01908712)
- Testing
- Lamazym
- Where
- France
- Volunteers
- 13 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
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