Clinical trials: Genetic Diseases
Atopic Dermatitis
Study of Generic Pimecrolimus Cream, 1% in the Treatment of Mild to Moderate Atopic Dermatitis
This is a Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multiple-Site Study to Evaluate the Therapeutic Equivalence of a Generic Pimecrolimus Cream, 1% (Glenmark Pharmaceuticals Ltd) to the Marketed Product…
- Trial name
- NCT02896101
- Testing
- Pimecrolimus Cream, 1%, Elidel® (pimecrolimus) Cream
- Where
- 27 sites in United States
- Volunteers
- 755 people
- Sponsor
- Glenmark Pharmaceuticals Ltd. India
Cervical Cancer
Tisotumab Vedotin vs Chemotherapy in Recurrent or Metastatic Cervical Cancer
This trial is being done to find out whether tisotumab vedotin works better than chemotherapy to treat cervical cancer. People in this study have cervical cancer that has spread to other parts of the body (metastatic) or has come…
- Trial name
- innovaTV 301 (NCT04697628)
- Testing
- tisotumab vedotin, topotecan, vinorelbine
- Where
- 211 sites in United States, Argentina and 23 more
- Volunteers
- 502 people
- Sponsor
- Seagen, a wholly owned subsidiary of Pfizer
Congenital Bleeding Disorder, Haemophilia A
Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries
This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- HAEMOcare (NCT01503567)
- Testing
- No treatment given
- Where
- 4 sites in India, Morocco and 2 more
- Volunteers
- 282 people
- Sponsor
- Novo Nordisk A/S
Myelofibrosis
Actuate 1901: 9-ING-41 in Myelofibrosis
9-ING-41 has anti-cancer clinical activity while not causing myelosuppression, and has both pre-clinical anti-fibrotic activity and activity against myelofibrosis.
- Trial name
- NCT04218071
- Testing
- Ruxolitinib, 9-ING-41
- Where
- 9 sites in United States
- Volunteers
- 17 people
- Sponsor
- Actuate Therapeutics Inc.
Pompe Disease, Glycogen Storage Disease Type II
A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease
The purpose of this study is to see how molecules called pharmacological chaperones affect the cells of patients with Pompe disease.
- Trial name
- NCT00515398
- Testing
- Observation
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Amicus Therapeutics
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
The purpose of this study was to evaluate the safety and efficacy of revusiran (ALN-TTRSC) in patients with transthyretin (TTR) mediated Familial Amyloidotic Cardiomyopathy.
- Conditions
- Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies
- Trial name
- NCT02319005
- Testing
- Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
- Where
- 61 sites in United States, Belgium and 7 more
- Volunteers
- 206 people
- Sponsor
- Alnylam Pharmaceuticals
Phenylketonurias
Evaluation of Low Phenylalanine Formulas
Ajinomoto Cambrooke has developed a PKU protein substitute that is a proprietary blend of purified Glycomacropeptide (GMP) and essential amino acids, under the brand name Glytactin®.
- Trial name
- NCT06332105
- Testing
- Glytactin, L-AA
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Ajinomoto Co., Inc.
Congenital Bleeding Disorder, Haemophilia A
Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects
This trial is conducted in Europe and Asia. The aim of this clinical trial is to compare two recombinant factor VIII drugs, turoctocog alfa (recombinant factor VIII (N8)) with Advate®, in haemophilia A subjects, investigating the…
- Trial name
- NCT00837356
- Testing
- Advate®, turoctocog alfa
- Where
- 7 sites in Germany, Israel and 3 more
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Atopic Dermatitis
Topical Solution for the Treatment of Atopic Dermatitis
The study is designed as a proof of concept, multi-center, randomized, double-blind and vehicle-controlled study with intra-individual comparison of treatments.
- Trial name
- CYCLATOP (NCT02865356)
- Testing
- Cyclosporine 5% solution
- Where
- 9 sites in Spain
- Volunteers
- 36 people
- Sponsor
- Spherium Biomed
Mucopolysaccharidosis VI
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in…
- Trial name
- NCT00048620
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Sponsor
- BioMarin Pharmaceutical
Atopic Dermatitis
Efficacy and Safety of Pimecrolimus Cream 1% in Patients ≥ 3 Months of Age With Mild or Moderate Atopic Dermatitis
To evaluate the efficacy and safety of pimecrolimus cream 1% in its registered indication (treatment of patients > 3 months of age with mild to moderate atopic dermatitis) This study is not enrolling patients in the United States.
- Trial name
- NCT00150059
- Testing
- Pimecrolimus Cream 1%
- Where
- Philippines
- Sponsor
- Novartis
Inherited Epidermolysis Bullosa
Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
The purpose of this study was to compare intra-individually the reepithelialization of skin lesion(s) in inherited Epidermolysis bullosa (either 1 wound ≥10 cm2 and ≤200 cm2 in size divided in 2 equal halves or 2 comparable…
- Trial name
- NCT01294241
- Testing
- Oleogel-S10, Mepilex® soft silicone faced polyurethane foam dressing
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Birken AG
Cystic Fibrosis
International Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
The purpose of this study was to evaluate the safety and efficacy of a 28-day course of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF1 (NCT00112359)
- Testing
- AZLI 75 mg three times a day (TID)
- Where
- 53 sites in United States, Australia and 2 more
- Volunteers
- 166 people
- Sponsor
- Gilead Sciences
Haemophilia A and B With and Without Inhibitors
A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors
This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use.
- Trial name
- Explorer10 (NCT05135559)
- Testing
- Concizumab
- Where
- 91 sites in United States, Algeria and 22 more
- Volunteers
- 153 people
- Sponsor
- Novo Nordisk A/S
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring
Idiopathic Pulmonary Fibrosis
Assess the Safety, Tolerability and Pharmacokinetics of AZD5055 Following Single and Multiple Ascending Doses in Healthy Participants
This is a phase I, First-in-Human study in healthy participants, performed at a single study center, consisting of 2 parts: Part 1 is a single ascending dose (SAD) study and Part 2 is a multiple ascending dose (MAD) study.
- Trial name
- NCT05134727
- Testing
- AZD5055
- Where
- United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Age-Related Macular Degeneration
NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration
This study will evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy administered by subretinal injections into a single selected eye as a single selected dose for patients with wet age-related…
- Trial name
- NCT05984927
- Testing
- NG101 AAV gene therapy
- Where
- 4 sites in United States, Canada
- Volunteers
- 20 people
- Sponsor
- Elisigen, Inc.
Leber Hereditary Optic Neuropathy
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.
- Trial name
- NCT02064569
- Testing
- GS010
- Where
- France
- Volunteers
- 19 people
- Sponsor
- GenSight Biologics
Down Syndrome
Phase 1/2 Trial of AEF0217 in Participants With Down Syndrome
AEF0217-102 clinical trial assesses the safety, tolerability, plasma exposure and preliminary indications of pharmacodynamic activity of AEF0217 in female and male adult participants with Down syndrome between 18 and 35 years…
- Trial name
- MDDS (NCT05748405)
- Testing
- AEF0217
- Where
- 3 sites in Spain
- Volunteers
- 40 people
- Sponsor
- Aelis Farma
Cutis Laxa
Effectiveness and Safety of the Ulthera® System for Skin Laxity in the Lower Face, Submentum and Neck
The purpose of this study is to demonstrate superiority of treatment with the Ulthera System compared to untreated control for the improvement of skin laxity of the lower face, submental area and neck and to demonstrate the…
- Trial name
- NCT07614997
- Testing
- Ulthera System
- Where
- 7 sites in China
- Volunteers
- 218 people, healthy volunteers welcome
- Sponsor
- Merz North America, Inc.
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