Clinical trials: Multiple Congenital Anomaly
Optic Pathway Glioma
Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)
Infantile optic pathway glioma (OPG) is generally benign and slow-growing, but due to infiltration and compression of sensitive neuronal structures in the optical pathways, progressive visual loss is a frequent and highly…
- Trial name
- NGF-OPG (NCT05733572)
- Testing
- CHF6467
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Benedetto Falsini
Down Syndrome, Sleep Apnea, Obstructive
19/5000 (SYNAPSOT21) Predictive Factors of Sleep Apnea Syndrome in Down Syndrome
The obstructive sleep apnea syndrome (OSAS) is frequently reported in subjects with trisomy 21. The consequences of this syndrome are expressed in various disorders such as cognitive and cardiovascular alterations.
- Conditions
- Down Syndrome, Sleep Apnea, Obstructive, Polysomnography
- Trial name
- SYNAPSOT21 (NCT03445962)
- Testing
- Assessment of OSAS predictive factors
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Grenoble
Cytomegalovirus Retinitis, HIV Infections
A Study of Foscarnet Plus Ganciclovir in the Treatment of Cytomegalovirus of the Eye in Patients With AIDS Who Have Already Been Treated With Ganciclovir
To examine the safety and tolerance of the administration of ganciclovir and foscarnet given together or alternately; to determine the interactive pharmacokinetics (blood level) profile of long-term combined and alternating…
- Trial name
- NCT00000970
- Testing
- Foscarnet sodium, Ganciclovir
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Fabry Disease, Proteinuria
The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study
The primary hypothesis is that titration of ACE inhibitor and Angiotensin Receptor Blockers (ARBs)to reduce urine protein excretion to < 500 mg per day in Fabry Patients receiving agalsidase beta therapy at 1 mg/kg every two…
- Trial name
- FAACET (NCT00446862)
- Testing
- enalapril and other angiotensin converting enzyme inhibitors; losartan and other angiotensin receptor blockers
- Where
- 6 sites in United States, Slovenia
- Volunteers
- 36 people
- Sponsor
- University of Alabama at Birmingham
Autosomal Dominant Polycystic Kidney
Thyroid Hormones in ADPKD
Autosomal dominant polycystic kidney disease (ADPKD) is a monogenic, rare and life-threatening disease, characterized by the pathological formation of multiple fluid-filled cysts that arise from renal tubules and alter kidney…
- Trial name
- REORIENTED (NCT05646420)
- Testing
- Blood sampling
- Where
- Italy
- Volunteers
- 91 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Down Syndrome
Role of Early Motor Experience in Infants With Down Syndrome
Infants with Down syndrome show significant delays and weaknesses in motor, cognitive, and language development compared to typically developing infants.
- Trial name
- NCT05144373
- Testing
- Gross motor intervention, gross and fine motor intervention
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- Georgia State University
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Multiple Osteochondroma, Enchondromatosis
Survey on Multiple Osteochondromas and Enchondromatoses During the Transition From Paediatric Care to Adult Care
This prospective observational study aims to understand the transition experiences and unmet needs of patients living with multiple osteochondromas or enchondromatoses during the paediatric to adult care, in order to produce a…
- Trial name
- PJMOE (NCT07839637)
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Tuberous Sclerosis Complex
Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex (TSC) is a multi-system disease, usually presenting with seizures, mental retardation and autism, and exhibiting a high variability in clinical findings both among and within families.
- Trial name
- TSC (NCT01289912)
- Testing
- RAD001
- Where
- 2 sites in United States
- Volunteers
- 52 people
- Sponsor
- Mustafa Sahin
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Immunologic Diagnostic Blood Test in Predicting Side-Effects in Patients Undergoing a Donor Stem Cell Transplant for Hematologic Cancer or Other Diseases
RATIONALE: Studying a diagnostic biomarker test in blood samples from patients who have undergone a donor stem cell transplant for cancer may help doctors plan treatment.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Infection, Leukemia
- Trial name
- NCT00813501
- Testing
- immunosuppressive therapy, immunological diagnostic method, allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- City of Hope Medical Center
Atopic Dermatitis, Psoriasis
Study in Healthy Participants and Participants With Moderate Atopic Dermatitis & Optionally, Moderate Psoriasis, and/or Mild Asthma
This Phase 1 study will investigate the safety and tolerability of EDP1867 in healthy volunteers, participants with atopic dermatitis, and, optionally, in participants with psoriasis and/or asthma.
- Conditions
- Atopic Dermatitis, Psoriasis, Asthma
- Trial name
- NCT04927195
- Testing
- EDP1867
- Where
- 7 sites in United Kingdom
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Evelo Biosciences, Inc.
Body Image
Impact of Exposure to Images of the Idealised Physique on Body Satisfaction, Dieting Intentions and Mood in Men.
Every day, people are exposed to huge amounts of media. Research has explored the impact of viewing traditional media and advertising (such as television and magazines) on how a person thinks and feels about their body (body…
- Trial name
- NCT03991351
- Testing
- Exposure to images
- Where
- United Kingdom
- Volunteers
- 214 people, healthy volunteers welcome
- Sponsor
- University of Leeds
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Phenylketonuria
Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)
This study is an international, multi-center, randomized, double-blind, placebo-controlled, two-treatment, two-period cross-over study to evaluate the pharmacodynamics, safety, tolerability and pharmacokinetics of a single oral…
- Trial name
- NCT04085666
- Testing
- CDX 6114
- Where
- 2 sites in Australia, Germany
- Volunteers
- 18 people
- Sponsor
- Société des Produits Nestlé (SPN)
Down Syndrome
Effects of Play Based Therapy on Attention Level of Children With Down Syndrome
The aim of the study is to examine the attention level of children with down syndrome receiving play based therapy as compared to children receiving child centered play therapy.
- Trial name
- NCT06893666
- Testing
- Play based therapy, Child centered play therapy
- Where
- Pakistan
- Volunteers
- 20 people
- Sponsor
- Riphah International University
Encephalopathy, HIV Infections
A Phase I/II Pilot Treatment Study Of CSF Penetration And Response To Ganciclovir And Foscarnet In CMV Neurologic Disease.
To determine the safety and CSF penetration of combined ganciclovir and foscarnet treatment for presumed cytomegalovirus encephalitis or radiculomyelopathy.
- Conditions
- Encephalopathy, HIV Infections, Radiculitis
- Trial name
- NCT00000856
- Testing
- Foscarnet sodium, Ganciclovir
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Myeloproliferative Disorders, Leukemia
Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- Bu Flu TBI (NCT00245037)
- Testing
- therapeutic allogeneic lymphocytes, busulfan, cyclosporine
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- OHSU Knight Cancer Institute
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis
The goal of this clinical trial is to learn if drug SPL84 is safe for adult patients with cystic fibrosis (CF). It will also learn if the drug works to treat works to treat CF with a specific mutation (3849 +10kb C-->T).
- Trial name
- NCT06429176
- Testing
- SPL84
- Where
- 3 sites in United States
- Volunteers
- 64 people
- Sponsor
- SpliSense Ltd.
Epidermolysis Bullosa
Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa
The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).
- Trial name
- NCT02014376
- Testing
- SD-101 dermal cream (3%), SD-101 Dermal Cream (6%), Vehicle (SD-101 0%)
- Where
- 7 sites in United States
- Volunteers
- 48 people
- Sponsor
- Scioderm, Inc.
Atopic Dermatitis
A Phase Ib/IIa, Double-Blind, Randomized Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of S-777469 in Subjects With Atopic Dermatitis
The purpose of this randomized, double-blind, placebo-controlled, sequential-cohort, dose-escalation study was to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of S-777469, a CB2 cannabinoid receptor…
- Trial name
- NCT00697710
- Testing
- 50 mg S-777469, 200 mg S-777469, 800 mg S-777469
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Shionogi