Clinical trials: Orphan Diseases
Posttraumatic Stress Disorder, Depression
A Randomized Controlled Clinical Trial With Orphans and Widows Who Experienced the Rwandan Genocide
In the framework of the epidemiological study, local psychologists are trained in Butare in carrying out diagnostic interviews with orphans and widows who experienced the Rwandan genocide.
- Conditions
- Posttraumatic Stress Disorder, Depression, Prolonged Grief Disorder
- Trial name
- NCT00553956
- Testing
- Narrative Exposure Therapy/Interpersonal Psychotherapy
- Where
- Rwanda
- Volunteers
- 150 people
- Sponsor
- University of Konstanz
Colorectal Neoplasms, Secondary
Study of Genetic Polymorphisms of Drug Transporters and Orphan Nuclear Receptors on Treatment Effects of Irinotecan
From 100 colorectal cancer patients being treated with FOLFIRI regimen or any kind of irinotecan containing regimen, blood samples for irinotecan and its metabolites levels and genotypes related with its metabolism will be…
- Trial name
- NCT00507143
- Testing
- irinotecan
- Where
- South Korea
- Volunteers
- 100 people
- Sponsor
- National Cancer Center, Korea
Dental Caries, Knowledge
Implementation of Oral Health Educational Program on Orphan Children
Oral diseases are most prevalent among children. Orphans children are one of the most vulnerable groups to diseases especially oral diseases.
- Conditions
- Dental Caries, Knowledge, Practice
- Trial name
- NCT06428474
- Testing
- Health education
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Pharos University in Alexandria
Dysmorphia, Orphan Diseases
Automatic Phenotyping of Patients on 2D Photography
The field of artificial intelligence is booming in medicine and in the field of diagnosis. The data can be varied: x-rays, pathology sections, or photographs.
- Conditions
- Dysmorphia, Orphan Diseases, Dysmorphies Craniofaciales
- Trial name
- AIDY2 (NCT06219421)
- Testing
- Clinical data reuse
- Where
- France
- Volunteers
- 22,000 people
- Sponsor
- Imagine Institute
Takotsubo Cardiomyopathy, Cardiac X Syndrome
Brain-heart Interactions in Tako-Tsubo Cardiomyopathy and Cardiac Syndrome X:
The Tako-Tsubo Cardiomyopathy (TTC) and the Cardiac Syndrome X (CSX) are respectively acute and chronic heart diseases, which mimic myocardial infarction and stable angina pectoris without alterations of large coronary vessels.
- Conditions
- Takotsubo Cardiomyopathy, Cardiac X Syndrome, Acute Myocardial Infarction
- Trial name
- BRAINHEART (NCT02759341)
- Testing
- Manual for the Assessment and Documentation of Psychopathology (AMDP 8), Million Clinical Multiaxial Inventory 3 (MCMI-III), State-Trait Anxiety Inventory form Y (STAI-Y)
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- Ospedale San Giovanni Bellinzona
N-acetylglutamate Synthase (NAGS) Deficiency
Orphan Europe Carbaglu® Surveillance Protocol
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information.
- Trial name
- NCT03409003
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicholas Ah Mew
Fragile X Syndrome (FXS), Neurotypical Adults
Neural Biomarkers in Fragile X Syndrome
The purpose of this non-interventional test /re-test study is to assess neural biomarkers in adult subjects with Fragile X syndrome compared to those measured in a population of typically developing adults
- Trial name
- EXP-CTH120 (NCT06957054)
- Testing
- Any
- Where
- 2 sites in Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Connecta Therapeutics, S.L.
Narcolepsy, Cataplexy
Efficacy and Safety Study of BF2.649 and BF2.649 Add on Modafinil on Cataplexy in Patients With Narcolespy
The objective of this study is to evaluate and compare the efficacy and safety of escalating doses of BF2.649 and BF2.649 add on Modafinil on cataplexy in patients with narcolepsy
- Conditions
- Narcolepsy, Cataplexy, Excessive Daytime Sleepiness
- Trial name
- Harmony2 (NCT01067235)
- Testing
- BF2.649, BF2.649 add on Modafinil
- Where
- Switzerland
- Volunteers
- 14 people
- Sponsor
- Bioprojet
Autoimmune Hepatitis
Effect of JKB-122 on Prednisolone and Azathioprine Induced Remission in Autoimmune Hepatitis (AIH)
This is a Phase 2 study. All patients will receive prednisolone and AZA as standard of care (SOC) during the study. At the end of the study, all data collected will be analyzed the efficacy and safety of JKB-122 on SOC reduction…
- Trial name
- NCT04371718
- Testing
- JKB-122
- Volunteers
- 120 people
- Sponsor
- TaiwanJ Pharmaceuticals Co., Ltd
Rare Diseases, Orphan Diseases
Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases
In people suffering from a rare disease the diagnostic process and the confirmation of a final diagnosis is often ongoing for many years.
- Trial name
- ZSE-DUO (NCT03563677)
- Testing
- dual expert guidance structure
- Where
- 12 sites in Germany
- Volunteers
- 1,379 people
- Sponsor
- Wuerzburg University Hospital
Ewing Sarcoma, Ewing Family of Tumors
A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide
The goal of this clinical trial was to compare participants with first relapse or refractory Ewing's sarcoma when treated with investigational product (Vigil) in addition to the standard treatment of irinotecan and temozolomide…
- Conditions
- Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic
- Trial name
- VITA (NCT03495921)
- Testing
- Vigil, Irinotecan, Temozolomide
- Where
- 15 sites in United States
- Volunteers
- 32 people
- Sponsor
- Gradalis, Inc.
Advanced/Metastatic Solid Tumors, Relapse/Refractory Lymphoma
First in Human Study of EMB-07 in Locally Advanced/Metastatic Solid Tumors or Relapse/Refractory Lymphoma
For solid tumors and lymphoma, respectively: This study is to evaluate the safety and tolerability of EMB-07 and to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D).
- Trial name
- NCT05607498
- Testing
- EMB07
- Where
- 10 sites in Australia, China
- Volunteers
- 150 people
- Sponsor
- EpimAb Biotherapeutics (Suzhou)Co., Ltd.
Limb Girdle Muscular Dystrophy Type 2C, Gamma-sarcoglycanopathy
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
The purpose of this trial is to study the evaluation of clinical safety and feasibility of gene therapy in patients with limb girdle muscular dystrophy type 2C (gamma-sarcoglycanopathy).
- Trial name
- NCT01344798
- Testing
- AAV1-gamma-sarcoglycan vector injection
- Where
- France
- Volunteers
- 9 people
- Sponsor
- Genethon
Fibrosis Syndrome, Lymphedema
Reversing External-beam Radiotherapy-associated Fibrosis Syndrome: an Interventional Bayesian Adaptive Randomized-controlled Orphan Drug Platform Trial for Orodental Sequelae (Reverse-fibrose)
To find out if adding medication can help treat or prevent lymphedema and/or fibrosis related to radiation therapy, in survivors of head and neck cancer.
- Conditions
- Fibrosis Syndrome, Lymphedema, Head &Amp; Neck Cancer, Fibrosis
- Trial name
- NCT06912763
- Testing
- Pravastatin (drug), Pentoxifylline, ketoprofen
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- M.D. Anderson Cancer Center
Amyotrophic Lateral Sclerosis
RAPA-501 Therapy for ALS
RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
- Trial name
- NCT04220190
- Testing
- RAPA-501 Autologous T stem cells
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Rapa Therapeutics LLC
Poverty, Orphaned Children
Bridges to the Future: Economic Empowerment for AIDS-Orphaned Children in Uganda
Bridges to the Future: Economic Empowerment for AIDS-Orphaned Children in Uganda, represents the first study that measures medium-term efficacy and cost-effectiveness of a family economic empowerment intervention for…
- Conditions
- Poverty, Orphaned Children, AIDS Orphans
- Trial name
- NCT01447615
- Testing
- Bridges, Bridges PLUS, Usual Care
- Where
- Uganda
- Volunteers
- 1,410 people, healthy volunteers welcome
- Sponsor
- Columbia University
Orphan Diseases, Rare Disorders
Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients
"Rare Diseases" is an umbrella term including more than 8.000 different diseases which individually affect only a small percentage of people.
- Conditions
- Orphan Diseases, Rare Disorders, Pediatric Diseases, Inborn Errors of Metabolism Disorders
- Trial name
- NCT06729554
- Testing
- Education & Care in RARE
- Where
- 7 sites in Austria
- Volunteers
- 100 people
- Sponsor
- Medical University of Vienna
Narcolepsy, Excessive Daytime Sleepiness
Efficacy and Safety Study of BF2.649 in the Treatment of Excessive Daytime Sleepiness in Narcolepsy
The objective of this study is to evaluate the efficacy and safety of BF2.649 administered by individual titration in narcoleptic patients with excessive daytime sleepiness (EDS)
- Conditions
- Narcolepsy, Excessive Daytime Sleepiness, Cataplexy, Sleep Disorders
- Trial name
- Harmony1 (NCT01067222)
- Testing
- BF2.649, Modafinil
- Where
- Switzerland
- Volunteers
- 110 people
- Sponsor
- Bioprojet
Mitochondrial Enzyme Deficiencies
Dichloroacetate Treatment of Congenital Lactic Acidosis Phase B: Expanded Access
Expanded access to DCA as continued treatment for congenital lactic acidosis.
- Trial name
- CLA (NCT01797276)
- Testing
- Dichloroacetate
- Where
- United States
- Sponsor
- University of Florida
Fanconi Anemia Complementation Group A
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
- Trial name
- NCT04069533
- Testing
- RP-L102
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.