Clinical trials: Orphan Diseases
Dental Plaque, Gingival Diseases
Effectiveness of Dental Health Education Methods on Oral Hygiene, Oral Health Care Self-Efficacy and Self-Esteem of Young Orphans
Background: Orphan children are at higher risk of developing oral diseases, compared to non-orphans. They also face various emotional, social, and psychological distresses which affect their general as well as oral health.
- Conditions
- Dental Plaque, Gingival Diseases, Oral Health, Self Efficacy
- Trial name
- NCT07771634
- Testing
- Conventional dental health education, Game based dental health education, Peer-group based dental health education
- Where
- Egypt
- Volunteers
- 264 people
- Sponsor
- Alexandria University
Cystinosis
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
This was a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who were naïve to any form of cysteamine treatment.
- Trial name
- NCT01744782
- Testing
- RP103
- Where
- 2 sites in United States, Brazil
- Volunteers
- 17 people
- Sponsor
- Amgen
Lung Diseases, Interstitial, Pulmonary Alveolar Proteinosis
European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank
Generation of a common European database and biobank Continous assessment and implementation of guidelines and treatment protocols Establishment of a large observational cohort of chILD patients Determination the value of…
- Conditions
- Lung Diseases, Interstitial, Pulmonary Alveolar Proteinosis, Pulmonary Eosinophilia, Pulmonary Fibrosis
- Trial name
- chILD-EU (NCT02852928)
- Where
- 3 sites in Germany, Turkey (Türkiye) and 1 more
- Volunteers
- 1,000 people
- Sponsor
- Matthias Griese
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat…
- Conditions
- Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
- Trial name
- ATALANTa (NCT03679598)
- Testing
- Alvelestat (MPH966)
- Where
- 10 sites in United States
- Volunteers
- 63 people
- Sponsor
- University of Alabama at Birmingham
Cystinosis
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis…
- Trial name
- NCT01733316
- Testing
- RP103, Cystagon®
- Where
- 14 sites in United States, Belgium and 4 more
- Volunteers
- 41 people
- Sponsor
- Amgen
HIV/AIDS
Bridges2Scale: Testing Implementation Strategies
The goal of this clinical trial is to compare two multifaceted strategies (standard vs enhanced) for scaling Bridges in a two-arm Hybrid III effectiveness-implementation cluster randomized controlled trial (RCT) in adolescent and…
- Trial name
- NCT06347146
- Testing
- Financial Literacy Training (FLT) workshops, Mentorship, Income Generation Activity (IGA)
- Where
- Uganda
- Volunteers
- 1,440 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Narcolepsy
Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients
The initial portion of the protocol involves discontinuing any medications for cataplexy that the patient may be taking. Subsequently, the patient is prescribed a dose of oral solution of study drug or placebo over a 10-11 week…
- Trial name
- NCT00049803
- Testing
- sodium oxybate
- Where
- 45 sites in United States, Canada and 1 more
- Volunteers
- 200 people
- Sponsor
- Orphan Medical
Porphyria Cutanea Tarda
Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea Tarda
Porphyria cutanea tarda (PCT) is an iron-related disorder that responds to treatment by phlebotomy or low-dose hydroxychloroquine, but comparative data on these treatments are limited.
- Trial name
- NCT01573754
- Testing
- Hydroxychloroquine, Phlebotomy
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Eczema/Atopic Dermatitis, Cancer
Patient Forward Access to Clinical and Technological Research: Genetic Influences on Cancer and Atopic Dermatitis
This Patient Forward study intends to conduct research to investigate potential genetic factors causing cancer and eczema/atopic dermatitis.
- Trial name
- PFACTR02 (NCT04362852)
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Syndrome X, Takotsubo Cardiomyopathy
Tako-Tsubo Cardiomyopathy and Cardiac Syndrome X: New Insights Into the Pathophysiology
Tako-Tsubo Cardiomyopathy (TTC) and Cardiac Syndrome X (CSX) are respectively acute and chronic cardiac conditions whose clinical presentation, mimicking the onset of acute myocardial ischemia in absence of epicardial coronary…
- Trial name
- ENDAUT (NCT02307214)
- Testing
- BaroReflex Sensitivity, endothelial function measurement
- Where
- Switzerland
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Ospedale San Giovanni Bellinzona