Clinical trials: Multiple Disability
Genetic Disorder, Prader-Willi Syndrome
Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers
This trial is conducted in United States of America (USA). The aim of this trial is to examine the bioequivalence of Norditropin® versus Genotropin® in healthy adult volunteers.
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature
- Trial name
- NCT01401244
- Testing
- somatropin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome
Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome
The purpose of this is study is to evaluate the long-term safety of DCCR (diazoxide choline) extended-release tablets) in patients with Prader-Willi syndrome.
- Trial name
- NCT05701774
- Testing
- DCCR
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 77 people
- Sponsor
- Soleno Therapeutics, Inc.
Down Syndrome, Fetal Aneuploidy
Specimen Collection From Pregnant Women at Increased Risk for Fetal Aneuploidy
The specimen collection is designed for the purpose of the development of a noninvasive prenatal test for T21.
- Trial name
- NCT01429389
- Where
- 14 sites in United States, Canada
- Volunteers
- 2,000 people
- Sponsor
- Sequenom, Inc.
Urothelial Carcinoma
Study of Mocetinostat in Patients With Urothelial Carcinoma Having Inactivating Alterations of Specific Genes
Mocetinostat is an orally administered histone deacetylase (HDAC) inhibitor. This study is a Phase 2 trial evaluating the efficacy of mocetinostat in patients that have advanced urothelial carcinoma that has specific changes in…
- Trial name
- NCT02236195
- Testing
- Mocetinostat
- Where
- 14 sites in United States
- Volunteers
- 17 people
- Sponsor
- Mirati Therapeutics Inc.
Chromosome 13 Aneuploidy, Chromosome 18 Aneuploidy
Non-invasive Prenatal Diagnostic Validation Study
The primary purpose of this study is to collect maternal blood samples from pregnant women to develop a non-invasive prenatal diagnostic test based on fetal DNA isolated from maternal blood.
- Conditions
- Chromosome 13 Aneuploidy, Chromosome 18 Aneuploidy, Chromosome 21 Aneuploidy, Sex Chromosome Aberrations
- Trial name
- NIPD (NCT01574781)
- Testing
- Blood draw, Cheek swab/Saliva Sampling
- Where
- United States
- Volunteers
- 1,781 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Trisomy 21
Paternal Age and Fetal Aneuploidy
Trisomy 21, commonly known as down syndrome, is the most common chromosomal abnormality in humans. Advanced maternal age (AMA) is a well-recognized risk factor for trisomy 21, with the risk increasing significantly beyond the age…
- Trial name
- NCT05981521
- Testing
- Optimo prenatal screening test
- Where
- Canada
- Volunteers
- 7,141 people
- Sponsor
- Clinique Ovo
Multiple Sclerosis, Relapsing-Remitting
A Canadian Study Assessing the Utility of the Treatment Optimization Recommendations in Multiple Sclerosis
The Canadian Multiple Sclerosis Working Group (CMSWG) has developed practical recommendations on how neurologists can assess the status of subjects on disease modifying drugs (DMDs) and decide when it may be necessary to modify…
- Trial name
- CanTOR (NCT01142583)
- Volunteers
- 192 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Prader-Willi Syndrome
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
- Trial name
- NCT03554031
- Testing
- Recombinant Human Growth Hormone (rhGH) Injection
- Where
- 6 sites in China
- Volunteers
- 30 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Trisomy 13, Trisomy 18
Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our new NATUS diagnostic method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278536
- Where
- 8 sites in United States, India and 1 more
- Volunteers
- 354 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Secondary Progressive Multiple Sclerosis With Inflammatory Disease Activity
Swiss Study of the Impact of Mayzent on SPMS Patients in a Long-term Non-interventional Study
This study is a national, prospective, multicenter, non-interventional (observational) study with the aim to describe the impact of Siponimod treatment in a real-world SPMS population in Switzerland who are treated with Siponimod…
- Trial name
- SWISSMASIA (NCT04895202)
- Testing
- Siponimod
- Where
- 6 sites in Switzerland
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals
Down Syndrome (Trisomy 21)
AEF0217 in Participants With Down Syndrome
The goal of this clinical trial is to identify if AEF0217 show an improvement in adaptive behaviors (daily life activities) in adults and older adolescents with Down Syndrome. It will also learn about the safety of AEF0217.
- Trial name
- NCT07334912
- Testing
- AEF0217 100 µg, AEF0217 300 µg
- Where
- 10 sites in France, Italy and 1 more
- Volunteers
- 188 people
- Sponsor
- Aelis Farma
Down Syndrome, Edwards Syndrome
Non-Invasive Screening for Fetal Aneuploidy
The purpose of this study is to detect whole chromosome abnormalities in maternal blood.
- Trial name
- NCT02317965
- Testing
- Maternal Blood Draw
- Where
- 10 sites in United States
- Volunteers
- 340 people
- Sponsor
- Progenity, Inc.
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Vanishing Twin, Trisomy 21
Verification of Risk Assignment for Whole Chromosome Using SNP-based NIPT in Vanishing Twin Pregnancies
The purpose of this study is to collect blood samples from women carrying a vanishing twin pregnancy to further develop Natera's non-invasive prenatal screening test to provide information about possible chromosomal conditions…
- Conditions
- Vanishing Twin, Trisomy 21, Trisomy 13, Trisomy 18
- Trial name
- VANISH (NCT05004337)
- Where
- 21 sites in United States
- Volunteers
- 136 people
- Sponsor
- Natera, Inc.
Trisomy 13, Trisomy 18
Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial
This prospective blinded study will assess the diagnostic capability of an informatics enhanced SNP based technology (Parental Support) to identify pregnant women who are carrying a fetus with an aneuploidy from fee floating DNA…
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
- Trial name
- PreNATUS (NCT01545674)
- Testing
- Blood Draw
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Hypothalamic Obesity, Prader-Willi Syndrome
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Trial name
- NCT06239116
- Testing
- Part C: RM-718, Part D: RM-718
- Where
- 7 sites in United States
- Volunteers
- 111 people, healthy volunteers welcome
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Down Syndrome, Edwards Syndrome
Non-Invasive Screening for Fetal Aneuploidy
The purpose of this study is to determine if a laboratory test developed by the Sequenom Center for Molecular Medicine (SCMM) that uses a new marker found in the mother's blood can better identify pregnancies that have a child…
- Trial name
- NCT00847990
- Testing
- Maternal blood screening test for fetal aneuploidy
- Where
- 25 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- Sequenom, Inc.
Pediatric Obstructive Sleep Apnea, Down Syndrome (DS)
Pediatric Down Syndrome Post-Approval Study
The purpose of this Post Approval research study is to assess ongoing safety and effectiveness of the Inspire therapy in adolescents and young adults (age 13-18) with Down syndrome and severe sleep apnea.
- Trial name
- NCT06851338
- Testing
- Hypoglossal Nerve Stimulation
- Where
- 6 sites in United States
- Volunteers
- 60 people
- Sponsor
- Inspire Medical Systems, Inc.