Clinical trials: Development Disorders
Acne Vulgaris
Bioequivalence of Adapalene Topical Gel in Patients With Acne Vulgaris
The objective of this study was to evaluate the efficacy and safety of the test formulation of adapalene gel 0.1% (PLIVA Research \& Development Ltd.) as compared to the already marketed formulation, Differin® (adapalene 0.1%)…
- Trial name
- NCT01182636
- Testing
- Adapalene, Differin®
- Where
- 22 sites in United States, Belize
- Volunteers
- 601 people
- Sponsor
- Teva Pharmaceuticals USA
UC (Urothelial Cancer)
A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Urothelial Carcinoma - (FIGHT-201)
The purpose of this study is to evaluate the overall response rate (ORR) of pemigatinib as a monotherapy in the treatment of metastatic or surgically unresectable urothelial carcinoma harboring FGF/FGFR alterations.
- Trial name
- NCT02872714
- Testing
- pemigatinib
- Where
- 96 sites in United States, Belgium and 9 more
- Volunteers
- 263 people
- Sponsor
- Incyte Corporation
Achondroplasia
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH.
- Trial name
- NCT03875534
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 260 people
- Sponsor
- Ascendis Pharma A/S
Autism
Synthetic Human Secretin in Children With Autism
The purpose of the study is to determine whether multiple doses of secretin are safe and effective in the treatment of children with autism.
- Trial name
- NCT00036244
- Testing
- RG1068 (Synthetic Human Secretin)
- Where
- 15 sites in United States
- Sponsor
- Repligen Corporation
Relapsed Refractory Multiple Myeloma
A Study Comparing AZD0120, a Dual-targeted CAR-T Against B-cell Maturation Antigen (BCMA) and CD19, Versus Standard Regimens in Participants With Relapsed Refractory Multiple Myeloma (DURGA-4)
This is a randomised, multicentre, controlled, open-label, Phase III global study comparing the efficacy and safety of AZD0120 versus standard regimens (DKd [daratumumab, carfilzomib, and dexamethasone], DPd [daratumumab…
- Trial name
- DURGA-4 (NCT07391657)
- Testing
- AZD0120, Daratumumab, Carfilzomib
- Where
- 143 sites in United States, Australia and 13 more
- Volunteers
- 508 people
- Sponsor
- AstraZeneca
Hypothalamic Obesity
Study of Tesomet With Open-label Extension in Subjects With Hypothalamic Obesity (HO)
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects 18 years of age or older, with HO
- Trial name
- NCT05147415
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Circadian Rhythm Sleep Disorders, Non-24 Hour Sleep-Wake Disorder
Study of the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents
Open-label Study to Investigate the Pharmacokinetics and Safety of Tasimelteon in Children and Adolescents.
- Conditions
- Circadian Rhythm Sleep Disorders, Non-24 Hour Sleep-Wake Disorder, Autism Spectrum Disorder, Smith-Magenis Syndrome
- Trial name
- NCT02776215
- Testing
- tasimelteon
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Vanda Pharmaceuticals
Turner Syndrome
The Effect of Growth Hormone in Very Young Girls With Turner Syndrome
This study investigated the effect of growth hormone on the growth of infants and toddlers with Turner syndrome during 2 years of treatment with growth hormone.
- Trial name
- NCT00406926
- Testing
- Somatropin
- Where
- 10 sites in United States
- Volunteers
- 100 people
- Sponsor
- Eli Lilly and Company
Down Syndrome, Edwards Syndrome
Clinical Performance of the MaterniT21 PLUS LDT in Multiple Gestation Pregnancies
This study will evaluate the clinical performance of massively parallel sequencing (MPS) using the MaterniT21 PLUS LDT in the detection of fetal aneuploidy in circulating cfDNA extracted from a maternal blood sample obtained from…
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Turner Syndrome
- Trial name
- NCT02226315
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Sequenom, Inc.
Growth Hormone Disorder, Adult Growth Hormone Deficiency
Long-term Trial on Growth Hormone Deficiency in Adults (GHDA)
This trial is conducted in Japan. To investigate the long-term efficacy as assessed by change in fat mass and safety after 48 weeks of treatment of Growth Hormone in adults with Growth Hormone Deficiency, comparing two different…
- Trial name
- NCT00184743
- Testing
- somatropin
- Where
- Japan
- Volunteers
- 112 people
- Sponsor
- Novo Nordisk A/S
Pregnancy, Down Syndrome
Comparison of Aneuploidy Risk Evaluations
This is a prospective, multi-center observational study designed to compare the test results of the Verinata Health Prenatal Aneuploidy Test to results of conventional prenatal screening for fetal chromosome abnormalities in…
- Conditions
- Pregnancy, Down Syndrome, Edwards Syndrome, Patau Syndrome
- Trial name
- CARE (NCT01663350)
- Where
- 26 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- Verinata Health, Inc.
Bowel Cancer, Bowel Disease
Exploratory Study of Rectal Mucus for Diagnosing Disease
The identification of patients with colorectal cancer is challenging as they present with a variable symptom profile and require invasive tests (colonoscopy) for diagnosis (through histological analysis of biopsies) and…
- Conditions
- Bowel Cancer, Bowel Disease, Genomic, Epigenetic Disorder
- Trial name
- ORI-EGI-02 (NCT04659590)
- Where
- 4 sites in United Kingdom
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Origin Sciences
Developmental Disability, Intellectual Disability
Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
The purpose of this research use only (RUO) study is to detect genomic structural variants (SVs) in human DNA by Optical Genome Mapping (OGM) using the Bionano Genomics Saphyr system.
- Conditions
- Developmental Disability, Intellectual Disability, Autism Spectrum Disorder, Congenital Anomaly
- Trial name
- NCT05295277
- Testing
- Standard of care genetic testing group
- Where
- 8 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Bionano Genomics
Autism Spectrum Disorder
Effectiveness of the Autism Pupil Screen (APS) Detect Pupillometer for Identifying Autism Spectrum Disorder (ASD) in Routine Healthcare
Autism spectrum disorder (ASD) is a developmental condition that can affect communication, social interaction, and behavior. Identifying ASD early helps children and families access support services sooner.
- Trial name
- APS001 (NCT07903675)
- Testing
- Autism Pupil Screen (APS) Detect PLR Assessment #1, Modified Checklist for Autism in Toddlers, Revised with Follow-up (M-CHAT/R-F), Social Responsiveness Scale, Second Edition (SRS-2)
- Where
- 4 sites in United States
- Volunteers
- 330 people, healthy volunteers welcome
- Sponsor
- Schweitzer Engineering Laboratories
Multiple Myeloma
A Study to Learn About the Study Medicine Elranatamab Alone and With Daratumumab in People With Multiple Myeloma Who Have Received Other Treatments
The purpose of this clinical trial is to (1) learn whether the BCMA-CD3 bispecific antibody elranatamab can provide more benefit to people with multiple myeloma compared to a combination therapy including daratumumab…
- Trial name
- MAGNETISMM-5 (NCT05020236)
- Testing
- Elranatamab, Daratumumab, Pomalidomide
- Where
- 237 sites in United States, Argentina and 24 more
- Volunteers
- 893 people
- Sponsor
- Pfizer
Growth Hormone Deficiency
Safety, Tolerability and Efficacy of Weekly TV-1106 in Adults With Growth Hormone Deficiency
The primary objective of this study is to evaluate the clinical effect of TV-1106.
- Trial name
- NCT01811576
- Testing
- TV-1106, Recombinant human growth hormone
- Where
- 23 sites in United States, Czechia and 7 more
- Volunteers
- 52 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Growth Hormone Deficiency (GHD)
Saizen® Solution for Injection Adult Growth Hormone Deficiency (GHD) Immunogenicity Study
To assess the immunogenicity of Saizen® solution for injection in adult subjects with documented Growth Hormone Deficiency (GHD).
- Trial name
- NCT01237340
- Testing
- Saizen®
- Where
- United States
- Volunteers
- 59 people
- Sponsor
- EMD Serono
Growth Hormone Deficiency
Phase 1 Safety Study of ALRN-5281 in Healthy Subjects
The primary objective of this study is to evaluate the safety and tolerability of a single dose of ALRN-5281 administered by subcutaneous injection to healthy adult volunteers.
- Trial name
- NCT01775358
- Testing
- ALRN-5281 0.015 mg/kg, ALRN-5281 0.05 mg/kg, ALRN-5281 0.15 mg/kg
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Aileron Therapeutics, Inc.
Locally Advanced Cholangiocarcinoma, Intrahepatic Cholangiocarcinoma
Safety and Anti-Tumor Activity of TYRA-200 in Advanced Cholangiocarcinoma With Activating FGFR2 Gene Alterations
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of TYRA-200 in cancers with FGFR2 activating gene alterations, including unresectable locally…
- Conditions
- Locally Advanced Cholangiocarcinoma, Intrahepatic Cholangiocarcinoma, Solid Tumor, Metastatic Cholangiocarcinoma
- Trial name
- SURF201 (NCT06160752)
- Testing
- Phase 1 Part A - dose escalation TYRA-200 taken once daily by mouth in 28-day cycles, Phase 1 Part B - dose expansion TYRA-200 taken once daily by mouth in 28-day cycles
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- Tyra Biosciences, Inc
Autism
Allogeneic Umbilical Cord Mesenchymal Stem Cell Therapy for Autism
Allogeneic (not from the subject) human umbilical cord tissue-derived stem cells administered intravenously (IV) in a series of 4 infusions every 3 months over the course of one year is safe and will induce a therapeutic effect…
- Trial name
- NCT02192749
- Testing
- Umbilical cord mesenchymal stem cells
- Where
- Panama
- Volunteers
- 20 people
- Sponsor
- Translational Biosciences
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