Clinical trials: Chromosome Disorders
Philadelphia Chromosome Negative, BCR-ABL1 Positive Chronic Myelogenous Leukemia
ABL001 for the Treatment of Chronic Myeloid Leukemia in Patients Who Are on Therapy With Tyrosine Kinase Inhibitor
This phase II trial studies how well ABL001 works in treating patients with chronic myeloid leukemia who are on therapy with tyrosine kinase inhibitor.
- Trial name
- NCT04216563
- Testing
- Asciminib
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- M.D. Anderson Cancer Center
SGA, Turner Syndrome
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature.
- Conditions
- SGA, Turner Syndrome, Noonan Syndrome, ISS
- Trial name
- REAL 9 (NCT05723835)
- Testing
- Somapacitan
- Where
- 18 sites in United States, Malaysia and 4 more
- Volunteers
- 47 people
- Sponsor
- Novo Nordisk A/S
Down Syndrome
Rivastigmine Study in Adolescents With Down Syndrome
The purpose of this study is to determine if short term use of rivastigmine can improve functional abilities (for example, language, memory, and executive function) in adolescents with Down syndrome.
- Trial name
- DS-Riv (NCT01084135)
- Testing
- Rivastigmine
- Where
- 2 sites in United States
- Volunteers
- 42 people
- Sponsor
- Duke University
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Chronic Myeloid Leukemia, Ph+ Acute Lymphoblastic Leukemia
Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)
The purpose of this study is to determine the efficacy of ponatinib in patients with chronic myeloid leukemia (CML) in chronic phase (CP), accelerated phase (AP) or blast phase (BP) or with philadelphia chromosome-positive (Ph+)…
- Trial name
- PACE (NCT01207440)
- Testing
- Ponatinib
- Where
- 42 sites in Australia, Belgium and 9 more
- Volunteers
- 449 people
- Sponsor
- Ariad Pharmaceuticals
Chronic Myelogenous Leukemia, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
Rollover Study of BMS-354825 in Patients With CML and Ph+ALL
To assess the safety of dasatinib (BMS-354825) in subjects with Imatinib resistant or intolerant chronic myelogenous leukemia (CML) and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL) who are resistant…
- Trial name
- NCT01030718
- Testing
- dasatinib
- Volunteers
- 54 people
- Sponsor
- Bristol-Myers Squibb
Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative
Combination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia
This randomized phase III trial studies combination chemotherapy with blinatumomab to see how well it works compared to induction chemotherapy alone in treating patients with newly diagnosed breakpoint cluster region (BCR)-c-abl…
- Trial name
- NCT02003222
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Biospecimen Collection, Blinatumomab
- Where
- 544 sites in United States, Canada and 2 more
- Volunteers
- 488 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Leukemia
Dasatinib in Imatinib Resistant/Intolerant Chinese CML (Chronic and Advanced Phase) Subjects
The primary objective of this study is to estimate the major cytogenetic response (MCyR) rate to Dasatinib in subjects with CP CML, complete and overall hematologic response (CHR and OHR) rate in subjects with AD CML or Ph+ ALL…
- Trial name
- NCT00529763
- Testing
- Dasatinib
- Where
- 14 sites in China
- Volunteers
- 121 people
- Sponsor
- Bristol-Myers Squibb
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia in Remission
Vorinostat, Tacrolimus, and Methotrexate in Preventing GVHD After Stem Cell Transplant in Patients With Hematological Malignancies
This pilot phase II trial studies how well giving vorinostat, tacrolimus, and methotrexate works in preventing graft-versus-host disease (GVHD) after stem cell transplant in patients with hematological malignancies.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01789255
- Testing
- vorinostat, tacrolimus, cyclosporine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- National Cancer Institute (NCI)
Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Philadelphia Chromosome Positive
Venetoclax, Ponatinib, and Dexamethasone in Participants With Philadelphia Chromosome or BCR-ABL Positive Relapsed or Refractory Acute Lymphoblastic Leukemia or Chronic Myelogenous Leukemia
This phase I/II trial studies the best dose of venetoclax when given together with ponatinib and dexamethasone and to see how well they work in treating participants with Philadelphia chromosome or BCR-ABL positive acute…
- Conditions
- Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Philadelphia Chromosome Positive, Recurrent Acute Lymphoblastic Leukemia, Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Trial name
- NCT03576547
- Testing
- Dexamethasone, Ponatinib Hydrochloride, Rituximab
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- M.D. Anderson Cancer Center
B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
Phase I/II Study of Bosutinib in Combination With Inotuzumab Ozogamicin in CD22-positive PC Positive ALL and CML
This phase I/II trial studies the side effects and best dose of bosutinib when given together with inotuzumab ozogamicin and to see how well it works in treating patients with acute lymphoblastic leukemia or chronic myeloid…
- Conditions
- B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Blasts More Than 5 Percent of Bone Marrow Nucleated Cells, CD22 Positive
- Trial name
- NCT02311998
- Testing
- Bosutinib, Inotuzumab Ozogamicin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- M.D. Anderson Cancer Center
Prader Willi Syndrome
Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants
The Prader-Willi syndrome (PWS) includes severe neonatal hypotonia with impaired suckling leading to failure to thrive in the most severe cases, subsequently followed by an early onset of morbid obesity with hyperphagia and…
- Trial name
- OTBB2 (NCT02205034)
- Testing
- oxytocin
- Where
- France
- Volunteers
- 18 people
- Sponsor
- University Hospital, Toulouse
Turner Syndrome, Hypogonadism
Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism
Estrogen is necessary for feminization during puberty and to decrease bone resorption, the latter critical for the achievement of peak bone mass and normal bone health in the female.
- Conditions
- Turner Syndrome, Hypogonadism, Premature Ovarian Failure
- Trial name
- NCT00837616
- Testing
- 17 B estradiol orally, 17 B estradiol
- Where
- 3 sites in United States, Chile
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Angelman Syndrome
A Trial of Levodopa in Angelman Syndrome
This study is designed to determine whether levodopa will lead to an improvement in the development and tremor in children with Angelman syndrome (AS).
- Trial name
- NCT01281475
- Testing
- Levodopa
- Where
- 7 sites in United States
- Volunteers
- 67 people
- Sponsor
- Wen-Hann Tan
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Leukemia
Imatinib Mesylate and Interferon Alfa in Treating Patients With Chronic Myelogenous Leukemia
RATIONALE: Imatinib mesylate and interferon alfa may interfere with the growth of the cancer cells. Combining imatinib mesylate with interferon alfa may kill more cancer cells.
- Trial name
- NCT00015847
- Testing
- recombinant interferon alfa, imatinib mesylate
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- OHSU Knight Cancer Institute
Down Syndrome, Mild Cognitive Impairment
Nicotine Treatment of Cognitive Decline in Down Syndrome
This study will ascertain whether nicotine is safe and tolerable in DS patients, help with dose-ranging of nicotine in DS, look for evidence of enhancements in cognitive functioning, and establish evidence for biological and…
- Trial name
- NCT01778946
- Testing
- Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
Down Syndrome, Postural Balance
Balance Bike Training in Down Syndrome
Background: Children with Down syndrome (DS) present with hypotonia, impaired balance and poor gross motor proficiency which lead to difficulty learning new motor skills, decreased engagement in physical activity, and limited…
- Trial name
- NCT03677245
- Testing
- Strider Balance Bike
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Mississippi Medical Center
Aneuploidy, Pregnancy Complications
Use of a Novel Computerized Decision Aid for Prenatal Aneuploidy Screening
The investigators aim to assess whether use of a novel, tablet-based computerized decision aid for aneuploidy screening is similar to routine care with a brief genetic counseling visit in improving patient knowledge and…
- Trial name
- DAAS (NCT02991729)
- Testing
- iPad-based decision aid, Genetic counseling appointment
- Where
- United States
- Volunteers
- 197 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Chronic Myelogenous Leukemia
Study of Nilotinib as First Line Treatment in Philadelphia Chromosome Positive(Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
The study was a local multicentric, open-label, non-randomized phase II study of nilotinib as a first line treatment in adult patients with newly-diagnosed Philadelphia chromosome-positive (Ph+) and chronic phase myeloid leukemia…
- Trial name
- NCT01274351
- Testing
- Nilotinib
- Where
- 13 sites in Turkey (Türkiye)
- Volunteers
- 112 people
- Sponsor
- Novartis Pharmaceuticals