Clinical trials: Chromosome Disorders
Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia
Blinatumomab and Combination Chemotherapy or Dasatinib, Prednisone, and Blinatumomab in Treating Older Patients With Acute Lymphoblastic Leukemia
This phase II trial studies the side effects and how well blinatumomab and combination chemotherapy or dasatinib, prednisone, and blinatumomab work in treating older patients with acute lymphoblastic leukemia.
- Conditions
- Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia, B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Recurrent Acute Lymphoblastic Leukemia
- Trial name
- NCT02143414
- Testing
- Biopsy Procedure, Biospecimen Collection, Blinatumomab
- Where
- 195 sites in United States
- Volunteers
- 53 people
- Sponsor
- National Cancer Institute (NCI)
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Williams Beuren Syndrome
Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
The Williams-Beuren syndrome (WBS) is a sporadic congenital disorder characterized by a multisystem developmental impairment. This syndrome is caused by a microdeletion in chromosome 7q11.23 that encompasses loss of the elastin…
- Trial name
- Williams (NCT00876200)
- Testing
- Minoxidil
- Where
- 18 sites in France
- Volunteers
- 21 people
- Sponsor
- Hospices Civils de Lyon
Myeloproliferative Neoplasm
Intra-patient Dose Escalation Study to Investigate Safety and Feasibility of Vactosertib in Treating Anemic MPN Patients
This study assesses the potential of using a TGFβ receptor inhibitor for the treatment of anemic patients with myeloproliferative neoplasms.
- Trial name
- NCT04103645
- Testing
- Vactosertib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Weill Medical College of Cornell University
Acute Lymphoblastic Leukemia, BCR-ABL1 Fusion Protein Expression
Combination Chemotherapy and Dasatinib in Treating Participants With Philadelphia Positive or BCR-ABL Positive Acute Lymphoblastic Leukemia.
This phase II trial studies how well combination chemotherapy and dasatinib works in treating participants with Philadelphia-positive or B-cell receptor-ABL positive acute lymphoblastic leukemia.
- Conditions
- Acute Lymphoblastic Leukemia, BCR-ABL1 Fusion Protein Expression, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Philadelphia Chromosome Positive
- Trial name
- NCT00390793
- Testing
- Cyclophosphamide, Cytarabine, Dasatinib
- Where
- United States
- Volunteers
- 107 people
- Sponsor
- M.D. Anderson Cancer Center
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy
This phase II trial studies how well sirolimus and azacitidine works in treating patients with high-risk myelodysplastic syndrome or recurrent acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT01869114
- Testing
- Sirolimus, Azacitidine
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
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
Down Syndrome
An Online Health Tool About Down Syndrome: Down Syndrome Clinic to You
The investigators conducted a national two-arm, randomized controlled trial of caregivers of individuals with DS to assess the efficacy of DSC2U in assuring adherence to evidence-based guidelines.
- Trial name
- DSC2U (NCT04227197)
- Testing
- Down Syndrome Clinic to You (DSC2U)
- Where
- United States
- Volunteers
- 281 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Obstructive Sleep Apnea, Down Syndrome
Medications for Obstructive Sleep Apnea In Children With Down Syndrome
This is a randomized, double blind, cross-over study of the combination of atomoxetine and oxybutynin (ato-oxy) in children with DS and OSA documented by polysomnography (PSG).
- Trial name
- MOSAIC (NCT04115878)
- Testing
- Atomoxetine and oxybutynin (ato-oxy)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Arizona
Leukemia
Phase 2 Dasatinib Combo With Smoothened (SMO) Antagonist (BMS-833923)
The purpose of the study is to compare response rates in newly diagnosed Chronic Phase (CP) CML subjects treated with dasatinib plus BMS-833923 versus dasatinib alone.
- Trial name
- NCT01357655
- Testing
- Dasatinib, BMS-833923
- Where
- 24 sites in United States, Argentina and 6 more
- Volunteers
- 70 people
- Sponsor
- Bristol-Myers Squibb
Turner's Syndrome
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years.
- Trial name
- NCT01066052
- Testing
- r-hGH
- Where
- France
- Volunteers
- 115 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
22Q11 Deletion Syndrome
Mindfulness Program for Adolescents With 22q11DS
The goal of this study is to evaluate the effectiveness of the Aware Program, an online mindfulness education program, with adolescents with 22q11DS and their parents.
- Trial name
- NCT05849441
- Testing
- Aware Program
- Where
- United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Innovation Research & Training
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)
Aggressive Non-Hodgkin Lymphoma, Blasts Under 5 Percent of Bone Marrow Nucleated Cells
Donor Atorvastatin Treatment in Preventing Severe Acute GVHD After Nonmyeloablative Peripheral Blood Stem Cell Transplant in Patients With Hematological Malignancies
This phase II trial studies how well donor atorvastatin treatment works in preventing severe graft-versus-host disease (GVHD) after nonmyeloablative peripheral blood stem cell (PBSC) transplant in patients with hematological…
- Conditions
- Aggressive Non-Hodgkin Lymphoma, Blasts Under 5 Percent of Bone Marrow Nucleated Cells, Chronic Lymphocytic Leukemia, Loss of Chromosome 17p
- Trial name
- NCT01527045
- Testing
- Atorvastatin Calcium, Cyclosporine, Fludarabine Phosphate
- Where
- 2 sites in United States
- Volunteers
- 47 people
- Sponsor
- Fred Hutchinson Cancer Center
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
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
Prader-Willi Syndrome
Growth Hormone Use in Adults With Prader-Willi Syndrome
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation…
- Trial name
- NCT00444964
- Testing
- Nutropin AQ
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Mercy Hospital Kansas City
Fragile x Syndrome
Ganaxolone Treatment in Children With Fragile X Syndrome
This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS).
- Trial name
- NCT01725152
- Testing
- Ganaxolone
- Where
- 2 sites in United States, Belgium
- Volunteers
- 59 people
- Sponsor
- Marinus Pharmaceuticals
Down Syndrome
Blood Flow Regulation in Individuals With Down Syndrome - Training Study
Work capacity is an important predictor of declining health or physical function, and of mortality, and is commonly measured as peak oxygen consumption.
- Trial name
- NCT04854122
- Testing
- Combined exercise intervention
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Nevada, Las Vegas
Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia
A Pharmacokinetic (PK) Study of Nilotinib in Pediatric Patients With Philadelphia Chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) or Acute Lymphoblastic Leukemia (ALL)
This study will assess the pharmacokinetics of nilotinib in Ph+ CML pediatric patients that are newly diagnosed or resistant or intolerant to imatinib or dasatinib or refractory or relapsed Ph+ ALL compared to the adult…
- Trial name
- NCT01077544
- Testing
- Nilotinib
- Where
- 12 sites in France, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- Novartis Pharmaceuticals