Clinical trials: Chromosome Disorders
Down Syndrome, Health, Subjective
Developing a Down Syndrome Health Instrument
Although over 200,000 individuals with DS live in the United States, studies to date have focused on outcomes apart from health.
- Trial name
- NCT04631237
- Testing
- Observational, no intervention
- Where
- United States
- Volunteers
- 758 people
- Sponsor
- Massachusetts General Hospital
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Fragile X Syndrome, Genetic Diseases
Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome
The purpose of the study is to test the efficacy of a 20 week multi-modal treatment comprised of lovastatin or placebo, and the Parent-implemented Language Intervention (PILI) in children with fragile X syndrome (FXS).
- Trial name
- NCT02642653
- Testing
- Lovastatin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Philadelphia Chromosome Negative, Recurrent B Acute Lymphoblastic Leukemia
Low-Intensity Chemotherapy and Blinatumomab in Treating Patients With Philadelphia Chromosome Negative Relapsed or Refractory Acute Lymphoblastic Leukemia
This phase II trial studies how well low-intensity chemotherapy and blinatumomab work in treating patients with Philadelphia chromosome negative acute lymphoblastic leukemia that has come back or does not respond to treatment.
- Conditions
- Philadelphia Chromosome Negative, Recurrent B Acute Lymphoblastic Leukemia, Refractory B Acute Lymphoblastic Leukemia
- Trial name
- NCT03518112
- Testing
- Blinatumomab, Cyclophosphamide, Cytarabine
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- M.D. Anderson Cancer Center
Klinefelter Syndrome
Body Composition in Infants With Klinefelter Syndrome and Effects of Testosterone Treatment
This research study in infant males with Klinefelter syndrome (47,XXY) will learn more about body composition (muscle and fat) and male hormones and look at the effect of testosterone shots on body composition.
- Trial name
- NCT02408445
- Testing
- testosterone cypionate 200mg/ml
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Colorado, Denver
Chronic Lymphocytic Leukemia (CLL)
A Study Evaluating the Impact of Venetoclax on the Quality of Life for Subjects With Relapsed (Your Cancer Has Come Back) or Refractory (no Response to Previous Cancer Treatments) Chronic Lymphocytic Leukemia (CLL) While Receiving Venetoclax Monotherapy (a Single Agent).
The purpose of this open-label, single-arm study was to evaluate the impact of venetoclax on the quality of life of participants including those with with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL; a type of…
- Trial name
- VENICE II (NCT02980731)
- Testing
- Venetoclax
- Where
- 33 sites in Argentina, Australia and 8 more
- Volunteers
- 210 people
- Sponsor
- AbbVie
Myeloproliferative Disorders
Pegasys® in Patients With Myeloproliferative Diseases
The goal of this clinical research study is to see if Pegasys (IFN-alpha2a) can help to control the disease in patients with ET, PV, AMM/MF, and Ph-negative CML. The safety of this treatment will also be studied.
- Trial name
- NCT00452023
- Testing
- IFN-alpha2a
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- M.D. Anderson Cancer Center
Leukemia
Pegaspargase and Combination Chemotherapy in Treating Patients With Newly Diagnosed Acute Lymphoblastic Leukemia
RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT01005914
- Testing
- cyclophosphamide, cytarabine, dexamethasone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- OHSU Knight Cancer Institute
Leukemia
Long-term Safety of Dasatinib in Patients With Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
This study assesses the long-term safety and tolerability of dasatinib administered to patients with chronic myelogenous leukemia or Philadelphia chromosome positive acute lymphoblastic leukemia and experienced clinical benefit…
- Trial name
- START rollover (NCT00982488)
- Testing
- Dasatinib, Imatinib
- Where
- 72 sites in United States, Argentina and 22 more
- Volunteers
- 238 people
- Sponsor
- Bristol-Myers Squibb
Myelogenous Leukemia, Chronic
A Study of Imatinib Versus Nilotinib in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
In this study, the efficacy and safety of two nilotinib doses, 300 mg twice daily and 400 mg twice daily, were compared with imatinib 400 mg once daily in newly diagnosed patients with Philadelphia chromosome-positive (Ph+)…
- Trial name
- ENESTnd (NCT00471497)
- Testing
- nilotinib, imatinib
- Where
- 213 sites in United States, Argentina and 33 more
- Volunteers
- 846 people
- Sponsor
- Novartis Pharmaceuticals
Down Syndrome
Gait in Children With Down Syndrome While Wearing Orthoses
On average, Down syndrome (DS) occurs once in every 700 live births and results in life-long disability and increased risk for comorbidities.1 Individuals with DS are also susceptible to secondary physical impairments and…
- Trial name
- NCT01598350
- Testing
- Supramalleolar Orthoses (Cascade)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Puget Sound
Philadelphia Chromosome Positive, Chronic Myelogenous Leukemia in Chronic Phase
Study to Evaluate Nilotinib in Chronic Myelogenous Leukemia (CML) Patients With SubOptimal Response
To evaluate the major molecular response (MMR) rate at 12 months of nilotinib treatment on study in patients with Philadelphia Chromosome Positive (Ph+) chronic myelogenous leukemia in chronic phase (CML-CP) who have a suboptimal…
- Trial name
- MACS0911 (NCT01043874)
- Testing
- Nilotinib
- Where
- 20 sites in Japan
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma
Risk-Adapted Chemotherapy in Treating Younger Patients With Newly Diagnosed Standard-Risk Acute Lymphoblastic Leukemia or Localized B-Lineage Lymphoblastic Lymphoma
This partially randomized phase III trial studies the side effects of different combinations of risk-adapted chemotherapy regimens and how well they work in treating younger patients with newly diagnosed standard-risk acute…
- Conditions
- Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma
- Trial name
- NCT01190930
- Testing
- Cyclophosphamide, Cytarabine, Dexamethasone
- Where
- 243 sites in United States, Australia and 5 more
- Volunteers
- 9,350 people
- Sponsor
- Children's Oncology Group
Myeloid Leukemia, Chronic
A Phase III Study of Dasatinib vs Imatinib in Patients With Newly Diagnosed Chronic Phase Chronic Myeloid Leukemia
The purpose of this clinical research study is to compare the confirmed complete cytogenetic response of dasatinib with that of imatinib within 12 months after randomization in patients with newly diagnosed chronic-phase…
- Trial name
- DASISION (NCT00481247)
- Testing
- Dasatinib, Imatinib
- Where
- 109 sites in United States, Argentina and 25 more
- Volunteers
- 547 people
- Sponsor
- Bristol-Myers Squibb
Chronic Myeloid Leukemia
Safety and Efficacy of Nilotinib vs. Imatinib in the Treatment of Newly Diagnosed Chinese Ph+ CML-CP Patients
The study will compare the efficacy and safety of Nilotinib versus Imatinib in newly diagnosed Chinese patients with CML-CP.
- Trial name
- NCT01275196
- Testing
- Nilotinib, Imatinib
- Where
- 12 sites in China
- Volunteers
- 267 people
- Sponsor
- Novartis Pharmaceuticals
Hypothyroidism, Trisomy 21
Use of Liquid Stable Levothyroxine in Trisomy 21 Pediatric Patients
Children with levothyroxine (T21) have developmental delay and other functional gastrointestinal (GI) issues that may negatively affect L-T4 tolerability and absorption.
- Trial name
- NCT04747275
- Testing
- Liquid stable levothyroxine (L-T4) Tirosint-SOL, Oral tablet levothyroxine (L-T4)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Mercy Hospital Kansas City
Prader-Willi Syndrome
Intranasal Oxytocin for Infants With Prader-Willi Syndrome
The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional…
- Trial name
- NCT03245762
- Testing
- Oxytocin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Florida
Chronic Myeloid Leukemia
Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients
This study will further investigate the safety and efficacy of nilotinib in newly diagnosed chronic myeloid leukemia patients in the chronic phase
- Trial name
- ENESTxtnd (NCT01254188)
- Testing
- Nilotinib
- Where
- 100 sites in Algeria, Argentina and 17 more
- Volunteers
- 421 people
- Sponsor
- Novartis Pharmaceuticals
Angelman Syndrome, Nervous System Diseases
Dietary Supplements for the Treatment of Angelman Syndrome
Angelman syndrome (AS) is a complex genetic disorder that affects the nervous system. The purpose of this study is to determine the effectiveness of certain dietary supplements in treating the symptoms of AS.
- Trial name
- NCT00348933
- Testing
- Betaine, Creatine, Metafolin
- Where
- 4 sites in United States
- Volunteers
- 90 people
- Sponsor
- University of California, San Diego