Clinical trials: Chromosome Disorders
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
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
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
Hyperphagia, Prader-Willi Syndrome
Pilot Study of Startle-response Test to Assess Transcranial Direct Current Stimulation-induced Modulation of Hyperphagia in Prader-Willi Syndrome
The purpose of this study is to determine the effects of transcranial direct current stimulation (tDCS) as it modifies hyperphagia in obese subjects, non-obese subjects, and subjects with Prader-Willi syndrome (PWS).
- Trial name
- NCT01863017
- Testing
- tDCS
- Where
- 3 sites in United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Myelodysplastic Syndrome, MDS
A Dose Range Finding Study of Lenalidomide in Non-5q Chromosome Deletion in Low and Intermediate Risk Myelodysplastic Syndrome (MDS) Patients
Revlimid® (Lenalidomide) is indicated for a type of blood cancer, myelodysplastic syndrome (MDS), at 10mg for a specific type of myelodysplastic syndrome with a genetic abnormality called "deletion 5q" in Low and Intermediate-1…
- Conditions
- Myelodysplastic Syndrome, MDS, Low to Intermediate-1 MDS, Non-deletion 5q
- Trial name
- NCT00699842
- Testing
- Lenalidomide
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Myelodysplastic Syndromes
Lenalidomide Safety/Efficacy in Myelodysplastic Syndromes (MDS) Associated With a Deletion (Del)(5q) Cytogenetic Abnormality
This study is a multicenter, single-arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion-dependent subjects with low- or intermediate-1-risk Myelodysplastic Syndromes (MDS)…
- Trial name
- NCT00065156
- Testing
- lenalidomide
- Where
- 32 sites in United States, Germany
- Volunteers
- 148 people
- Sponsor
- Celgene
Burden, Caregiver
Social Support and Stress Reduction for Caregivers of Young Adults With IDD
The objective of this study is to evaluate the feasibility and stress reduction impacts of a yoga and support group intervention on caregivers of individuals with intellectual and developmental disabilities.
- Trial name
- CareWell (NCT05938998)
- Testing
- Yoga; Support Group
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Klinefelter Syndrome, XXY Syndrome
Study of Psychological and Motor Effects of Testosterone in Adolescents With XXY/Klinefelter Syndrome
The purpose of this study is to determine if testosterone replacement therapy leads to changes in psychological factors and/or motor skills in adolescent males with 47,XXY (also called Klinefelter syndrome).
- Trial name
- TestoXXY/KS (NCT01585831)
- Testing
- Testosterone gel 1%
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- University of Colorado, Denver
Acute Leukemias of Ambiguous Lineage, B-cell Adult Acute Lymphoblastic Leukemia
S1312, Inotuzumab Ozogamicin and Combination Chemotherapy in Treating Patients With Relapsed or Refractory Acute Leukemia
This phase I trial studies the side effects and best dose of inotuzumab ozogamicin when given together with combination chemotherapy in treating patients with relapsed or refractory acute leukemia.
- Conditions
- Acute Leukemias of Ambiguous Lineage, B-cell Adult Acute Lymphoblastic Leukemia, Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic Leukemia, Recurrent Adult Acute Lymphoblastic Leukemia
- Trial name
- NCT01925131
- Testing
- cyclophosphamide, vincristine sulfate, prednisone
- Where
- 5 sites in United States
- Volunteers
- 50 people
- Sponsor
- SWOG Cancer Research Network
Fragile X Syndrome
ERG/5-HTP in Fragile X Syndrome (FXS)
The purpose of this study is to assess the safety, tolerability and efficacy of Ergoloid mesylates (EM) and 5-hydroxytryptophan (5-HTP) and the combination (EM + 5-HTP) compared to placebo in males aged 18-45 years old with…
- Trial name
- NCT05030129
- Testing
- 5-Hydroxytryptophan, Ergoloid Mesylates
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Elizabeth Berry-Kravis
CML, Philadelphia Chromosome Positive (Ph+)
Study of Nilotinib in Ph+ CML-CP Patients With Low Imatinib Trough Plasma Concentrations
This study is to determine the number of European Leukemia Network (ELN)guideline defined treatment failure events from time of study entry in CML-CP patients with low imatinib trough concentrations treated with nilotinib.
- Conditions
- CML, Philadelphia Chromosome Positive (Ph+), Chronic Myelogenous Leukemia Chronic Phase(CML-CP) Patients With Low Imatinib Trough Levels
- Trial name
- MACS1148 (NCT01131325)
- Testing
- nilotinib
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Novartis Pharmaceuticals
Leukemia
Vaccine Therapy in Treating Patients With Philadelphia Chromosome-Positive Chronic Myelogenous Leukemia
RATIONALE: Vaccines made from peptides may help the body build an effective immune response to kill cancer cells. Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or…
- Trial name
- CML0206 (NCT00466726)
- Testing
- bcr-abl p210-b3a2 breakpoint-derived multipeptide vaccine, sargramostim
- Where
- 17 sites in Italy
- Volunteers
- 57 people
- Sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
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
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
Philadelphia Chromosome Positive Adult Precursor Acute Lymphoblastic Leukemia, Recurrent Adult Acute Lymphoblastic Leukemia
Entinostat And Imatinib Mesylate In Treating Patients With Relapsed or Refractory Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
This phase I/II trial is studying the side effects and best dose of entinostat when given together with imatinib mesylate and to see how well it works in treating patients with relapsed or refractory Philadelphia…
- Trial name
- NCT01383447
- Testing
- entinostat, imatinib mesylate, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Cancer Institute (NCI)
Fragile X Syndrome
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have…
- Trial name
- NCT01348087
- Testing
- AFQ056
- Where
- 28 sites in United States, Australia and 8 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Chronic Myeloid Leukemia, Philadelphia-Positive Myeloid Leukemia
Dasatinib (BMS-354835) Versus Imatinib Mesylate in Subjects With Chronic Myeloid Leukemia
The primary purpose of this study is to estimate the major cytogenetic response rates of BMS-354825 and imatinib (800 mg/d) in subjects with chronic phase, Philadelphia chromosome positive, chronic myeloid leukemia (PH+ CML) with…
- Trial name
- NCT00103844
- Testing
- Dasatinib, Imatinib
- Where
- 131 sites in United States, Argentina and 30 more
- Volunteers
- 150 people
- Sponsor
- Bristol-Myers Squibb
Fragile X Syndrome
Trial of Minocycline to Treat Children With Fragile X Syndrome
This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant.
- Trial name
- NCT01053156
- Testing
- minocycline hydrochloride
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- University of California, Davis
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda