Clinical trials: Chromosome Disorders
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
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
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
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
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
Leukemia
Chronic Myelogenous Leukemia or Philadelphia Chromosome Positive Acute Lymphoblastic Leukemic Study
To determine the long term safety and tolerability of dasatinib exposure in subjects previously treated in CA180-002.
- Trial name
- NCT00978731
- Testing
- Dasatinib
- Volunteers
- 46 people
- Sponsor
- Bristol-Myers Squibb
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
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
Philadelphia Positive Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia
An Extension Study to Determine the Safety and Anti-Leukemic Effects of Imatinib Mesylate in Adult Participants With Ph+ Leukemia
The objectives of Part 1 of the study were: To determine the rate of hematologic response (HR) lasting ≥4 weeks in participants with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the accelerated phase…
- Conditions
- Philadelphia Positive Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT00171249
- Testing
- STI571 400 mg, STI571 600 mg
- Where
- 12 sites in United States, France and 3 more
- Volunteers
- 293 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myeloid Leukemia
Study Assessing Deep Molecular Response in Adult Patients With CML in Chronic Phase Treated With Nilotinib Firstline.
The main purpose of this study was to evaluate the rate of deep molecular response (MR4.5) after 24 months of therapy with nilotinib in newly diagnosed patients with chronic phase chronic myeloid leukemia (CML) using EUTOS…
- Trial name
- NILOdeepR (NCT02546674)
- Testing
- Nilotinib
- Where
- 70 sites in Germany
- Volunteers
- 171 people
- Sponsor
- Novartis 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
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
Nilotinib vs Imatinib in Adult Patients With Philadelphia (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
In this study, the efficacy and safety of nilotinib 400 mg twice daily, will be compared with imatinib 400 mg twice daily in patients with a suboptimal response to imatinib for their Philadelphia chromosome-positive (Ph+) Chronic…
- Trial name
- ENEST (NCT00519090)
- Testing
- Imatinib, Nilotinib (AMN107)
- Where
- 80 sites in United States, Australia and 8 more
- Volunteers
- 6 people
- Sponsor
- Novartis Pharmaceuticals
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
22q11 Deletion Syndrome
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
This is a Phase 2, randomized, placebo-controlled crossover trial to assess the safety and efficacy of NB-001 in children and adolescents with 22q11DS that manifest commonly associated neuropsychiatric symptoms.
- Trial name
- NCT05290493
- Testing
- NB-001
- Where
- 4 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Nobias Therapeutics, Inc.
Myelodysplastic Syndromes
Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality
The purpose of this study was to compare 2 doses (10 mg and 5 mg) of lenalidomide to that of placebo in subjects with red blood cell (RBC) transfusion-dependent low- or intermediate-1-risk IPSS MDS associated with a deletion…
- Trial name
- NCT00179621
- Testing
- Lenalidomide 5 mg, Lenalidomide 10 mg
- Where
- 38 sites in Belgium, France and 7 more
- Volunteers
- 205 people
- Sponsor
- Celgene Corporation
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.
Angelman Syndrome
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
- Trial name
- NCT02996305
- Testing
- OV101 Regimen 1, OV101 regimen 2
- Where
- 13 sites in United States, Israel
- Volunteers
- 88 people
- Sponsor
- Healx AI
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.