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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT03764215
Date of registration: 27/11/2018
Prospective Registration: No
Primary sponsor: Georgetown University
Public title: Nilotinib in Huntington's Disease Tasigna HD
Scientific title: An Open Label, Phase Ib Study to Evaluate the Impact of Low Doses of Nilotinib Treatment on Safety, Tolerability and Biomarkers in Huntington's Disease
Date of first enrolment: November 15, 2018
Target sample size: 10
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03764215
Study type:  Interventional
Study design:  Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1
Countries of recruitment
United States
Contacts
Name:     Hope Heller
Address: 
Telephone: 202-687-1366
Email: hope.heller@gunet.georgetown.edu
Affiliation: 
Name:     Hope Heller
Address: 
Telephone: 202-687-1366
Email: hope.heller@gunet.georgetown.edu
Affiliation: 
Key inclusion & exclusion criteria

Inclusion Criteria:

- Written informed consent

- Capable of providing informed consent and complying with study procedures. Subjects
who are unable to provide consent may use a Legally Authorized Representative (LAR).

- Patients between the age of 25-90 years, medically stable

- Clinical diagnosis of HD with either a confirmed family history or positive CAG repeat
(CAG=35)

- MoCA = 22

- Able to perform the TMT-B in =240 seconds

- Total Functional Capacity 7-12

- Stable concomitant medical and/or psychiatric illnesses, in the judgement of the PI.

- QTc interval 350-460 ms, inclusive

- Participants must be willing to undergo LP at baseline and 3 months after treatment

Exclusion Criteria:

- Patients with hypokalemia, hypomagnesaemia, or long QT syndrome- QTc=461 ms

- Concomitant drugs known to prolong the QTc interval and history of any cardiovascular
disease, including myocardial infarction or cardiac failure, angina, arrhythmia

- History or presence of cardiac conditions including:

1. Cardiovascular or cerebrovascular event (e.g. myocardial infarction, unstable
angina, or stroke)

2. Congestive heart failure

3. First, second- or third-degree atrioventricular block, sick sinus syndrome, or
other serious cardiac rhythm disturbances

4. Any history of Torsade de Pointes

- Treatment with any of the following drugs at the time of screening or the preceding 30
days, and/or planned use over the course of the trial:

1. Treatment with Class IA or III antiarrhythmic drugs (e.g. quinidine)

2. Treatment with QT prolonging drugs (www.crediblemeds.org)- excluding Selective
Serotonin Reuptake Inhibitors (SSRIs) (e.g. Citalopram, Escitalopram, Paroxetine,
Sertraline, Duloxetine, Trazodone, etc.)

3. Strong CYP3A4 inhibitors (including grapefruit juice). The concomitant use of
strong CYP3A4 inhibitors (e.g., ketoconazole, itraconazole, clarithromycin,
atazanavir, indinavir, nefazodone, nelfinavir, ritonavir, saquinavir,
telithromycin, voriconazole) must be avoided. Grapefruit products may also
increase serum concentrations of Nilotinib. Should treatment with any of these
agents be required, therapy with Nilotinib should be interrupted.

4. Anticoagulants, including Coumadin (warfarin), heparin, enoxaparin, daltiparin,
xarelto, etc.

5. St. John's Wort and the concomitant use of strong other CYP3A4 inducers (e.g.,
dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, rifapentine,
phenobarbital) must be avoided since these agents may reduce the concentration of
Nilotinib.

- Abnormal liver function defined as AST and/or ALT > 100% the upper limit of the normal

- Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of
normal

- History of HIV, clinically significant chronic hepatitis, or other active infection

- Females must not be lactating, pregnant or with possible pregnancy

- Medical history of liver or pancreatic disease

- Clinical signs indicating syndromes other than idiopathic PD, including corticobasal
degeneration, supranuclear gaze palsy, multiple system atrophy, chronic traumatic
encephalopathy, signs of frontal dementia, history of stroke, head injury or
encephalitis, cerebellar signs, early severe autonomic involvement, Babinski sign

.Current evidence or history in past two years of epilepsy, focal brain lesion, head
injury with loss of consciousness or DSM-IV criteria for any active major psychiatric
disorder including psychosis, major depression, bipolar disorder, alcohol or substance
abuse

- Evidence of any significant clinical disorder or laboratory finding that renders the
participant unsuitable for receiving an investigational drug including clinically
significant or unstable hematologic, hepatic, cardiovascular, pulmonary,
gastrointestinal, endocrine, metabolic, renal or other systemic disease or laboratory
abnormality

- Active neoplastic disease, history of cancer five years prior to screening, including
breast cancer (history of skin melanoma or stable prostate cancer are not
exclusionary)

- Contraindications to LP: prior lumbosacral spine surgery, severe degenerative joint
disease or deformity of the spine, platelets < 100,000, use of Coumadin/warfarin, or
history of a bleeding disorder

- Must not be on any immunosuppressant medications (e.g. IVig)

- Must not be enrolled as an active participant in another clinical study



Age minimum: 25 Years
Age maximum: 90 Years
Gender: All
Health Condition(s) or Problem(s) studied
Huntington Disease
Intervention(s)
Drug: Nilotinib 150 MG
Primary Outcome(s)
CSF levels of biomarkers linked to Disease symptoms Chorea and behavioral symptoms [Time Frame: 3 months]
Number of participants experiencing any Adverse events and Serious Adverse Events [Time Frame: 3 months]
Number of participants tolerating the drug by the ability of remaining on treatment [Time Frame: 3 months]
Secondary Outcome(s)
Secondary ID(s)
2017-0440
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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