Is NCT01245179 recruiting in Augusta, GA?

Yes, NCT01245179 is currently recruiting participants at its Augusta, GA location. This sickle cell disease clinical trial is actively enrolling qualified participants.

How do I join the sickle cell disease clinical trial in Augusta?

To join this clinical trial in Augusta, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Augusta clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Sickle Cell Disease Clinical Trial in Augusta, GA

About This Sickle Cell Disease Study in Augusta

The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.

Sponsor: Abdullah Kutlar

Who Can Participate

Inclusion Criteria

Male or female patients ages ≥ 18 years

Confirmed diagnosis of homozygous SS or S-β0Thalassemia

Intolerance to hydroxyurea therapy, refusal of hydroxyurea therapy, or failure to respond (refractoriness) to hydroxyurea therapy, either clinically or hematologically.

Clinically significant sickle cell disease as defined by:

At least two hospitalizations over the past twelve months for any complication of sickle cell disease; or

At least three pain crises over the past twelve months that last four or more hours and require a visit to a medical facility for treatment with oral or parenteral narcotics; or

History of recurrent leg ulcers; or

History of Acute Chest Syndrome within the past five years; or

History of priapism requiring medical intervention within the past two years; or

History of stroke (but not currently on a chronic blood transfusion regimen).

Ability to provide written informed consent obtained prior to participation in the study and any related procedures being performed.

Clinically euthyroid. Note: Patients are permitted to receive thyroid hormone supplements to treat underlying hypothyroidism.

Exclusion Criteria

Use of agents that can induce Hb F within 60 days of Day 1 (i.e. hydroxyurea, butyrates, decitabine, 5-azacytidine, IMiDs®, or erythropoietin). Prior use of HDACi, including panobinostat, is not an exclusion criterion if discontinued \> 60 days.

Patients who have had a vaso-occlusive crisis within the past 2 weeks that required treatment with parenteral medication.

Impairment of GI function or GI disease that may significantly alter the absorption of panobinostat (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)

Patients on a chronic transfusion regimen, or any patient who has Hb A% \> 20% from a recent transfusion

Any of the following laboratory abnormalities derived from the screening visit:

Absolute neutrophil count (ANC) \< 1.5 x 109/L

Hemoglobin \< 6 g/dl

Platelets \< 100x 109/L

Serum creatinine \>1.5 x Upper limits of normal (ULN)

AST and ALT \> 2.5 x ULN

Serum total bilirubin \> 10 mg/dL

Serum direct bilirubin \> 1 mg/dL

Albumin \<3.0 g/dl

Serum potassium \< Lower limits of normal (LLN)

Total serum calcium \[corrected for serum albumin\] or ionized calcium \<LLN

Serum magnesium \< LLN

Serum phosphorus \< LLN

Known impaired cardiac function or clinically significant cardiac diseases, including any one of the following:

Left ventricular ejection fraction (LVEF) \< lower limit of the institutional normal as determined by screening echocardiogram

Complete left bundle branch block

Obligate use of a cardiac pacemaker

Congenital long QT syndrome

History or presence of ventricular tachyarrhythmia

Presence of unstable atrial fibrillation (ventricular response \> 100 bpm). Patients with stable atrial fibrillation are eligible, provided they do not meet any of the other cardiac exclusion criteria.

Clinically significant resting bradycardia (\< 50 bpm)

QTc \> 470 msec on screening ECG

Right bundle branch block + left anterior hemiblock (bifasicular block)

Angina pectoris 3 months prior to starting study drug

Acute MI 3 months prior to starting study drug

Other clinically significant heart disease (e.g., CHF, uncontrolled hypertension, history of labile hypertension, or history of poor compliance with an antihypertensive regimen)

Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes, active or uncontrolled infection, chronic obstructive or chronic restrictive pulmonary disease) that could cause unacceptable safety risks or compromise compliance with the protocol

Patients who are currently receiving treatment with any study drug or have been on any study medications within the past 60 days.

Patients who have undergone major surgery 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.

Women of child-bearing potential (WCBP) who are pregnant or breast feeding or who do not agree to use two methods of birth control, including a barrier method, if they are sexually active. WCBP, defined as sexually mature women who have not undergone a hysterectomy or who have not been naturally postmenopausal for at least 12 consecutive months (i.e., who has had menses any time in the preceding 12 consecutive months), must have a negative serum pregnancy test at screening and negative urine pregnancy test within 72 hours prior to starting study treatment. In addition, all sexually active WCBP must agree to use double method of contraception (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) during the study and 3 months after the end of treatment. One of these methods of contraception must be a barrier method.

Male patients whose sexual partners are WCBP not using a double method of contraception during and 3 months after the end of treatment. Males must agree to use a condom during any sexual contact with WCBP during study drug treatment, during dose interruptions, and for 3 months after the end of treatment.

Known diagnosis of HIV infection, Hepatitis B; or acute/chronic, active Hepatitis C

Patients with a prior malignancy with in the last 5 years (except for basal or squamous cell carcinoma, or in situ cancer of the cervix)

Patients with any significant history of non-compliance to medical regimens or unwilling or unable to comply with the instructions given to him/her by the study staff.

Patients who are currently receiving treatment with certain prohibited medications and cannot either discontinue this treatment or switch to a different medication prior to study enrollment.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Augusta?

Yes, this clinical trial (NCT01245179) has an active research site in Augusta, GA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Sickle Cell Disease Treatment Options in Augusta, GA

If you're searching for sickle cell disease treatment options in Augusta, GA, this clinical trial (NCT01245179) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Augusta research site is actively enrolling participants for this phase 1 clinical trial. You'll receive care from experienced sickle cell disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all sickle cell disease clinical trials near you to find additional studies recruiting in your area.

Sickle Cell Disease Clinical Trial in Augusta, GA

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