A study to investigate the effects Aes-103 in patients with Sickle Cell Disease

Conditions: Sickle Cell Disease
MedDRA version: 18.0Level: LLTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2013-001534-18-GB

Lead Sponsor: AesRx

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 100

Inclusion Criteria

1. Be male or female, aged 18-60 years old, inclusive
2. Have SCD (hemoglobin SS or S-beta-zero) without hospitalization for any reason in the 14 days before enrollment. Subjects are allowed concomitant usage of HU and other scheduled prescription drugs if the dosage is stable for the 2 months before screening and is at a dosage that does not exceed the product’s labeling. These scheduled prescription medications will be continued during the study. All other medications administered on an as-needed basis, including over-the-counter medications used according to the product labeling, will be permitted except for disulfiram, dextromethorphan, or dextrorphan. Medications for pain management will be allowed as needed.
3. Have normal organ function as defined below:
• Direct bilirubin <19 µmol/L
• Alanine transaminase (serum glutamic pyruvic transaminase) =114 IU/L
• Creatinine =115 µmol/L
• ?-glutamyltransferase =110 IU/L
4. Have at least one of the following baseline values: hemoglobin level of <10 g/dL, numerical pain rating scale (NPRS) score of =4, or 6-minute walk distance (6MWD) of =500 m
5. If female, be non-pregnant and non-breast feeding and be surgically sterile or using an acceptable method of contraception throughout the study and for 3 months after the last dose of study medication
6. Have completed an outpatient screening visit consisting of medical history, physical examination, 12-lead ECG, vital signs, hematology and chemistry tests, urinalysis, urine drug screen, urine or serum pregnancy test (females), hemoglobin electrophoresis, hepatitis B and C screening, and HIV serology (Note: Subjects with abnormal screening values may be eligible if the results are not clinically significant or represent expected manifestations of SCD, as judged by the investigator in agreement with the sponsor or medical monitor.)
7. Be able to understand and have provided written informed consent including signature on an informed consent form (ICF) approved by an institutional review board (IRB) or independent ethics committee (IEC)
8. Have provided written authorization for use and disclosure of protected health information (PHI)
9. Agree to abide by the study schedule and to return for the required assessments
10. Be willing to abstain from foods high in 5-HMF (e.g., malt, barley, beer, balsamic vinegar, dried fruits, and caramel products) and limit consumption of coffee to 2 cups per day during the study
11. Subjects with poor venous access who are unlikely to be able to provide repeated blood samples for pharmacokinetic analyses may be enrolled in the study if their venous access is sufficient to provide blood samples for safety assessments of clinical laboratory and hematology measures. (No more than 3 subjects per dose cohort with poor venous access may be enrolled.)
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Have been hospitalized in the 14 days before enrollment, for any reason
2. Have evidence of clinically significant cardiovascular, respiratory, renal, hepatic, pulmonary, gastrointestinal, hematological, neurological, psychiatric, or other disease that may interfere with the objectives of the study or the safety of the subject, as judged by the investigator in agreement with the sponsor or medical monitor, or have been hospitalized in the past 6 months as a result of these conditions (for SCD-related morbidity, a minimum of 14 days from the last hospitalization is required)
3. Have a history of development of abnormal liver function test (LFT) values in association with administration of xenobiotic provided the causality relationship of the xenobiotic to the LFT abnormality is judged to be at least probably related.
4. Be considered not suitable for participation in this study for any reason, as judged by the investigator
5. Have taken any other investigational drug within 30 days before the screening visit
6. Consumed more than 21 alcohol units per week in the last month or more than 3 units per day in the last week
7. Have received disulfiram or 4-methoxypyrazole within 30 days before dosing (may affect PK results due to assay interference)
8. Have received dextromethorphan or dextrorphan within 7 days before dosing
9. Have taken herbal preparations in the 2 weeks before dosing
10. Have positive result for urine drug test (cocaine, opiates [except prescribed], amphetamines, methamphetamines, benzodiazepines [except prescribed]) at screening visit (A positive test for THC will not be an exclusion)