A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease

STATUS

Recruiting
End date

Mar 3, 2023
participants needed

20
sponsor

EpiDestiny, Inc.

Send

Updated on 14 April 2022

See if I qualify

hydroxyurea

narcotics

electrophoresis

acute pain

acute chest syndrome

chest syndrome

Summary

A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

Description

A randomized control trial comparing oral THU-decitabine to nicotinamide (1:1 randomization), and then comparing the effects of the combination of nicotinamide with THU-decitabine vs either treatment alone. Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks. Patients have the option to enter an extension phase of combination treatment for an additional 24 weeks (total of 48 weeks)

Details

Condition	Sickle Cell Disease
Treatment	Nicotinamide
Clinical Study Identifier	NCT04055818
Sponsor	EpiDestiny, Inc.
Last Modified on	14 April 2022

How understandable was the trial content above?

Hard to understand

Easy to understand

Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Age 18 years or older
	Written informed consent provided by the subject before study entry
	Confirmed sickle cell disease (SCD) as determined by hemoglobin electrophoresis or liquid chromatography
	Subject is in his/her steady state and not having any acute complication due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome in the past 14 days)
	Weight at least 40kg
	Regular compliance with comprehensive care and previous therapy
	Symptomatic SCD is defined as having one of following, despite at least 6 months of hydroxyurea therapy, or refuse to take hydroxyurea for personal reasons: fetal hemoglobin <0.5 g/dL, or 3 or more pain episodes per year requiring parenteral narcotics, or 1 or more acute chest syndrome episodes, or Hemoglobin <9 g/dL and absolute reticulocyte count <250,000/mm3
Exclusion Criteria
	Inability to give informed consent
	Experienced severe sepsis or septic shock within the previous 12 weeks
	Last HU dose was ingested within the previous 4 weeks
	Currently pregnant or breast-feeding
	Alanine Aminotransferase (ALT) ≥ 3 times the upper limit of normal or albumin <2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl
	Serum creatinine >2.9 mg/dL and calculated creatinine clearance <30 mL/min
	Platelet count >800 x 109/L
	Absolute neutrophil count <1.5 x 109/L
	Female of active childbearing potential who is unwilling to use at least one of the two following forms of birth control: (i) not having heterosexual sexual contact beginning at the screening visit and continuing until 4 weeks after the last dose of decitabine OR (ii) intrauterine device (IUD)
	Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a female with child-bearing potential, beginning at the screening visit and continuing until 4 weeks after taking the last dose of THU and decitabine. This requirement applies also to males who have had a successful vasectomy
	Altered mental status or recurrent seizures requiring anti-seizure medications
	Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely
	Concurrent diagnosis of malignancy including known Myelodysplastic syndrome, leukemia, or an abnormal karyotype
	New York Heart Association (NYHA) class III/IV status
	Eastern Co-operative Oncology Group (ECOG) performance status ≥3
	Participant is on chronic transfusion therapy
	Known history of illicit drug or alcohol abuse within the past 12 months
	Other experimental or investigational drug therapy in the past 28 days
	Taking l-glutamine within the last 28 days
	Being positive for HIV infection

How to participate?

Step 1 Connect with a study center

Select a site
Send a message

You have contacted , on

Your message has been sent to the study team at ,

A copy of the message has been sent to your email

What happens next?

You can expect the study team to contact you via email or phone in the next few days.
Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Primary Contact

0/250

By sending a message, I agree that my data will be sent to a representative for the selected site and processed in accordance with this website's Privacy Policy (see link in footer).

Step 2 Get screened

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

Step 3 Enroll in the clinical study

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Step 4 Get your study results

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease