Combination Therapy for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

Overseen byRobert Molokie

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Waitlist Available

Sponsor: EpiDestiny, Inc.

No Placebo Group

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

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

Research Team

Robert Molokie

Principal Investigator

University of Illinois at Chicago College of Medicine

Eligibility Criteria

Adults over 18 with confirmed sickle cell disease, symptomatic despite hydroxyurea treatment or refusing it. They must be stable (no recent acute SCD complications), weigh at least 40kg, and comply with care. Excluded are those unable to consent, pregnant/breastfeeding women, individuals with certain organ dysfunctions or severe concurrent diseases, drug/alcohol abusers within a year, and anyone on other experimental drugs recently.

Inclusion Criteria

I am 18 years old or older.

I haven't had any severe sickle cell complications or hospital visits in the last 2 weeks.

I weigh at least 40kg.

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Exclusion Criteria

Platelet count >800 x 109/L

I am a man who will not use a condom during sex with a woman who can have children, even though I am asked to do so for 4 weeks after my last treatment.

You have used illegal drugs or abused alcohol in the past year.

See 17 more

Treatment Details

Interventions

Nicotinamide (Other)

Trial OverviewThe trial is testing the effectiveness of nicotinamide alone and in combination with oral THU-decitabine in treating high-risk sickle cell disease. It's a randomized control study involving 20 subjects who will receive either one of the treatments or both together.

Participant Groups

2Treatment groups

Experimental Treatment

Group I: THU DecitabineExperimental Treatment1 Intervention

Oral 250 mg THU and 5 mg decitabine Once per week

Group II: NicotinamideExperimental Treatment1 Intervention

Oral Nicotinamide 1000 mg twice daily