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Combination Therapy for Sickle Cell Disease

Phase 1
Recruiting
Led By Robert Molokie
Research Sponsored by EpiDestiny, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 18 years or older
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)
Must not have
Concurrent diagnosis of malignancy including known Myelodysplastic syndrome, leukemia, or an abnormal karyotype
Being positive for HIV infection
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 weeks
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment for sickle cell disease, which is a blood disorder. The treatment is a combination of three drugs, and it is being compared to a standard treatment of just one drug.

Who is the study for?
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.
What is being tested?
The 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.
What are the potential side effects?
Potential side effects for this trial have not been explicitly listed but may include typical reactions to medications such as gastrointestinal issues, possible liver enzyme elevations due to nicotinamide or decitabine, fatigue, blood count changes and increased risk of infection.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I haven't had any severe sickle cell complications or hospital visits in the last 2 weeks.
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I weigh at least 40kg.
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I have been diagnosed with sickle cell disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have another cancer diagnosis such as leukemia or a genetic abnormality in my cells.
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I am HIV positive.
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I am unable to understand and give consent for treatment.
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I took my last dose of HU within the last 4 weeks.
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My kidney function is severely impaired.
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My heart condition severely limits my physical activity.
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I need considerable assistance and am unable to carry out any work activities.
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I do not have a severe illness that could cause death within 6 months.
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I am on medication for seizures or altered mental status.
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I am on a long-term blood transfusion treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Blood Hemoglobin

Side effects data

From 2022 Phase 2 trial • 46 Patients • NCT03061474
25%
Diarrhoea
17%
Urinary tract infection
17%
Fall
8%
Constipation
8%
Vomiting
8%
Anxiety
8%
Laboratory test abnormal
8%
Back pain
4%
Cognitive disorder
4%
Upper respiratory tract infection
4%
Coccydynia
4%
Agitation
4%
Fungal infection
4%
Dizziness
4%
Osteoporosis
4%
Basal cell carcinoma
4%
Vitamin D deficiency
4%
Glomerular Ffiltration rate decreased
4%
Hypokalaemia
4%
Pneumonia
4%
Syncoper
4%
Tremor
4%
Blood testosterone decreased
4%
Weight decreased
4%
Hyperlipidaemia
4%
Foot deformity
4%
Dysgeusia
4%
Headache
4%
Eructation
4%
Pleocytosis
4%
Limb injury
4%
Renal cyst
4%
Laceration
4%
Myalgia
4%
Diabetes mellitus
4%
Arthralgia
4%
Thrombocytopenia
4%
Dyspepsia
4%
Infection
4%
Post lumbar puncture syndrome
4%
Neuropathy peripheral
4%
Colorectal cancer
4%
Tooth abscess
4%
Colitis ulcerative
4%
Hiatus hernia
4%
Dehydration
4%
Parkinson's disease
4%
Transaminases increased
4%
Viral upper respiratory tract infection
4%
Foot fracture
4%
Head Injury
4%
Actinic keratosis
4%
Miliaria
4%
Penile ulceration
4%
Psoriasis
4%
Rash
4%
Urticaria
4%
Cancer surgery
100%
80%
60%
40%
20%
0%
Study treatment Arm
Nicotinamide
Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

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
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Nicotinamide
2021
Completed Phase 3
~2810

Find a Location

Who is running the clinical trial?

National Institutes of Health (NIH)NIH
2,810 Previous Clinical Trials
8,159,612 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,922 Previous Clinical Trials
47,760,726 Total Patients Enrolled
EpiDestiny, Inc.Lead Sponsor
3 Previous Clinical Trials
72 Total Patients Enrolled

Media Library

Sickle Cell Disease Research Study Groups: Nicotinamide, THU Decitabine
~3 spots leftby Nov 2025
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