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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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 weeks

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 weeks

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Blood Hemoglobin

Side effects data

From 2022 Phase 2 trial • 46 Patients • NCT03061474

25%

Diarrhoea

17%

Fall

17%

Urinary tract infection

Constipation

Vomiting

Anxiety

Back pain

Laboratory test abnormal

Rash

Transaminases increased

Head Injury

Colorectal cancer

Renal cyst

Dyspepsia

Upper respiratory tract infection

Arthralgia

Headache

Neuropathy peripheral

Fungal infection

Penile ulceration

Syncoper

Foot fracture

Osteoporosis

Tooth abscess

Agitation

Weight decreased

Blood testosterone decreased

Hiatus hernia

Glomerular Ffiltration rate decreased

Miliaria

Urticaria

Coccydynia

Pleocytosis

Actinic keratosis

Viral upper respiratory tract infection

Thrombocytopenia

Hyperlipidaemia

Laceration

Limb injury

Post lumbar puncture syndrome

Parkinson's disease

Pneumonia

Diabetes mellitus

Myalgia

Cognitive disorder

Colitis ulcerative

Infection

Dehydration

Tremor

Cancer surgery

Vitamin D deficiency

Foot deformity

Hypokalaemia

Psoriasis

Dizziness

Dysgeusia

Basal cell carcinoma

Eructation

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Nicotinamide

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

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Institutes of Health (NIH)NIH

2,696 Previous Clinical Trials

6,952,519 Total Patients Enrolled

National Heart, Lung, and Blood Institute (NHLBI)NIH

3,835 Previous Clinical Trials

47,310,622 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What medical uses does Nicotinamide have?

"Nicotinamide is generally used to address the symptoms of refractory anemia. Additionally, it can be beneficial in cases of hepatic insufficiency, secondary anemia, and as a dietary supplement."

Answered by AI

What prior attempts at employing Nicotinamide have been made?

"Currently, 113 Nicotinamide studies are operational with 18 trials in the third phase. Chicago, Illinois is housing most of these clinical experiments; however there are 1510 medical sites running research for this medication."

Answered by AI

Has the FDA sanctioned Nicotinamide as a therapeutic agent?

"The safety profile of Nicotinamide is rated a 1 since this is an early phase trial, indicating that there are not comprehensive studies on its efficacy or risk."

Answered by AI

What is the current enrollment capacity for this trial?

"Affirmative, the clinicaltrials.gov website provides information that this medical trial is open for enrollment at present. This project was first made public on January 24th 2020 and recently updated on March 21st 2022; they are seeking to recruit 20 patients from a single site."

Answered by AI

Are there still opportunities to join this clinical trial?

"As of this moment, the trial is open to new participants. Its original posting date was January 24th 2020 and has been revised most recently on March 21st 2022."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2020. "A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04055818.

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