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AB1 for Sickle Cell Disease ((SCD) Trial)

Phase 1 & 2

Waitlist Available

Led By Nirmish Shah, MD

Research Sponsored by Nirmish Shah

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 18 to 45 years of age, inclusive at screening

Confirmed SS or S-b0-thalassemia SCD

Must not have

Plans for hospitalization, surgery, or other major procedures during the duration of the study or between screening and baseline

Acute complications due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome) in the 28 days prior to screening visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from the time of consent up to 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial involves giving a drug in increasing doses to find the safest and most effective amount. It targets patients who might benefit from increased HbF levels. The dose is increased if no serious side effects occur. Hydroxyurea has been studied for its ability to increase fetal hemoglobin (HbF) levels in patients with sickle cell anemia, showing varying degrees of effectiveness and manageable side effects.

Who is the study for?

Adults aged 18-45 with Sickle Cell Disease (SCD), specifically SS or S-b0-thalassemia, who have had 2-10 sickle crises in the past year but none in the last month. Participants must not be pregnant, agree to contraception use, and should have a history of following medical care. They can't join if they have severe kidney issues, abnormal blood counts, certain heart conditions, recent drug abuse, or are on specific other treatments.

What is being tested?

The trial is testing AB1 at increasing doses from 2mg to 32mg taken orally once daily for eight weeks. It's an open-label study where everyone knows what treatment they're getting and it aims to find out how much of the drug can be given safely.

What are the potential side effects?

Potential side effects aren't specified here but generally could include typical reactions to new medications such as digestive discomfort, headaches, possible allergic reactions or changes in blood counts due to the nature of SCD.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 18 and 45 years old.

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I have been diagnosed with SS or Sβ0-thalassemia sickle cell disease.

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I've had 2-10 sickle cell crises in the last year, but none in the past month.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any planned surgeries or major procedures during the study.

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I haven't had any severe sickle cell complications in the last 28 days.

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My heart condition severely limits my physical activity.

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I am on medication for seizures or altered mental status.

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I am not willing or able to follow the birth control requirements of the study.

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I have another cancer diagnosis such as MDS, leukemia, or an abnormal karyotype.

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I do not have a severe illness that could cause death within 6 months.

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My kidney function is severely impaired.

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I am a man and cannot follow the study's birth control rules.

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I need considerable assistance and am unable to carry out any work activities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from the time of consent up to 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from the time of consent up to 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and from the time of consent up to 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of ≥Grade 2 study related adverse events as measured by patient report/medical record

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: AB1Experimental Treatment1 Intervention

AB1 is the investigational product in this study taken orally, once daily, for 8 weeks. This will be an open-label, dose escalating study with a starting dose of 2mg. Up to 6 additional cohorts will be enrolled at subsequently higher doses of 4mg, 8mg, 10 mg, 12 mg, 16mg, and 32mg.

0 / 13Eligibility criteria met