Isoquercetin in Sickle Cell Anemia

NCT04474626 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: 20-087
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This research study is being done to assess the safety and effectiveness of isoquercetin to reduce levels of soluble P-Selectin in patients with sickle cell disease. Isoquercetin is a naturally occurring flavonoid-or vitamin. You will find quercetin and isoquercetin in fruits and vegetables. The names of the study drug involved in this study are/is: \- Isoquercetin

Conditions

Sickle Cell Disease; Sickle Cell-Beta0-Thalassemia

Keywords

Sickle Cell Disease; Sickle Cell-Beta0-Thalassemia; isoquercetin

Outcome Measures

Primary Outcomes (1)

• Change in sP Selectin levels with isoquercetin

  Comparisons between baseline and follow-up measurements (i.e. change in sP-Selectin), will be performed using a two-tailed, paired t-test analyses.

  baseline to 28 Days

Secondary Outcomes (4)

• Platelet dependent thrombin generation (coagulation)

  baseline to 1 year

• sE-selectin (adhesion)-Biomarker

  baseline to 1 year

• C-reactive protein CRP

  baseline to 1 year

• Number of Participants With Treatment-Related Adverse Events

  start of study treatment up to 13 months

Study Arms (1)

ISOQUERCETIN

EXPERIMENTAL

The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits \- ISOQUERCETIN: Oral Study Drug, 1 time per day, per predetermined dosed per 28 treatment cycle. This will continue for up to 337 days.

Drug: Isoquercetin

Interventions

Isoquercetin DRUG

Oral, 1 time per day, per predetermined dosed per 28 treatment cycle.

Also known as: IQC-950AN

ISOQUERCETIN

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Eligible subjects require an established diagnosis of sickle cell disease/homozygous hemoglobin S (SCD-SS) or sickle cell disease hemoglobin β0-thalassemia (SCD-Sβ0-thal).
• Patients on other therapy including hydroxyurea will be included.
• Age 18-50 years.
• Participants must have preserved organ and marrow function as defined below:
• leukocytes ≥2,000/mcL
• platelets ≥75,000/mcL
• AST(SGOT)/ALT(SGPT) ≤2.5 × institutional upper limit of normal
• Estimated creatinine clearance ≥45 mL/min/1.73 m2 for participants with creatinine levels above institutional normal.
• Subjects with no evidence of worsening over the last 4 weeks (e.g. any acute complication of SCD including but not limited to VOC, acute chest syndrome and stroke, that required unscheduled medical attention or intervention) as determined by the investigator will be included.
• Patients on anticoagulation therapy will be excluded.
• The effects of isoquercetin on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of isoquercetin administration.
• Ability to understand and the willingness to sign a written informed consent document

You may not qualify if:

• Participants may not be concurrently receiving any other study agents.
• Subjects with no evidence of worsening over the last 1 month (e.g. any acute complication of SCD including but not limited to VOC, acute chest syndrome and stroke, that required unscheduled medical attention or intervention) as determined by the investigator will be included.
• Familial bleeding diathesis.
• Known diagnosis of disseminated intravascular coagulation.
• Currently receiving anticoagulant therapy.
• Currently using daily use of aspirin (\>81mg daily), Clopidogrel (Plavix), cilostazol (Pletal), aspirin-dipyridamole (Aggrenox) (within 10 days)
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to isoquercetin.
• Uncontrolled intercurrent illness including but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina, cardiac arrhythmia, or psychiatric illness/social situations that would limit study compliance.
• Pregnant women are excluded from this study because isoquercetin is a PDI inhibitor with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk of adverse events in nursing infants secondary to treatment of the mother with isoquercetin, breastfeeding should be discontinued if the mother is treated with isoquercetin. These potential risks may also apply to other agents used in this study.

Sponsors & Collaborators

• Jeffrey Zwicker, MD: lead
• Quercegen Pharmaceuticals: collaborator

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

isoquercitrin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jeffrey Zwicker, MD

  Beth Israel Deaconess Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 6, 2020
• First Posted: July 17, 2020
• Study Start: December 1, 2020
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2024
• Last Updated: January 11, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: BIDMC - Contact the Beth Israel Deaconess Medical Center Technology Ventures Office at tvo@bidmc.harvard.edu