Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease (SCD-001)

November 6, 2019 updated by: Celgene

A Prospective, Multi-Center, Open-Label, Dose-Escalation Study to Determine the Maximum Tolerated Dose, Safety and Effect on Induction of Fetal Hemoglobin of CC-4047 In Subjects With Sickle Cell Disease

The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
12

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Michigan
      • Detroit, Michigan, United States, 48201-2097
        • Karmanos Cancer Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 60 years (ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Ages 18 to 60 years, inclusive, at the time of signing the informed consent form
  • Clinically significant Sickle Cell Disease (SCD) documented as Sickle Cell Anemia or Sickle Beta-Zero Thalassemia
  • Clinically significant SCD defined as at least 1 documented pain episode per year averaged over the past 3 years or one episode of active leg ulcers, priapism, or acute chest syndrome over the past 3 years
  • Failed to achieve at least an absolute 5% increase in hemoglobin F while taking Hydroxyurea (HU) or unable to tolerate HU as described by the treating physician and may include but is not limited to lack of efficacy (such as people who have continued to have pain episodes more than 2 times a year or who have had acute chest or multiorgan failure syndromes or an episode of priapism), or other severe side effects while on HU (severe side effects include significant myelosuppression; skin cancer; or cytotoxicity evidenced by gastrointestinal symptoms, dermatological reactions, hepatic enzyme elevations, pulmonary fibrosis or neurological disturbances), or refusal of hydroxyurea therapy by the informed patient
  • Able to adhere to the study visit schedule and other protocol requirements
  • Females must be surgically sterile (post hysterectomy or bilateral oophorectomy) or naturally postmenopausal for at least 24 consecutive months (i.e., have not had menses at any time in the preceding 24 consecutive months)
  • Male subjects must agree to use a latex condom during any sexual contact with females of child bearing potential (FCBP) during study drug treatment, during dose interruptions, and for at least 28 days following discontinuation of study drug even if they have undergone a successful vasectomy. Counseling about the requirement for latex condom use during sexual contact with FCBP and the potential risks of fetal exposure must be conducted at a minimum of every 28 days.
  • Male subjects must agree to abstain from donating semen or sperm while taking study drug and for 28 days after stopping study drug.
  • Both males and females must agree to abstain from donating blood while taking study drug and for 28 days after stopping study drug.
  • Both males and females must agree that they will not share study drug and will be counseled about the potential risks of fetal exposure.

Exclusion Criteria:

  • Known positive status for human immune virus (HIV), Hepatitis B; or acute/chronic, active Hepatitis C
  • Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form
  • Females of childbearing potential, pregnant or lactating females
  • Any condition, including the presence of laboratory abnormalities, which place the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
  • Subjects unlikely to comply with birth control, medication dosing, or study visit requirements
  • Subjects with severe or life threatening, active, unresolved infections

  • Any of the following laboratory abnormalities derived from the Screening Visit:

    • Platelet count or white blood cell count (WBC) less than the lower limit of normal (LLN)
    • Total hemoglobin less than or equal to 6.0 g/dL
    • Hemoglobin A (HbA) from transfusion greater than 20% at baseline
    • Creatinine greater than Upper Limit of Normal (ULN)
    • Alanine Aminotransferase / Serum Glutamic Pyruvic Transaminase (ALT/SGPT) greater than 3 x ULN
    • Total bilirubin greater than 10 mg/dL
  • Subjects on a chronic transfusion program
  • History of non-catheter related Deep Vein Thrombosis (DVT) or stroke
  • Chronic symptomatic constipation
  • History of cancer (except basal cell or squamous cell carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been free of disease for at least three years.
  • Use of agents that can induce fetal hemoglobin within 90 days (three months) of Day 1 (i.e. HU, butyrates, decitabine, 5-azacytidine, or erythropoietin)
  • Use of experimental drug or treatment within 30 days of the first dose of study drug
  • History of allergic reaction to thalidomide or lenalidomide
  • Prior desquamating (blistering) rash while taking thalidomide or lenalidomide
  • Greater than or equal to a Grade 2 neuropathy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
EXPERIMENTAL: Cohort 1: 0.5 mg pomalidomide
0.5 mg pomalidomide orally daily for 84 days
Drug: pomalidomide
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
Other Names:
  • CC-4047
EXPERIMENTAL: Cohort 2: 1.0 mg pomalidomide
1.0 mg pomalidomide orally daily for 84 days
Drug: pomalidomide
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
Other Names:
  • CC-4047
EXPERIMENTAL: Cohort 3: 2.0 mg pomalidomide
2.0 mg pomalidomide orally daily for 84 days
Drug: pomalidomide
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
Other Names:
  • CC-4047
EXPERIMENTAL: Cohort 4: 3.0 mg pomalidomide
3.0 mg pomalidomide orally daily for 84 days
Drug: pomalidomide
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
Other Names:
  • CC-4047
EXPERIMENTAL: Cohort 5: 4.0 mg pomalidomide
4.0 mg pomalidomide orally daily for 84 days
Drug: pomalidomide
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
Other Names:
  • CC-4047

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Maximum Tolerated Dose (MTD)
Time Frame: Up to 84 days
Maximum Tolerated Dose
Up to 84 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Adverse Events
Time Frame: Up to 169 days
Type, frequency, and severity of adverse events, and relationship of adverse events to pomalidomide
Up to 169 days
Absolute fetal hemoglobin change
Time Frame: UP to 169 days
Percent of subjects with an absolute increase of 5% in percent fetal hemoglobin levels during study treatment
UP to 169 days
% total hemoglobin
Time Frame: Up to 169 days
Percent change in total hemoglobin from baseline to highest level
Up to 169 days
Rate of total hemoglobin change
Time Frame: Up to 169 days
Rate of change of total hemoglobin from baseline to highest level
Up to 169 days
Inflammation markers and cytokines
Time Frame: Up to 169 days
Change in serum inflammation markers and cytokines from baseline, during and at end of study treatment
Up to 169 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Celgene

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 1, 2007

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 1, 2012

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2013

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 27, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 27, 2012

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 31, 2012

Study Record Updates

Last Update Posted (ACTUAL)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 8, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 6, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CC-4047-SCD-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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