NCT01522547

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2007

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2007

Completed
4.5 years until next milestone

First Submitted

Initial submission to the registry

January 27, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

January 31, 2012

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2012

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

November 8, 2019

Status Verified

November 1, 2019

Enrollment Period

5.2 years

First QC Date

January 27, 2012

Last Update Submit

November 6, 2019

Conditions

Anemia, Sickle Cell

Keywords

PomalidomideSickle Cell DiseaseFetal Hemoglobin

Outcome Measures

Primary Outcomes (1)

  • Maximum Tolerated Dose (MTD)

    Maximum Tolerated Dose

    Up to 84 days

Secondary Outcomes (5)

  • Adverse Events

    Up to 169 days

  • Absolute fetal hemoglobin change

    UP to 169 days

  • % total hemoglobin

    Up to 169 days

  • Rate of total hemoglobin change

    Up to 169 days

  • Inflammation markers and cytokines

    Up to 169 days

Study Arms (5)

Cohort 1: 0.5 mg pomalidomide

EXPERIMENTAL

0.5 mg pomalidomide orally daily for 84 days

Drug: pomalidomide

Cohort 2: 1.0 mg pomalidomide

EXPERIMENTAL

1.0 mg pomalidomide orally daily for 84 days

Drug: pomalidomide

Cohort 3: 2.0 mg pomalidomide

EXPERIMENTAL

2.0 mg pomalidomide orally daily for 84 days

Drug: pomalidomide

Cohort 4: 3.0 mg pomalidomide

EXPERIMENTAL

3.0 mg pomalidomide orally daily for 84 days

Drug: pomalidomide

Cohort 5: 4.0 mg pomalidomide

EXPERIMENTAL

4.0 mg pomalidomide orally daily for 84 days

Drug: pomalidomide

Interventions

pomalidomideDRUG

Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg

Also known as: CC-4047
Cohort 1: 0.5 mg pomalidomideCohort 2: 1.0 mg pomalidomideCohort 3: 2.0 mg pomalidomideCohort 4: 3.0 mg pomalidomideCohort 5: 4.0 mg pomalidomide

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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
  • +7 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Karmanos Cancer Institute

Detroit, Michigan, 48201-2097, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cellbeta-Thalassemia

Interventions

pomalidomide

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesThalassemia

Study Officials

  • Robert Knight, MD

    Celgene

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 27, 2012

First Posted

January 31, 2012

Study Start

August 1, 2007

Primary Completion

October 1, 2012

Study Completion

December 1, 2013

Last Updated

November 8, 2019

Record last verified: 2019-11

Locations

US(1)