Anemia, Sickle CellPhase 1SCD-001NCT01522547
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
A Phase 1 study testing pomalidomide in people with Anemia, Sickle Cell, run by Celgene at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- pomalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2007to 1 Dec 2013
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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)
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Karmanos Cancer Institute
Detroit, Michigan, 48201-2097
About this study
Official title: 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.
- Conditions
- Anemia, Sickle Cell
- Being tested
- pomalidomide
- Keywords
- Pomalidomide, Sickle Cell Disease, Fetal Hemoglobin
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pomalidomideDrug
Also called CC-4047
Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: 0.5 mg pomalidomide | Experimental | 0.5 mg pomalidomide orally daily for 84 days Receives: Drug: pomalidomide |
| Cohort 2: 1.0 mg pomalidomide | Experimental | 1.0 mg pomalidomide orally daily for 84 days Receives: Drug: pomalidomide |
| Cohort 3: 2.0 mg pomalidomide | Experimental | 2.0 mg pomalidomide orally daily for 84 days Receives: Drug: pomalidomide |
| Cohort 4: 3.0 mg pomalidomide | Experimental | 3.0 mg pomalidomide orally daily for 84 days Receives: Drug: pomalidomide |
| Cohort 5: 4.0 mg pomalidomide | Experimental | 4.0 mg pomalidomide orally daily for 84 days Receives: Drug: pomalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
Maximum Tolerated Dose
Measured: Up to 84 days
Secondary outcomes (5)
Adverse Events
Type, frequency, and severity of adverse events, and relationship of adverse events to pomalidomide
Measured: Up to 169 days
Absolute fetal hemoglobin change
Percent of subjects with an absolute increase of 5% in percent fetal hemoglobin levels during study treatment
Measured: UP to 169 days
% total hemoglobin
Percent change in total hemoglobin from baseline to highest level
Measured: Up to 169 days
Rate of total hemoglobin change
Rate of change of total hemoglobin from baseline to highest level
Measured: Up to 169 days
Inflammation markers and cytokines
Change in serum inflammation markers and cytokines from baseline, during and at end of study treatment
Measured: Up to 169 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2012 (estimated)
- Study start1 Aug 2007
- Primary completion1 Oct 2012
- Study completion1 Dec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Sickle CellStudies of the same condition, open and under way.
- More trials run by CelgeneThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Knight, MD, Study director, Celgene
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01522547
- Sponsor's protocol ID
- CC-4047-SCD-001
- Acronym
- SCD-001
- Status last verified
- Nov 2019
- First submitted
- 27 Jan 2012
- Last updated
- 8 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01522547
- Acronym / sponsor ID
- SCD-001 / CC-4047-SCD-001
- Conditions
- Anemia, Sickle Cell
- Keywords
- Pomalidomide, Sickle Cell Disease, Fetal Hemoglobin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 1 Aug 2007 (actual)
- Primary completion
- 1 Oct 2012 (actual)
- Study completion
- 1 Dec 2013 (actual)
- First posted
- 31 Jan 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: 0.5 mg pomalidomide | Experimental | Drug: pomalidomide0.5 mg pomalidomide orally daily for 84 days |
| Cohort 2: 1.0 mg pomalidomide | Experimental | Drug: pomalidomide1.0 mg pomalidomide orally daily for 84 days |
| Cohort 3: 2.0 mg pomalidomide | Experimental | Drug: pomalidomide2.0 mg pomalidomide orally daily for 84 days |
| Cohort 4: 3.0 mg pomalidomide | Experimental | Drug: pomalidomide3.0 mg pomalidomide orally daily for 84 days |
| Cohort 5: 4.0 mg pomalidomide | Experimental | Drug: pomalidomide4.0 mg pomalidomide orally daily for 84 days |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (22 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| pomalidomideAlso: CC-4047Pomalidomide orally for 84 days daily in doses ranging from 0.5 mg to 4.0 mg | Drug | Cohort 1: 0.5 mg pomalidomide, Cohort 2: 1.0 mg pomalidomide, Cohort 3: 2.0 mg pomalidomide, Cohort 4: 3.0 mg pomalidomide, Cohort 5: 4.0 mg pomalidomide |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: Up to 84 days
Maximum Tolerated Dose
5 secondary outcomes
Adverse Events
Time frame: Up to 169 days
Absolute fetal hemoglobin change
Time frame: UP to 169 days
% total hemoglobin
Time frame: Up to 169 days
Rate of total hemoglobin change
Time frame: Up to 169 days
Inflammation markers and cytokines
Time frame: Up to 169 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Knight, MD study director
Celgene
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing pomalidomide in people with Anemia, Sickle Cell, run by Celgene at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) (time frame: Up to 84 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2019.