Sickle Cell DiseasePhase 1NCT01783691
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
A Phase 1 study testing NKTT120 in people with Sickle Cell Disease, run by NKT Therapeutics at 6 sites in United States.
Trial summary
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- NKTT120
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 6 sitesUnited States
- Study dates
- Feb 2013to May 2015
- 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
- Age 18 to 50 years
- Subject has a confirmed diagnosis of HbSS or HbSβ0thal
- Subject has stable SCD defined as not having acute VOC, ACS, or other major SCD associated event during the month prior to enrollment
Who cannot join
- Subject had an SCD-related VOC or ACS that required hospitalization or treatment in acute care outpatient setting in the month prior to enrollment
- Subject requires a program of prescheduled regularly administered packed red blood cell (pRBC) transfusions or Subject received a pRBC transfusion in the month prior to enrollment
- Subject has evidence of latent or active tuberculosis
- Subject has a major concurrent illness or medical condition
- Subject is pregnant or nursing
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Johns Hopkins School of Medicine
Baltimore, Maryland, 21205
Brigham and Women's Hospital
Boston, Massachusetts, 02115
UNC Sickle Cell Program
Chapel Hill, North Carolina, 27514
Blood Center of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospital & Research Center at Oakland
Oakland, California, 94609
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Single Ascending Dose Phase 1 Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biologic Activity of Intravenous NKTT120 in Adults With Stable Sickle Cell Disease
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.
- Conditions
- Sickle Cell Disease
- Being tested
- NKTT120
- Keywords
- Sickle cell disease
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NKTT120Drug
| Study group | Role | What they receive |
|---|---|---|
| NKTT120 | Experimental | Not described Receives: Drug: NKTT120 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.
Measured: 180 days
Secondary outcomes (2)
Pharmacokinetics assessed by plasma drug concentration levels
Measured: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days
Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets
Measured: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2013 (estimated)
- Study startFeb 2013
- Primary completionMay 2015
- Study completionMay 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- NKT Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rosemary Mazanet, MD, Study director, NKT Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01783691
- Sponsor's protocol ID
- 120-SCD1
- Status last verified
- Jun 2015
- First submitted
- 1 Feb 2013
- Last updated
- 26 Jun 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG. NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease. PLoS One. 2017 Feb 2;12(2):e0171067. doi: 10.1371/journal.pone.0171067. eCollection 2017.
PubMed 28152086
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single Ascending Dose Phase 1 Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biologic Activity of Intravenous NKTT120 in Adults With Stable Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01783691
- Acronym / sponsor ID
- 120-SCD1
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle cell disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- NKT TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Feb 2013
- Primary completion
- May 2015 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 5 Feb 2013 (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 this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NKTT120 | Experimental | Drug: NKTT120 |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Age 18 to 50 years
- Subject has a confirmed diagnosis of HbSS or HbSβ0thal
- Subject has stable SCD defined as not having acute VOC, ACS, or other major SCD associated event during the month prior to enrollment
Key exclusion criteria (5 listed)
- Subject had an SCD-related VOC or ACS that required hospitalization or treatment in acute care outpatient setting in the month prior to enrollment
- Subject requires a program of prescheduled regularly administered packed red blood cell (pRBC) transfusions or Subject received a pRBC transfusion in the month prior to enrollment
- Subject has evidence of latent or active tuberculosis
- Subject has a major concurrent illness or medical condition
- Subject is pregnant or nursing
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 |
|---|---|---|
| NKTT120 | Drug | NKTT120 |
Primary outcome
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam.
Time frame: 180 days
2 secondary outcomes
Pharmacokinetics assessed by plasma drug concentration levels
Time frame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days
Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets
Time frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 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 26 Jun 2015.
- Derived from this study: PubMed 28152086Field JJ, Majerus E, Ataga KI, Vichinsky EP, Schaub R, Mashal R, Nathan DG. NNKTT120, an anti-iNKT cell monoclonal antibody, produces rapid and sustained iNKT cell depletion in adults with sickle cell disease. PLoS One. 2017 Feb 2;12(2):e0171067. doi: 10.1371/journal.pone.0171067. eCollection 2017.
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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.
Rosemary Mazanet, MD study director
NKT Therapeutics
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NKTT120 in people with Sickle Cell Disease, run by NKT Therapeutics at 6 sites 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 3 inclusion and 5 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 26 Jun 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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?
Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. (time frame: 180 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01783691, which also lists 1 linked publication.
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 26 Jun 2015.