Immune Thrombocytopenia PurpuraPhase 1NCT01963260
Single Rising Dose Study of MK-8723 in Healthy Participants and Participants With Immune Thrombocytopenia Purpura (MK-8723-001)
A Phase 1 study testing MK-8723 in people with Immune Thrombocytopenia Purpura, run by Merck Sharp & Dohme LLC.
Trial summary
The primary objectives of this study are to assess the safety and tolerability of single rising doses of MK-8723 in healthy adult participants and adult participants with chronic immune thrombocytopenia purpura (ITP) and to assess pharmacodynamics of MK-8723 in participants with ITP. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Immune Thrombocytopenia Purpura
- What is being tested?
- MK-8723
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- 31 Oct 2013to 26 Apr 2015
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Female participants must be non-pregnant, non-breast feeding, and of non-childbearing potential
- Has a Body Mass Index (BMI) <=32 kg/m^2
- Has a body weight >= 50 kg and <= 100 kg
- Has been judged to be in good health based on medical history, physical examination, vital sign measurements, electrocardiogram (ECG), and laboratory safety tests
- Non-smoker or has not used nicotine or nicotine-containing products for at least 3 months
- Has been diagnosed with ITP at least 3 months prior
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- Female ITP participants must be non-pregnant, non-breast feeding, and either of 1) non-childbearing potential or 2) must have serum beta human chorionic gonadotropin (HCG) level consistent with a non-pregnant state, and agree to use acceptable contraception from pretrial period until 84 days postdose
- Has a BMI <=36 kg/m^2
- Has been judged to be in good health, other than ITP diagnosis, based on medical history, physical examination, vital sign measurements, ECG, and laboratory safety tests
Who cannot join
- Has a history or clinically significant endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, genitourinary, or major neurological abnormalities or diseases
- Has a history of cancer (malignancy)
- Has a history of significant multiple and/or severe allergies or has had an anaphylactic reaction or significant intolerability to prescription or non-prescription drugs or food
- Is positive for hepatitis B surface antigen, hepatitis C antibodies, or human immunodeficiency virus (HIV)
- Has had major surgery or donated or lost 1 unit of blood in the 4 weeks prior
- Has participated in another investigational trial within 4 weeks (12 weeks for biologics)
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- Has received a live virus vaccination within 42 days or plans to receive such while participating in the trial
- Is unable to refrain from or anticipates the use of any medication, including prescription and non-prescription drugs and herbal remedies from 2 weeks prior and for the duration of the trial
- Consumes greater than 3 glasses of alcoholic beverages per day
- Consumes greater than 6 servings of caffeine-containing beverages per day
- Is currently a regular user of any illicit drugs or has a history of drug and/or alcohol abuse within 3 months
- Has a history of ITP or other autoimmune disease
- Has an active infection that is clinically significant
- Has a comorbid and significant hematological or immunological disorder
- Has a history of significant multiple and/or severe allergies or has had an anaphylactic reaction or significant intolerability to prescription or non-prescription drugs or food
- Is positive for hepatitis B surface antigen, hepatitis C antibodies, or HIV
- Has had major surgery or donated or lost 1 unit of blood within 4 weeks
- Has participated in another investigational trial within 4 weeks (12 weeks for biologics), excluding prior participation in the current study
- Has a history of ITP unresponsive to intravenous immunoglobulin (IVIG)
- Has had systemic corticosteroid use within 1 month (with the exception of stable low dose oral corticosteroids)
- Has had systemic IVIG or other systemic immunomodulatory therapy, excluding MK-8723 administration in the current study, within 3 months
- Has received a thrombopoietin receptor antagonist within 3 months
- Is unable to refrain from using thrombopoietin receptor agonists and/or systemic immune modulatory medications throughout the study
- Has received a live virus vaccine within 42 days prior or plans to receive such during the trial
- Consumes greater than 3 alcoholic beverages per day
- Consumes greater than 6 servings of caffeine-containing beverages per day
- Is currently a regular user of any illicit drugs or has a history of drug and/or alcohol abuse within 3 months
- Has clinical evidence of bleeding or coagulopathy including petechial rash, easy bruising, or excessive gingival bleeding with routine dental hygiene
- Has an active infection that is clinically significant
About this study
Official title: A Two-Part, Single Rising Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of MK-8723 in Healthy Adults and Patients With Immune Thrombocytopenia Purpura
The primary objectives of this study are to assess the safety and tolerability of single rising doses of MK-8723 in healthy adult participants and adult participants with chronic immune thrombocytopenia purpura (ITP) and to assess pharmacodynamics of MK-8723 in participants with ITP. The primary hypothesis is that the true placebo-adjusted platelet response rate to MK-8723 in adult patients with chronic ITP is >50%.
Read the detailed description
In Part 1 of the trial, safety and pharmacokinetics of MK-8723 will be evaluated in healthy participants. In Part 2 of the trial, safety, pharmacokinetics, and pharmacodynamics will be evaluated among participants with ITP. In Part 1, dose escalation will occur in up to 5 serial panels of participants; each participant will receive a single intravenous (IV) dose of MK-8723 (or placebo). In Part 2, dose escalation will occur in up to 3 serial panels of participants with ITP; each participant will receive a single IV dose of MK-8723 (or placebo), once safety and tolerability of the corresponding dose is shown in Part 1. Amendment 3 specified a re-enrollment procedure for eligible participants in Part 2 to participate in more than one dosing panel.
- Conditions
- Immune Thrombocytopenia Purpura
- Being tested
- MK-8723, Matching Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MK-8723Drug
MK-8723 administered as a single IV infusion over approximately 4 hours on Day 1.
Matching PlaceboDrug
Matching placebo to MK-8723 administered as a single IV infusion over approximately 4 hours on Day 1.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: MK-8723 1 mg/kg in Healthy Participants | Experimental | MK-8723 1 mg/kg administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: MK-8723 |
| Part 1: MK-8723 3 mg/kg in Healthy Participants | Experimental | MK-8723 3 mg/kg administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: MK-8723 |
| Part 1: MK-8723 10 mg/kg in Healthy Participants | Experimental | MK-8723 10 mg/kg administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: MK-8723 |
| Part 1: MK-8723 30 mg/kg in Healthy Participants | Experimental | MK-8723 30 mg/kg administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: MK-8723 |
| Part 1: MK-8723 100 mg/kg in Healthy Participants | Experimental | MK-8723 100 mg/kg administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: MK-8723 |
| Part 1: Matching Placebo to MK-8723 | Placebo comparator | Matching placebo to MK-8723 administered as a single IV infusion to healthy participants in Part 1. Receives: Drug: Matching Placebo |
| Part 2: MK-8723 10 mg/kg in ITP Participants | Experimental | MK-8723 10 mg/kg administered as a single IV infusion to participants with ITP in Part 2. Receives: Drug: MK-8723 |
| Part 2: MK-8723 30 mg/kg in ITP Participants | Experimental | MK-8723 30 mg/kg administered as a single IV infusion to participants with ITP in Part 2. Receives: Drug: MK-8723 |
| Part 2: MK-8723 100 mg/kg in ITP Participants | Placebo comparator | MK-8723 100 mg/kg administered as a single IV infusion to participants with ITP in Part 2. Receives: Drug: MK-8723 |
| Part 2: Matching Placebo to MK-8723 | Placebo comparator | Matching placebo to MK-8723 administered as a single IV infusion to participants with ITP in Part 2. Receives: Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing an Adverse Event
An AE is defined as any unfavorable and unintended medical occurrence in a clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Measured: Up to 84 days
Number of Participants Discontinuing Study Due to an Adverse Event (AE)
An AE is defined as any unfavorable and unintended medical occurrence in a clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Measured: Up to 84 Days
Number of Participants With a Positive Platelet Response to MK-8723
In participants with ITP, platelet response is a rapid, sensitive, and highly qualitative measure of response to anti-inflammatory therapy. A positive platelet response was defined as: 1) A doubling of platelet counts at the time point of maximum response (through Day 14) as compared to Day 0 AND an increase to an absolute level of ≥50,000/μL in participants with a baseline platelet count of <50,000/μL, OR 2) A 50% increase in the platelet count at the time point of maximum response (through Day 14) as compared to Day 0 in participants with a baseline platelet count of ≥50,000/μL. The analysis was specified only for participants with ITP (Part 2) that received treatment with MK-8723 or matching placebo.
Measured: Up to Day 14
Secondary outcomes (2)
Area Under the Concentration-time Curve of MK-8723 From Time 0 to Infinity (AUC0-∞) Among Healthy Participants and Participants With ITP
AUC0-∞ is a measure of total body exposure to drug. Serum samples for determination of AUC0-∞ were collected at pre-specified time-points.
Measured: All dose groups: Predose and 4 (end of infusion), 6, 12, 24 hrs postdose and Days 3, 4, 5, 7, 10, 14, 21, 28; 30 mg/kg and 100 mg/kg dose groups: Days 43, 56, 71, 84
Maximum Concentration (Cmax) of MK-8723 Among Healthy Participants and Participants With ITP
Serum samples for determination of Cmax were collected at pre-specified time-points.
Measured: All dose groups: Predose and 4 (end of infusion), 6, 12, 24 hrs postdose and Days 3, 4, 5, 7, 10, 14, 21, 28; 30 mg/kg and 100 mg/kg dose groups: Days 43, 56, 71, 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2013 (estimated)
- Study start31 Oct 2013
- Primary completion26 Apr 2015
- Study completion26 Apr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immune Thrombocytopenia PurpuraStudies of the same condition, open and under way.
- More trials run by Merck Sharp & Dohme LLCThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck Sharp & Dohme LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01963260
- Sponsor's protocol ID
- 8723-001
- Status last verified
- Feb 2019
- First submitted
- 11 Oct 2013
- Last updated
- 15 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Yes: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
Study at a glance
From the registry record, last updated by the sponsor on 15 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Part, Single Rising Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of MK-8723 in Healthy Adults and Patients With Immune Thrombocytopenia Purpura
- ClinicalTrials.gov ID
- NCT01963260
- Acronym / sponsor ID
- 8723-001
- Conditions
- Immune Thrombocytopenia Purpura
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Merck Sharp & Dohme LLCIndustry (pharma, biotech, devices)
- Study start
- 31 Oct 2013 (actual)
- Primary completion
- 26 Apr 2015 (actual)
- Study completion
- 26 Apr 2015 (actual)
- First posted
- 16 Oct 2013 (estimated)
- Results
- Posted 21 Mar 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objectives of this study are to assess the safety and tolerability of single rising doses of MK-8723 in healthy adult participants and adult participants with chronic immune thrombocytopenia purpura (ITP) and to assess pharmacodynamics of MK-8723 in participants with ITP. The primary hypothesis is that the true placebo-adjusted platelet response rate to MK-8723 in adult patients with chronic ITP is >50%.
Detailed description
In Part 1 of the trial, safety and pharmacokinetics of MK-8723 will be evaluated in healthy participants. In Part 2 of the trial, safety, pharmacokinetics, and pharmacodynamics will be evaluated among participants with ITP. In Part 1, dose escalation will occur in up to 5 serial panels of participants; each participant will receive a single intravenous (IV) dose of MK-8723 (or placebo). In Part 2, dose escalation will occur in up to 3 serial panels of participants with ITP; each participant will receive a single IV dose of MK-8723 (or placebo), once safety and tolerability of the corresponding dose is shown in Part 1. Amendment 3 specified a re-enrollment procedure for eligible participants in Part 2 to participate in more than one dosing panel.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 10
| Arm | Type | Receives |
|---|---|---|
| Part 1: MK-8723 1 mg/kg in Healthy Participants | Experimental | Drug: MK-8723MK-8723 1 mg/kg administered as a single IV infusion to healthy participants in Part 1. |
| Part 1: MK-8723 3 mg/kg in Healthy Participants | Experimental | Drug: MK-8723MK-8723 3 mg/kg administered as a single IV infusion to healthy participants in Part 1. |
| Part 1: MK-8723 10 mg/kg in Healthy Participants | Experimental | Drug: MK-8723MK-8723 10 mg/kg administered as a single IV infusion to healthy participants in Part 1. |
| Part 1: MK-8723 30 mg/kg in Healthy Participants | Experimental | Drug: MK-8723MK-8723 30 mg/kg administered as a single IV infusion to healthy participants in Part 1. |
| Part 1: MK-8723 100 mg/kg in Healthy Participants | Experimental | Drug: MK-8723MK-8723 100 mg/kg administered as a single IV infusion to healthy participants in Part 1. |
| Part 1: Matching Placebo to MK-8723 | Placebo comparator | Drug: Matching PlaceboMatching placebo to MK-8723 administered as a single IV infusion to healthy participants in Part 1. |
| Part 2: MK-8723 10 mg/kg in ITP Participants | Experimental | Drug: MK-8723MK-8723 10 mg/kg administered as a single IV infusion to participants with ITP in Part 2. |
| Part 2: MK-8723 30 mg/kg in ITP Participants | Experimental | Drug: MK-8723MK-8723 30 mg/kg administered as a single IV infusion to participants with ITP in Part 2. |
| Part 2: MK-8723 100 mg/kg in ITP Participants | Placebo comparator | Drug: MK-8723MK-8723 100 mg/kg administered as a single IV infusion to participants with ITP in Part 2. |
| Part 2: Matching Placebo to MK-8723 | Placebo comparator | Drug: Matching PlaceboMatching placebo to MK-8723 administered as a single IV infusion to participants with ITP in Part 2. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Female participants must be non-pregnant, non-breast feeding, and of non-childbearing potential
- Has a Body Mass Index (BMI) <=32 kg/m^2
- Has a body weight >= 50 kg and <= 100 kg
- Has been judged to be in good health based on medical history, physical examination, vital sign measurements, electrocardiogram (ECG), and laboratory safety tests
- Non-smoker or has not used nicotine or nicotine-containing products for at least 3 months
Key exclusion criteria (29 listed)
- Has a history or clinically significant endocrine, gastrointestinal, cardiovascular, hematological, hepatic, immunological, renal, respiratory, genitourinary, or major neurological abnormalities or diseases
- Has a history of cancer (malignancy)
- Has a history of significant multiple and/or severe allergies or has had an anaphylactic reaction or significant intolerability to prescription or non-prescription drugs or food
- Is positive for hepatitis B surface antigen, hepatitis C antibodies, or human immunodeficiency virus (HIV)
- Has had major surgery or donated or lost 1 unit of blood in the 4 weeks prior
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 |
|---|---|---|
| MK-8723MK-8723 administered as a single IV infusion over approximately 4 hours on Day 1. | Drug | Part 1: MK-8723 1 mg/kg in Healthy Participants, Part 1: MK-8723 10 mg/kg in Healthy Participants, Part 1: MK-8723 100 mg/kg in Healthy Participants, Part 1: MK-8723 3 mg/kg in Healthy Participants, Part 1: MK-8723 30 mg/kg in Healthy Participants, Part 2: MK-8723 10 mg/kg in ITP Participants, Part 2: MK-8723 100 mg/kg in ITP Participants, Part 2: MK-8723 30 mg/kg in ITP Participants |
| Matching PlaceboMatching placebo to MK-8723 administered as a single IV infusion over approximately 4 hours on Day 1. | Drug | Part 1: Matching Placebo to MK-8723, Part 2: Matching Placebo to MK-8723 |
Primary outcome
Number of Participants Experiencing an Adverse Event
Time frame: Up to 84 days
An AE is defined as any unfavorable and unintended medical occurrence in a clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Number of Participants Discontinuing Study Due to an Adverse Event (AE)
Time frame: Up to 84 Days
An AE is defined as any unfavorable and unintended medical occurrence in a clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Number of Participants With a Positive Platelet Response to MK-8723
Time frame: Up to Day 14
In participants with ITP, platelet response is a rapid, sensitive, and highly qualitative measure of response to anti-inflammatory therapy. A positive platelet response was defined as: 1) A doubling of platelet counts at the time point of maximum response (through Day 14) as compared to Day 0 AND an increase to an absolute level of ≥50,000/μL in participants with a baseline platelet count of <50,000/μL, OR 2) A 50% increase in the platelet count at the time point of maximum response (through Day 14) as compared to Day 0 in participants with a baseline platelet count of ≥50,000/μL. The analysis was specified only for participants with ITP (Part 2) that received treatment with MK-8723 or matching placebo.
2 secondary outcomes
Area Under the Concentration-time Curve of MK-8723 From Time 0 to Infinity (AUC0-∞) Among Healthy Participants and Participants With ITP
Time frame: All dose groups: Predose and 4 (end of infusion), 6, 12, 24 hrs postdose and Days 3, 4, 5, 7, 10, 14, 21, 28; 30 mg/kg and 100 mg/kg dose groups: Days 43, 56, 71, 84
Maximum Concentration (Cmax) of MK-8723 Among Healthy Participants and Participants With ITP
Time frame: All dose groups: Predose and 4 (end of infusion), 6, 12, 24 hrs postdose and Days 3, 4, 5, 7, 10, 14, 21, 28; 30 mg/kg and 100 mg/kg dose groups: Days 43, 56, 71, 84
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
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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.
Medical Director study director
Merck Sharp & Dohme LLC
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MK-8723 in people with Immune Thrombocytopenia Purpura, run by Merck Sharp & Dohme LLC.
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 9 inclusion and 29 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 15 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 21 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants Experiencing an Adverse Event (time frame: Up to 84 days). The study lists 3 primary outcomes in all. 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 15 Mar 2019.