Immune Thrombocytopenic Purpura (ITP)Phase 1/2NCT03866577

Safety and Tolerability of M254 in Healthy Volunteers and Immune Thrombocytopenic Purpura (ITP) Patients

A Phase 1/2 study testing Biological: M254 and Intravenous immunoglobulin (IVIg) in people with Immune Thrombocytopenic Purpura (ITP), run by Momenta Pharmaceuticals, Inc. at 28 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

The purpose of this study is to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 after administration of a single ascending dose and repeat doses in healthy volunteers and immune thrombocytopenic purpura (ITP) patients. Read more about this study

Who is it for?: Ages 18 years and olderHealthy volunteers can take part
Conditions the study is for: Immune Thrombocytopenic Purpura (ITP)
What is being tested?: Biological: M254 and Intravenous immunoglobulin (IVIg)
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 50 participants
Where: 28 sitesin 7 countries
Study dates: 21 Dec 2018to 9 Jun 2021
Phase: Phase 1/2
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 years and older
All sexes
Healthy volunteers accepted
Adult, Older adult

Key Criteria for Healthy Volunteers: Subject must be between the ages of 18 and 55 years; healthy as indicated by medical history, physical examination, vital signs, clinical laboratory tests, and 12-lead electrocardiogram, and all abnormal findings are assessed as not clinically significant by the Investigator; not pregnant or breastfeeding; and no other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate.

Key Criteria for Immune Thrombocytopenic Purpura (ITP) Patients: Patient must be aged ≥18 years and diagnosed with ITP at least 3 months prior to screening, stable maintenance therapy for at least 4 weeks prior to the first study visit, not pregnant or breastfeeding, and no other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate.

Trial locations and contacts

28 sites in 7 countries.

Country

City, postcode or hospital

28 sites in 7 countries

United States6 sites

Taussig Cancer Insititute Cleveland Clinic
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27710
University of Southern California
Los Angeles, California, 90089
Lakes Research
Miami Lakes, Florida, 33014
University of South Florida
St. Petersburg, Florida, 33701
Oncology Institute of Hope and Innovation
Whittier, California, 90603

Other trials in United States

Spain6 sites

Hosp Univ Vall D Hebron
Barcelona, 08035
Hosp. Univ. de Burgos
Burgos, 09003
Hosp. Regional. Carlos Haya
Málaga, 29010
Hosp. Gral. Univ. J.M. Morales Meseguer
Murcia, 30008
Hosp Clinico Univ de Salamanca
Salamanca, 37007
Hosp. Univ. Dr. Peset
Valencia, 46017

Other trials in Spain

Italy5 sites

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
Meldola, 47014
Azienda Unita Sanitaria Locale di Ravenna
Ravenna, 48100
Arcispedale Santa Maria Nuova - IRCCS
Reggio Emilia, 42123
Policlinico Universitario Agostino Gemelli
Roma, 168
Ospedale 'Casa Sollievo della Sofferenza' - U.O. Ematologia-
San Giovanni Rotondo, 71013

Other trials in Italy

Poland5 sites

Silesian Healthy Blood Clinic
Chorzów, 41 503
Samodzielny Publiczny Szpital Kliniczny Nr 1
Lublin, 20-081
Szpital Wojewodzki w Opolu
Opole, 45-061
Samodzielny Publiczny Zaklad Opieki Zdrowotnej MSW w Poznaniu im prof Ludwika Bierkowskiego
Poznan, 60-631
Uniwersytecki Szpital Kliniczny im Jana Mikulicza Radeckiego we Wroclawiu
Wroclaw, 50 367

Other trials in Poland

Hungary3 sites

Debreceni Egyetem Klinikai Kozpont
Debrecen, 4032
Somogy Megyei Kaposi Mor Oktato Korhaz
Kaposvár, 7400
Pecsi Tudomanyegyetem
Pécs, 7624

Other trials in Hungary

Netherlands2 sites

Universitair Medisch Centrum Groningen
Groningen, 9713 GZ
PRA Health Sciences
Groningen, NZ 9728

Other trials in Netherlands

Belgium1 site

Ucl de Mont-Godinne
Yvoir, 5530

Other trials in Belgium

About this study

Official title: A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura

Read the detailed description

The Part A of the study is currently not accepting healthy volunteers as the recruitment for the part A has completed.

Conditions: Immune Thrombocytopenic Purpura (ITP)
Being tested: Biological: M254, Placebo, Intravenous immunoglobulin (IVIg)
Keywords: Immune thrombocytopenic purpura, Healthy subjects, M254, Intravenous immunoglobulin (IVIg)
Medical subject headings: Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Biological: M254Biological
M254 administered as intravenous infusion
PlaceboDrug
Placebo administered as intravenous infusion
Intravenous immunoglobulin (IVIg)Biological
IVIg administered as intravenous infusion

Study groups in NCT03866577
Study group	Role	What they receive
Part A	Experimental	Healthy volunteers will receive a single ascending dose of M254 or placebo Receives: Biological: Biological: M254; Drug: Placebo
Part B	Experimental	Immune thrombocytopenic purpura (ITP) patients will receive a single ascending dose of M254 followed by IVIg Receives: Biological: Biological: M254; Biological: Intravenous immunoglobulin (IVIg)
Part C	Experimental	ITP patients will receive a single dose of M254 or IVIg, followed by a single dose of the other drug approximately 28 days later Receives: Biological: Biological: M254; Biological: Intravenous immunoglobulin (IVIg)
Part D	Experimental	ITP patients will receive repeated doses of M254 Receives: Biological: Biological: M254

What is being measured

The primary outcome is the main question the study is designed to answer.

Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity
An AE is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A TEAE was defined as any event not present prior to administration of the study drug or any event already present that worsened in either severity or frequency following exposure to the study drug. Severity was graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening, and Grade 5= Death related to adverse event. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. For Parts B and C, treatment period wise AE data were presented.
Measured: From Day 1 up to Day 29
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values
Number of participants with clinically significant laboratory abnormalities (chemistry, hematology, urinalysis and coagulation) were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Measured: From Day 1 up to Day 29
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Vital Signs
Number of participants with clinically significant abnormalities in vital signs (blood pressure [systolic blood pressure {SBP} and diastolic blood pressure {DBP}], pulse rate, respiratory rate, and body temperature) were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Measured: From Day 1 up to Day 29
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Electrocardiograms (ECGs)
Number of participants with clinically significant abnormalities in ECGs were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Measured: From Day 1 up to Day 29
Part C: Maximum Observed Response of M254 (Rmax) on Platelet Count
Rmax is defined as the maximum observed response of M254 on platelet count. Baseline was the pre-dose sample. Data was planned to be collected and analyzed Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Measured: Predose (baseline) up to Day 29 post dose
Part C: Change From Baseline in Rmax of M254 in Platelet Count
Change from baseline in Rmax of M254 in platelet count was reported. Rmax is defined as the maximum observed response of M254. Baseline was the predose sample. Therapeutic platelet count was defined as >=50*10^9 cells/L. Platelet response of >=20*10^9 cells/L was considered as increase from baseline. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Measured: Predose (baseline) up to Day 29 post dose
Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 14 (AUEC[0-Day 14]) of M254
AUEC(0-Day 14) is defined as the area under effect curve of the change from baseline in platelet count from Day 0 to Day 14 of M254. Baseline was the predose sample. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Measured: Predose (baseline) up to Day 14 post dose
Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 28 (AUEC[0-Day 28]) of M254
AUEC(0-Day 28) is defined as the area under effect curve of the change from baseline in platelet count from Day 0 to Day 28 of M254. Baseline was the predose sample. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) the participants received.
Measured: Predose (baseline) up to Day 29 post dose

Secondary outcomes (10)

Part C: Number of Participants With Overall Platelet Response After M254 Administration Compared to IVIg
Number of participants with overall platelet response after M254 administration compared to IVIg were reported. Overall platelet response rate is defined as reaching the therapeutic platelet count. A therapeutic platelet count is defined as greater than or equal to (>=) 50*10^9 cells/liter (L) and an increase from baseline of >=20*10^9 cells/L. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) the participants received.
Measured: Up to Day 29
Parts A, B, and C: Maximum Observed Plasma Concentration (Cmax) of M254
Cmax is defined as the maximum observed plasma concentration of M254. Baseline was the predose sample. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Time to Reach Maximum Observed Plasma Concentration (Tmax) of M254
Tmax is defined as the time to reach the maximum observed plasma concentration of M254. Baseline was the predose sample. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Infinite Time (AUC[0-Infinity]) of M254
AUC(0-Infinity) is defined as area under the plasma concentration-time curve from time 0 to infinite time of M254. Baseline was the predose sample. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Time of Last Quantifiable Concentration (AUC[0-Last]) of M254
AUC(0-last) is defined as area under the plasma concentration-time curve from time 0 to time of the last quantifiable concentration of M254. AUC(0-last) is calculated by linear-linear trapezoidal summation. Baseline was the predose sample. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Apparent Terminal Phase Half-life (t1/2) of M254
t1/2 is defined as the time measured for the plasma concentration to decrease by 1 half to its original concentration of M254. Baseline was the predose sample. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Volume of Distribution (Vz) of M254
Vz is defined as volume of distribution of M254 at terminal phase. Baseline was the predose sample. Data for this outcome measure was not planned for pooled placebo arm in Part A and thus no data was presented for placebo arm in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Clearance (CL) of M254
CL is defined as clearance of M254, calculated as dose/AUC(0-infinity). Baseline was the predose sample. Data for this outcome measure was not planned for pooled placebo arm in Part A and thus no data was presented for placebo arm in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Mean Residence Time (MRT) of M254
Mean residence time is the average time that drug dose remained in the body. Baseline was the predose sample. Data for this outcome measure was not planned for pooled placebo arm in Part A and thus no data was presented for placebo arm in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Percentage of the Estimated Part for the Calculation of AUC(0-infinity) (%AUCextra) of M254
Percentage of the estimated part for the calculation of AUC(0-infinity) (%AUCextra) of M254 were reported. Baseline was the predose sample. Data for this outcome measure was not planned for pooled placebo arm in Part A and thus no data was presented for placebo arm in Part A. In Part C, combined data of both arms of Group 1 is presented as planned.
Measured: Predose (baseline) up to Day 29 post dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Dec 20189 Jun 2021

First posted7 Mar 2019
Study start21 Dec 2018
Primary completion9 Jun 2021
Study completion9 Jun 2021

Why it stopped: Study was terminated based on Sponsor decision.

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 Thrombocytopenic Purpura (ITP)Studies 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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03866577
Sponsor's protocol ID: CR108979
Other IDs: 2018-003534-32, MOM-M254-001, 2018
Status last verified: May 2025
First submitted: 12 Feb 2019
Last updated: 28 May 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Immune Thrombocytopenic Purpura (ITP) trials

Study at a glance

From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 4-part Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of M254 in Healthy Volunteers and in Patients With Immune Thrombocytopenic Purpura
ClinicalTrials.gov ID: NCT03866577
Acronym / sponsor ID: CR108979
Conditions: Immune Thrombocytopenic Purpura (ITP)
Keywords: Immune thrombocytopenic purpura, Healthy subjects, M254, Intravenous immunoglobulin (IVIg)
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Study was terminated based on Sponsor decision.
Enrolment: 50 participants (actual)
Lead sponsor: Momenta Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
Study start: 21 Dec 2018 (actual)
Primary completion: 9 Jun 2021 (actual)
Study completion: 9 Jun 2021 (actual)
First posted: 7 Mar 2019 (actual)
Results: Posted 1 Jul 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The Part A of the study is currently not accepting healthy volunteers as the recruitment for the part A has completed.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 4

Arm	Type	Receives
Part A	Experimental	Biological: Biological: M254, Drug: PlaceboHealthy volunteers will receive a single ascending dose of M254 or placebo
Part B	Experimental	Biological: Biological: M254, Biological: Intravenous immunoglobulin (IVIg)Immune thrombocytopenic purpura (ITP) patients will receive a single ascending dose of M254 followed by IVIg
Part C	Experimental	Biological: Biological: M254, Biological: Intravenous immunoglobulin (IVIg)ITP patients will receive a single dose of M254 or IVIg, followed by a single dose of the other drug approximately 28 days later
Part D	Experimental	Biological: Biological: M254ITP patients will receive repeated doses of M254

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

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
Biological: M254M254 administered as intravenous infusion	Biological	Part A, Part B, Part C, Part D
PlaceboPlacebo administered as intravenous infusion	Drug	Part A
Intravenous immunoglobulin (IVIg)IVIg administered as intravenous infusion	Biological	Part B, Part C

Primary outcome

Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity
Time frame: From Day 1 up to Day 29
An AE is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product. A TEAE was defined as any event not present prior to administration of the study drug or any event already present that worsened in either severity or frequency following exposure to the study drug. Severity was graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening, and Grade 5= Death related to adverse event. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A. For Parts B and C, treatment period wise AE data were presented.
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values
Time frame: From Day 1 up to Day 29
Number of participants with clinically significant laboratory abnormalities (chemistry, hematology, urinalysis and coagulation) were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Vital Signs
Time frame: From Day 1 up to Day 29
Number of participants with clinically significant abnormalities in vital signs (blood pressure [systolic blood pressure {SBP} and diastolic blood pressure {DBP}], pulse rate, respiratory rate, and body temperature) were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Electrocardiograms (ECGs)
Time frame: From Day 1 up to Day 29
Number of participants with clinically significant abnormalities in ECGs were reported. Data was planned to be collected and analyzed on pooled population of participants who received placebo in Part A.
Part C: Maximum Observed Response of M254 (Rmax) on Platelet Count
Time frame: Predose (baseline) up to Day 29 post dose
Rmax is defined as the maximum observed response of M254 on platelet count. Baseline was the pre-dose sample. Data was planned to be collected and analyzed Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Part C: Change From Baseline in Rmax of M254 in Platelet Count
Time frame: Predose (baseline) up to Day 29 post dose
Change from baseline in Rmax of M254 in platelet count was reported. Rmax is defined as the maximum observed response of M254. Baseline was the predose sample. Therapeutic platelet count was defined as >=50*10^9 cells/L. Platelet response of >=20*10^9 cells/L was considered as increase from baseline. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 14 (AUEC[0-Day 14]) of M254
Time frame: Predose (baseline) up to Day 14 post dose
AUEC(0-Day 14) is defined as the area under effect curve of the change from baseline in platelet count from Day 0 to Day 14 of M254. Baseline was the predose sample. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) which the participants received.
Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 28 (AUEC[0-Day 28]) of M254
Time frame: Predose (baseline) up to Day 29 post dose
AUEC(0-Day 28) is defined as the area under effect curve of the change from baseline in platelet count from Day 0 to Day 28 of M254. Baseline was the predose sample. Data was planned to be collected and analyzed for Part C only. For Part C, data were collected and analyzed on the pooled population per the study drug (M254 120 mg/kg and IVIg 1000 mg/kg) the participants received.

10 secondary outcomes

Part C: Number of Participants With Overall Platelet Response After M254 Administration Compared to IVIg
Time frame: Up to Day 29
Parts A, B, and C: Maximum Observed Plasma Concentration (Cmax) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Time to Reach Maximum Observed Plasma Concentration (Tmax) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Infinite Time (AUC[0-Infinity]) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Time of Last Quantifiable Concentration (AUC[0-Last]) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Apparent Terminal Phase Half-life (t1/2) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Volume of Distribution (Vz) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Clearance (CL) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Mean Residence Time (MRT) of M254
Time frame: Predose (baseline) up to Day 29 post dose
Parts A, B, and C: Percentage of the Estimated Part for the Calculation of AUC(0-infinity) (%AUCextra) of M254
Time frame: Predose (baseline) up to Day 29 post dose

Results and safety

Results posted to the registry on 1 Jul 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part A: Pooled Placebo	7	7
Part A: M254 3 Milligrams/Kilogram (mg/kg)	3	3
Part A: M254 10 mg/kg	3	3
Part A: M254 30 mg/kg	3	3
Part A: M254 60 mg/kg	3	3
Part A: M254 120 mg/kg	3	3
Part A: M254 250 mg/kg	3	3
Part B: M254 20 mg/kg and IV Immunoglobulin (IVIg) 1000 mg/kg	2	2
Part B: M254 60 mg/kg and IVIg 1000 mg/kg	5	4
Part B: M254 120 mg/kg and IVIg 1000 mg/kg	5	3
Part B: M254 250 mg/kg and IVIg 1000 mg/kg	2	2
Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg	6	5
Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg	5	5

Primary outcome: Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity

Time frame: From Day 1 up to Day 29. Count of participants. Unit: Participants

Measure	Part A: Pooled Placebo n = 7	Part A: M254 3 Milligrams/Kilogram (mg/kg) n = 3	Part A: M254 10 mg/kg n = 3	Part A: M254 30 mg/kg n = 3	Part A: M254 60 mg/kg n = 3	Part A: M254 120 mg/kg n = 3	Part A: M254 250 mg/kg n = 3	Part B: M254 20 mg/kg - M254 Period n = 2	Part B: M254 20 mg/kg - IVIg Period n = 2	Part B: M254 60 mg/kg - M254 Period n = 5	Part B: M254 60 mg/kg - IVIg Period n = 4	Part B: M254 120 mg/kg - M254 Period n = 5	Part B: M254 120 mg/kg - IVIg Period n = 3	Part B: M254 250 mg/kg - M254 Period n = 2	Part B: M254 250 mg/kg - IVIg Period n = 2	Part C (Group 1) : M254 120 mg/kg and IVIg 1000 mg/kg - M254 Period n = 6	Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg - IVIg Period n = 5	Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg - IVIg Period n = 5	Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg - M254 Period n = 5
Value	6	3	3	3	0	3	3	1	0	2	2	1	1	0	0	1	0	2	2

Population analysed: Safety analysis set included all participants who received at least 1 dose of M254 or IVIg or placebo.

Primary outcome: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values

Time frame: From Day 1 up to Day 29. Count of participants. Unit: Participants

Measure	Part A: Pooled Placebo n = 7	Part A: M254 3 Milligrams/Kilogram (mg/kg) n = 3	Part A: M254 10 mg/kg n = 3	Part A: M254 30 mg/kg n = 3	Part A: M254 60 mg/kg n = 3	Part A: M254 120 mg/kg n = 3	Part A: M254 250 mg/kg n = 3	Part B: M254 20 mg/kg and IV Immunoglobulin (IVIg) 1000 mg/kg n = 2	Part B: M254 60 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 120 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 250 mg/kg and IVIg 1000 mg/kg n = 2	Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg n = 6	Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg n = 5
Value	0	0	0	0	0	0	0	0	0	0	0	0	1

Population analysed: Safety analysis set included all participants who received at least 1 dose of M254 or IVIg or placebo.

Primary outcome: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Vital Signs

Time frame: From Day 1 up to Day 29. Count of participants. Unit: Participants

Measure	Part A: Pooled Placebo n = 7	Part A: M254 3 Milligrams/Kilogram (mg/kg) n = 3	Part A: M254 10 mg/kg n = 3	Part A: M254 30 mg/kg n = 3	Part A: M254 60 mg/kg n = 3	Part A: M254 120 mg/kg n = 3	Part A: M254 250 mg/kg n = 3	Part B: M254 20 mg/kg and IV Immunoglobulin (IVIg) 1000 mg/kg n = 2	Part B: M254 60 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 120 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 250 mg/kg and IVIg 1000 mg/kg n = 2	Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg n = 6	Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg n = 5
Value	0	0	0	0	0	0	1	0	0	0	0	0	1

Population analysed: Safety analysis set included all participants who received at least 1 dose of M254 or IVIg or placebo.

Primary outcome: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Electrocardiograms (ECGs)

Time frame: From Day 1 up to Day 29. Count of participants. Unit: Participants

Measure	Part A: Pooled Placebo n = 7	Part A: M254 3 Milligrams/Kilogram (mg/kg) n = 3	Part A: M254 10 mg/kg n = 3	Part A: M254 30 mg/kg n = 3	Part A: M254 60 mg/kg n = 3	Part A: M254 120 mg/kg n = 3	Part A: M254 250 mg/kg n = 3	Part B: M254 20 mg/kg and IV Immunoglobulin (IVIg) 1000 mg/kg n = 2	Part B: M254 60 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 120 mg/kg and IVIg 1000 mg/kg n = 5	Part B: M254 250 mg/kg and IVIg 1000 mg/kg n = 2	Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg n = 6	Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg n = 5
Value	0	0	0	0	0	0	0	0	0	0	0	0	0

Population analysed: Safety analysis set included all participants who received at least 1 dose of M254 or IVIg or placebo.

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: From Day 1 up to Day 29. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part A: Pooled Placebo	0 of 7 (0%)	6 of 7 (85.7%)	0 of 7
Part A: M254 3 Milligrams/Kilogram (mg/kg)	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A: M254 10 mg/kg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A: M254 30 mg/kg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A: M254 60 mg/kg	0 of 3 (0%)	0 of 3 (0%)	0 of 3
Part A: M254 120 mg/kg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part A: M254 250 mg/kg	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Part B: M254 20 mg/kg - M254 Period	0 of 2 (0%)	1 of 2 (50%)	0 of 2
Part B: M254 20 mg/kg - IVIg Period	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Part B: M254 60 mg/kg - M254 Period	0 of 5 (0%)	2 of 5 (40%)	0 of 5
Part B: M254 60 mg/kg - IVIg Period	0 of 4 (0%)	2 of 4 (50%)	0 of 4
Part B: M254 120 mg/kg - M254 Period	0 of 5 (0%)	1 of 5 (20%)	0 of 5
Part B: M254 120 mg/kg - IVIg Period	0 of 3 (0%)	1 of 3 (33.3%)	0 of 3
Part B: M254 250 mg/kg - M254 Period	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Part B: M254 250 mg/kg - IVIg Period	0 of 2 (0%)	0 of 2 (0%)	0 of 2
Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg - M254 Period	0 of 6 (0%)	1 of 6 (16.7%)	0 of 6
Part C (Group 1): M254 120 mg/kg and IVIg 1000 mg/kg - IVIg Period	0 of 5 (0%)	0 of 5 (0%)	0 of 5
Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg - IVIg Period	0 of 5 (0%)	2 of 5 (40%)	0 of 5
Part C (Group 1): IVIg 1000 mg/kg and M254 120 mg/kg - M254 Period

Limitations and caveats (from the sponsor): Part C (Group 2) and Part D were planned but not conducted based on sponsor decision to discontinue the study after completion of Part A, Part B, and Part C (Group 1). No safety concerns were identified and study was terminated for business reasons.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

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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.

Momenta General Queries study director

Momenta Pharmaceuticals, Inc.

28 sites in 7 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Biological: M254 and Intravenous immunoglobulin (IVIg) in people with Immune Thrombocytopenic Purpura (ITP), run by Momenta Pharmaceuticals, Inc. at 28 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: stopped early, as of the sponsor's last update on 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in United States, Spain, Italy, Poland, Hungary and 2 more countries. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 1 Jul 2024). 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?

Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity (time frame: From Day 1 up to Day 29). The study lists 8 primary outcomes in all. 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 NCT03866577.

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 28 May 2025.