| CTRI Number |
CTRI/2025/08/092723 [Registered on: 08/08/2025] Trial Registered Prospectively |
| Last Modified On: |
07/08/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Other |
|
Public Title of Study
|
A study to assess the safety due to the change in treatment of medicine from Emicizumab to Marstacimab and learn the change after the discontinuation of the medicine Emicizumab in people with the bleeding disease known as Hemophilia A Without Inhibitors.
|
|
Scientific Title of Study
|
A Phase 1b Open-label Non-randomized Study to Assess the Safety,
Pharmacokinetics, and Pharmacodynamics of Marstacimab Treatment Following the
Discontinuation of Emicizumab Therapy in Adolescent and Adult Participants With Severe Hemophilia A Without Inhibitors |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| B7841014 Protocol Amendment V.01 dated 2-Dec-2024 |
Protocol Number |
| NCT06703606 |
ClinicalTrials.gov |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
|
| Designation |
|
| Affiliation |
|
| Address |
|
| Phone |
|
| Fax |
|
| Email |
|
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Seema Pai |
| Designation |
Director Clinical Site Operations – India Cluster |
| Affiliation |
Pfizer Limited |
| Address |
The Capital, 1802-1901, Plot No. C-70, G Block, Bandra Kurla
Complex, Bandra East
Mumbai
MAHARASHTRA
400051
India |
| Phone |
02266932000 |
| Fax |
|
| Email |
Seema.Pai@pfizer.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Seema Pai |
| Designation |
Director Clinical Site Operations – India Cluster |
| Affiliation |
Pfizer Limited |
| Address |
The Capital, 1802-1901, Plot No. C-70, G Block, Bandra Kurla
Complex, Bandra East
MAHARASHTRA
400051
India |
| Phone |
02266932000 |
| Fax |
|
| Email |
Seema.Pai@pfizer.com |
|
|
Source of Monetary or Material Support
|
| Pfizer Inc. 66 Hudson Boulevard East New York, NY 10001 |
|
|
Primary Sponsor
|
| Name |
Pfizer Inc. |
| Address |
66 Hudson Boulevard East New York, NY 10001 |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
|
Details of Secondary Sponsor
|
| Name |
Address |
| Pfizer Limited |
The Capital, 1802/1901, Plot No. C-70 G Block,
Bandra Kurla Complex, Bandra (E), Mumbai City
(India) - 400051 |
|
|
Countries of Recruitment
|
Canada
India
United States of America |
|
Sites of Study
|
| No of Sites = 2 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Shrinath Manikrao Kshirsagar |
Clinical Trial Research Unit (CTRU), K J Somaiya Hospital & Research Centre |
Somaiya Ayurvihar Complex, Eastern Express Highway, Sion East Mumbai 4000022
Mumbai
MAHARASHTRA |
9821556030
shrinath@somaiya.edu |
| Dr Nirmalkumar Choraria |
Nirmal Hospital Pvt. Ltd |
Ring Road, Surat-395002
Surat
GUJARAT |
9825142549
drnirmalchoraria@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 2 |
| Name of Committee |
Approval Status |
| Institutional Ethics Committee K J Somaiya Medical College & Hospital, Mumbai |
Approved |
| Nirmal Hospital Pvt. Ltd |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D67||Hereditary factor IX deficiency, (2) ICD-10 Condition: D66||Hereditary factor VIII deficiency, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
Marstacimab |
Drug: Marstacimab 150 mg
Unit Dose Strength(s): 150 mg/mL
Dosage Level(s): 150 mg per week
Route of Administration: Subcutaneous(SC) injection; administered once weekly
Participants will receive weekly 150 mg Marstacimab SC once weekly for 17 weeks. |
| Comparator Agent |
Not Applicable |
Not Applicable |
|
|
Inclusion Criteria
|
| Age From |
12.00 Year(s) |
| Age To |
74.00 Year(s) |
| Gender |
Male |
| Details |
1. Male and 12 to less than 75 years of age with a minimum body weight of 35 kg at the time of signing the informed consent.
2. Diagnosis of severe hemophilia A (FVIII activity less than 1%) without inhibitors.
3. On emicizumab therapy at a standard clinical dose for more than or equal to 6 months |
|
| ExclusionCriteria |
| Details |
1. Previous or current treatment for or history of coronary artery diseases, venous or arterial thrombosis, or ischemic disease.
2. Any medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator s judgment, make the participant inappropriate for the study.
3. Known hemostatic defect other than hemophilia A
4. Current use of any prohibited concomitant medications or unwillingness or inability to use a required concomitant medication
5. Previous administration of an investigational product, drug or vaccine within 30 days or 5 halflives preceding the first dose of study intervention used in this study, whichever is longer. Participation in studies of other investigational products at any time during participation in this study
6. Platelet count less than 100,000per microL or hemoglobin less than 10 g per dL
7. Clinically significant renal or hepatic function abnormality based on laboratory results at screening,or known kidney or liver disease.
8. CD4 cell countless than or equal to 200per microL if HIV positive.
9. Screening 12 lead ECG that demonstrates clinically significant abnormalities that, in the opinion of the investigator, may affect participant safety or interpretation of study results
10. Known planned surgical procedure
11. Hypersensitivity or allergic reaction to hamster protein or other components of the study intervention
12. Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members |
|
|
Method of Generating Random Sequence
|
Other |
|
Method of Concealment
|
Other |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
1. Incidence of marstacimab- elated adverse events
2. Incidence of marstacimab-related serious AEs
|
1. Incidence of marstacimab- elated adverse events - Approximately 178 days: from the time the participant provides informed consent through and
including a minimum of 28 calendar days after last dose of study treatment.
2. Incidence of marstacimab-related serious AEs - Approximately 178 days: from the time the participant provides informed consent through and including a minimum of 28 calendar days after last dose of study treatment. |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Plasma Concentration Versus Time of Marstacimab (Listings) |
Up to day 135 |
| Maximum Observed Plasma Concentration (Cmax) of Marstacimab |
Up to day 135 |
| Average Plasma Concentration (Cavg) of Marstacimab |
Up to day 135 |
| Minimum Observed Plasma Concentration (Cmin) |
Up to day 135 |
| Change from baseline in tissue factor pathway inhibitor (TFPI). |
Up to day 135 |
| Change from baseline in prothrombin fragment 1+2 (PF 1+2) |
Up to day 135 |
| Change from baseline in D-dimer |
Upto Day 135 |
| Change from baseline in TGA (thrombin generation assay). |
Upto Day 135 |
| Incidence of Anti-Drug Antibody (ADA) |
Upto Day 135 |
| Incidence of clinically significant persistent neutralizing antibodies (NAb) |
Upto Day 135 |
|
|
Target Sample Size
|
Total Sample Size="15"
Sample Size from India="8"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 1 |
|
Date of First Enrollment (India)
|
29/08/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
13/05/2025 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="0"
Months="6"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A. A rare bleeding disorder where the blood doesn’t clot normally. This causes a person to bleed a lot, even from a small cut. These patients can be with or without inhibitors who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines. This study is seeking for participants: - with severe Hemophilia A who are on emicizumab treatment for at least 6 months.
- must be 12 to less than 75 years old
- must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study.
|