| CTRI Number |
CTRI/2021/02/031507 [Registered on: 23/02/2021] Trial Registered Prospectively |
| Last Modified On: |
22/02/2021 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Randomized, Crossover Trial |
|
Public Title of Study
|
Study of Marzeptacog Alfa (Activated) in Subjects with Hemophilia A or B |
|
Scientific Title of Study
|
Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On-Demand Treatment and Control of Bleeding Episodes in Subjects with
Hemophilia A or Hemophilia B, with Inhibitors: The Crimson 1 Study |
| Trial Acronym |
|
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| 14789 (IND Number) |
Other |
| MAA-304 ,Amendment 1.0 dated 2 June 2020 |
Protocol Number |
| NCT04489537 |
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 |
Amruta Kabadi |
| Designation |
Regulatory Submissions Manager |
| Affiliation |
Medpace Clinical Research India Pvt. Ltd. |
| Address |
Office No. 817, 8th Floor, Rupa Solitaire
Building No. A-1, Sector-1, Millenium Business Park
Next to DAKC, Mahape, Navi Mumbai India
Thane
MAHARASHTRA
400710
India |
| Phone |
912241223014 |
| Fax |
|
| Email |
A.Kabadi@Medpace.com |
|
Details of Contact Person
Public Query
|
| Name |
Amruta Kabadi |
| Designation |
Regulatory Submissions Manager |
| Affiliation |
Medpace Clinical Research India Pvt. Ltd. |
| Address |
Office No. 817, 8th Floor, Rupa Solitaire
Building No. A-1, Sector-1, Millenium Business Park
Next to DAKC, Mahape, Navi Mumbai India
Thane
MAHARASHTRA
400710
India |
| Phone |
912241223014 |
| Fax |
|
| Email |
A.Kabadi@Medpace.com |
|
|
Source of Monetary or Material Support
|
| Catalyst Biosciences, Inc. |
|
|
Primary Sponsor
|
| Name |
Catalyst Biosciences Inc |
| Address |
611 Gateway Blvd., Suite 710
South San Francisco, CA 94080 |
| Type of Sponsor |
Other [Biopharmaceutical company] |
|
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Details of Secondary Sponsor
|
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Countries of Recruitment
|
Armenia
Denmark
Georgia
Hungary
India
Italy
Malaysia
Mexico
Poland
Russian Federation
South Africa
Spain
Taiwan
Turkey
Ukraine
United Kingdom
United States of America |
|
Sites of Study
|
| No of Sites = 8 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Uttam Kumar Nath |
All India Institute of Medical Sciences (AIIMS), |
Department of Medical Oncology Haematology,Rishikesh 249203, Uttarakhand
Dehradun
UTTARANCHAL |
919433982756
nath.uttam@gmail.com |
| Dr Vijay Ramanan |
Grant Medical Foundation Ruby Hall Clinic |
Department of Critical Care Medicine,40, Sassoon Road, Pune 411 001
Pune
MAHARASHTRA |
9325315471
mvijayr@gmail.com |
| Dr Savita Rangarajan |
K.J Somaiya Hospital and Research Centre |
Somaiya Ayurvihar Complex, Eastern Express Highway
Sion (East), Mumbai – 400022
India
Mumbai
MAHARASHTRA |
919619525341
Rangarajansavita@gmail.com |
| Dr Madhav Prabhu |
KLEs Dr Prabhakar Kore Hospital and MRC |
Ground floor, OPD no. 11,Nehru Nagar. Dist. Belagavi State. Karnataka, India -590010
Belgaum
KARNATAKA |
919923693949
wiseman2380@yahoo.com |
| Dr Mamta V Manglani |
MCGM Comprehensive Thalassemia PHO & BMT Centre, |
CCI Compound, Opp. Kanakia Exotica, Borivali East, Mumbai – 400066
Mumbai
MAHARASHTRA |
91982132207
mmanglani@hotmail.com |
| Dr Dharmesh Vaghasiya |
Nirmal Hospital Pvt Ltd |
Nirmal Hospital Pvt Ltd, 1st floor , OPD, Ring Road, Surat 395002, Gujarat, India
Surat
GUJARAT |
917767054520
drdharmeshrvaghasiya@gmail.com |
| Dr Shashikant Apte |
Sahyadri Super Speciality Hospital |
Department of Haematology & Bone Marrow Transplant Unit,Nagar Road
Survey No. 185A, Shastri Nagar, Near MSEB Office Yerwada, Nagar Road,Pune-411006, Maharashtra, India
Pune
MAHARASHTRA |
919822404983
shashikant.apte@gmail.com |
| Dr Shubha R Phadke |
SGPGIMS, |
Department of Medical Genetics, SGPGIMS, Raebareli Road, Lucknow 226014
Lucknow
UTTAR PRADESH |
919839220199
shubharaophadke@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 7 |
| Name of Committee |
Approval Status |
| Institutional Ethics Committee K.J Somaiya Medical College and Research Centre |
Approved |
| Institutional Ethics Committee of CTC, PHO and BMT Centre, CCI Compound, Opp. Kanakia Exotica, Borivali East, Mumbai – 400066 |
Submittted/Under Review |
| Institutional Ethics Committee Poona Medical Research Foundation E 4 –C to E 4-F , 4th Floor, Fifth Avenue Condominium , Dhole Patil Road, Pune – 411 001 ,Maharashtra, India |
Approved |
| Institutional Ethics Committee, AIIMS Rishikesh, Department of Physiology, AIIMS Rishikesh, Veerbhadra Marg, Rishikesh, Uttarakhand, Pin 249203, India |
Submittted/Under Review |
| Institutional Ethics Committee, Bioethics Cell, Administrative Block, Room No. 205, SGPGIMS, Raebareli Road, Lucknow 226014 |
Submittted/Under Review |
| Nirmal Hospital Pvt Ltd Ethics Committee, Nirmal Hospital Pvt Ltd, Ring Road, Surat 395002, Gujarat, India |
Approved |
| Sahyadri Hospitals Ltd. Ethics committee Sahyadri Clinical Research and Development Center |
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 |
Recombinant Factor VIIa (rFVlla), Marzeptacog alfa (activated) [MarzAA] |
Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis |
| Comparator Agent |
Standard of Care |
Standard of care administered on-demand during bleeding episodes |
|
|
Inclusion Criteria
|
| Age From |
12.00 Year(s) |
| Age To |
99.00 Year(s) |
| Gender |
Both |
| Details |
1.Diagnosis of congenital hemophilia A or B with inhibitors
2. Male or Female, age 12 or older
3. History of frequent bleeding episodes
4. Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities |
|
| ExclusionCriteria |
| Details |
1. Previous participation in a clinical trial evaluating a modified rFVIIa agent
2. Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect
3. Known hypersensitivity to trial or related product
4.Known positive antibody to FVII or FVIIa detected by central lab at screening
5. Have a coagulation disorder other than hemophilia A or B
6. Be immunosuppressed
7. Significant contraindication to participate |
|
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Method of Generating Random Sequence
|
Other |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Open Label |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Bleeding episode treatment success |
24 hours after the first administration of study drug |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
1)To cessation of bleeding
2)To To assess the ability of MarzAA to achieve and maintain hemostasis
3)To assess the number of doses and the cumulative dose
4)To assess the percentage of bleeds with treatment
5) To assess the use and amount of rescue therapy
6)To assess the SC PopPK of MarzAA |
Secondary outcome no. 4) at 24 hours |
|
|
Target Sample Size
|
Total Sample Size="60"
Sample Size from India="12"
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 3 |
|
Date of First Enrollment (India)
|
26/02/2021 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
16/12/2020 |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="1"
Months="3"
Days="2" |
|
Recruitment Status of Trial (Global)
|
Open to Recruitment |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
Publication Details
|
NIL
|
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Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
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Brief Summary
|
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors compared with their standard of care (SOC). |