| CTRI Number |
CTRI/2025/09/094511 [Registered on: 10/09/2025] Trial Registered Prospectively |
| Last Modified On: |
10/10/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
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Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
An Open-label Study for the Treatment and Prophylaxis in Severe Hemophilia a Patients |
|
Scientific Title of Study
|
A Phase 1/3, Open-label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of Human Plasma-derived Factor VIII (SKP-0141) for the treatment and prophylaxis in Male Patients with Severe Hemophilia A. |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| SKP-0141_HemA_l/III_2024 version No. 2.0 dated 19- MAR-2025 |
Protocol Number |
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Details of Principal Investigator or overall Trial Coordinator (multi-center study)
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| Name |
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| Designation |
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| Affiliation |
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| Address |
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| Phone |
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| Fax |
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| Email |
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Details of Contact Person Scientific Query
|
| Name |
Shweta Pradhan |
| Designation |
Head Clinical Operations |
| Affiliation |
IQVIA RDS (India) Private Limited |
| Address |
Omega Embassy Tech Square, Marathahalli - Sarjapura, Outer Ring Road, Kadubeesanahalli, Karnataka
Bangalore KARNATAKA 560103 India |
| Phone |
919513774664 |
| Fax |
|
| Email |
shweta.pradhan@iqvia.com |
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Details of Contact Person Public Query
|
| Name |
Shweta Pradhan |
| Designation |
Head Clinical Operations |
| Affiliation |
IQVIA RDS (India) Private Limited |
| Address |
Omega Embassy Tech Square, Marathahalli - Sarjapura, Outer Ring Road, Kadubeesanahalli, Karnataka
KARNATAKA 560103 India |
| Phone |
919513774664 |
| Fax |
|
| Email |
shweta.pradhan@iqvia.com |
|
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Source of Monetary or Material Support
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| SK Plasma Co., Ltd.
310 Pangyo-ro, Bundang-gu, Seongnam-si, Gyeonggi-do, Republic of Korea, 13494. |
|
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Primary Sponsor
|
| Name |
SK Plasma Co Ltd |
| Address |
310 Pangyo-ro, Bundang-gu, Seongnam-si, Gyeonggi-do, Republic of Korea, 13494. |
| Type of Sponsor |
Pharmaceutical industry-Global |
|
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Details of Secondary Sponsor
|
| Name |
Address |
| IQVIA RDSIndia Pvt Ltd |
Omega Embassy TechSquare,
Marathahalli-Sarjapur Outer Ring Road,
Kadubeesanahalli,
Bangalore – 560103, Karnataka |
|
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Countries of Recruitment
|
India Indonesia Malaysia Philippines Republic of Korea Thailand Turkey |
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Sites of Study
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| No of Sites = 8 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Vijay Ramanan |
Grant Medical Foundation Ruby Hall Clinic |
40 Sassoon Road, Pune-411001, Maharashtra India Pune MAHARASHTRA |
9822240022
bmtpune@gmail.com |
| Dr Smita Das |
Guwahati Medical College and Hospital |
Narakasur Hilltop Hospital Road Bhangagarh Guwahati
Assam - 781032
Kamrup ASSAM |
9864068293
smitabhuyanghy@gmail.com |
| Dr Sunita Aggarwal |
Maulana Azad Medical College and Associated Hospitals |
Room no 122, Department of Medicine,B L Taneja Block,Maulana Azad Medical College, New Delhi 110002, India New Delhi DELHI |
9686042866
drsunita.mamc@gmail.com |
| Dr Dharmesh Rameshbhai Vaghasiya |
Nirmal Hospital Pvt Ltd |
Ring Road, Surat-395002, Gujarat, India Surat GUJARAT |
7767054520
drdharmeshrvaghasiya@gmail.com |
| Dr KK Radhika |
Nizam s Institute of Medical Sciences |
Dept of Haematology. Nizam s Institute of Medical Sciences, Punjagutta, Hlyderabad, Telangana- 500082 Hyderabad TELANGANA |
9494649657
radhika_setti@yahoo.com |
| Dr Shashikant Janardan Apte |
Sahyadri super Speciality Hospital. |
Plot No. 30C, Erandawane, karve Road, Pune 411004 Maharashtra Pune MAHARASHTRA |
919175980913
shashikant.apte@gmail.com |
| Dr Cecil Reuben Ross |
St. John’s Medical College Hospital |
St. John’s Medical College Hospital, Sarjapur main road, John nagar, Koramangala, Bangalore - 560034, Karnataka, India Bangalore KARNATAKA |
9448493705
cecil.ross@stjohns.in |
| Dr Alok Ranjan |
State Cancer Institute Indira Gandhi Institute of Medical Sciences |
Sheikhpura, Patna-800014, Bihar, India Patna BIHAR |
9572240838
dralokranjansciigims@gmail.com |
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Details of Ethics Committee
Modification(s)
|
| No of Ethics Committees= 9 |
| Name of Committee |
Approval Status |
| Aurangabad Health Care and Research LLP IEC |
Approved |
| Institutional Ethics committee Poona Medical Research Foundation, Dr Vijay Ramanan |
Approved |
| Institutional Ethics Committee MAMC, Dr Sunita Aggarwal |
Submittted/Under Review |
| Institutional Ethics Committee State Cancer Institute Indira Gandhi, Dr. Alok Ranjan |
Approved |
| Institutional Ethics Committee, GMCH Gauhati Medical College,Dr. Smita Das |
Approved |
| Institutional Ethics Committee, St. John’s Medical College Hospital |
Submittted/Under Review |
| NIMS Institutional Ethics Committee,Dr K.K. Radhika |
Approved |
| Nirmal Hospital Pvt Ltd Ethics Committee, Dr. Dharmesh Vaghasiya |
Approved |
| Sahyadri Hospitals Pvt. Ltd. Ethics Committee, Dr. Shashikant Janardan Apte |
Approved |
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Regulatory Clearance Status from DCGI
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Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D66||Hereditary factor VIII deficiency, |
|
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Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
Not Applicable |
Not Applicable |
| Intervention |
Study Drug Name: SKP-0141 |
Route: IV injection
Dose: Lyophilized powder and solvent for solution for injection
Dose Range: 25 to 50 IU/kg
Treatment period: 25 weeks
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Inclusion Criteria
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| Age From |
12.00 Year(s) |
| Age To |
65.00 Year(s) |
| Gender |
Male |
| Details |
Patients are eligible to be included in the study only if all of the following criteria apply:
1. A patient or parent/legal guardian (as appropriate) who is capable of giving signed informed consent, and where required, the patient is capable of providing assent), which includes compliance with the requirements and restrictions listed in the Informed Consent Form (ICF), Assent Form, and in this protocol.
2. Patients assigned male at birth and must be 12 to 65 years old (inclusive) at the time of Screening (signing the ICF by the patient or their parent/legal guardian or provision of assent, as appropriate).
3. Diagnosis of severe congenital hemophilia A, defined as an FVIII level of greater than 1 percent as documented in the patient’s medical records. Note, if available documented evidence of FVIII level is not within 4 weeks of Screening visit, a baseline FVIII level assessments will be done at Screening.
4. Patients who have received or are currently receiving plasma-derived and or recombinant FVIII products and have had at least 150 EDs with a FVIII product.
5. Patients who can produce viable sperm and have a partner of childbearing potential must agree to take appropriate contraceptive measures consistently during the study, starting at Screening and until 30 days after the end of study (EOS) visit and also refrain from donating sperm during this period. Patients with a partner or partners who is (are) not of childbearing potential are exempt from these requirements. |
|
| ExclusionCriteria |
| Details |
Patients are excluded from the study if any of the following criteria apply:
1. Any history of or current FVIII inhibitors or any first order family history of FVIII inhibitors in terms of detectable FVIII inhibitors (ie, less than or equal to 0.6 Bethesda Units [BU]) using the Nijmegen-modification of the Bethesda assay
2. Any known congenital or acquired coagulation disorder other than the congenital hemophilia A.
3. Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction, and arterial embolus within 3 months prior to Visit 1.
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Method of Generating Random Sequence
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Not Applicable |
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Method of Concealment
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Not Applicable |
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Blinding/Masking
|
Not Applicable |
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Primary Outcome
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| Outcome |
TimePoints |
| Annualized bleeding rate (number of bleeding episodes per year) |
Visit 10 |
|
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Secondary Outcome
|
| Outcome |
TimePoints |
• Patient assessment of hemostatic response for any breakthrough bleeding episode, using a 4-point scale from start of treatment and until Visit 10
• Dose of SKP-0141 injections (IU/kg/year and IU/kg/month) required for prophylaxis from start of treatment and until Visit 10
|
Visit 10 |
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Target Sample Size
|
Total Sample Size="55" Sample Size from India="17"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
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Phase of Trial
|
Phase 3 |
|
Date of First Enrollment (India)
|
22/09/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
25/09/2025 |
| 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)
|
Not Yet Recruiting |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
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Publication Details
|
N/A |
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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
|
This is a prospective, multicenter,
open-label study of human plasma-derived FVIII in previously treated patients
with severe hemophilia A. Overall, 55 male PTPs with a FVIII level of less than
1percent and at least 150 treatment exposure days to previous FVIII products
will be enrolled.
Patients will undertake a Screening visit
between Week 4 to Week 0 to determine their eligibility to be enrolled in the
study. All eligible patients will enter the Treatment period of the study to
receive study treatment. During the Treatment period- Weeks 1 to 25, patients
will receive prophylactic treatment with SKP-0141.
At least the first 12 enrolled patients -PK
group, will participate in the PK assessments at Visit 1-single-dose PK, and Visit 10- steady-state PK. The single-dose
PK assessments will be performed at first exposure to SKP-0141- 25 to 50 IU per
kg after a minimum of 72-hour washout period, which may be extended up to 96
hours for long half-life FVIII products, from their last marketed FVIII
administration. The steady-state PK assessments will be performed around 25
weeks following initiation of prophylactic treatment, subsequent to a minimum
of 48-hour washout period from their prophylactic SKP-0141 administration. For
PK assessments, patients will undergo blood sampling at predefined time points.
If patients experience a bleeding episode (BE) during the initial PK assessment
period, they may be treated with their previous FVIII product. Once the BE is
controlled the patient may restart the PK assessment following the appropriate
washout period from their last marketed FVIII administration.
After the 12 patients in the PK group
complete the single-dose PK assessments- Visit 1, the available safety and PK
endpoints data will be reviewed by the Independent Data Monitoring Committee to
provide recommendations on whether the study should continue. Patient
recruitment and enrollment of the patients in the non-PK group will continue
during the IDMC review.
Patients will be followed-up for a period
of 1 week after completion of Visit 10. The patients will return to the site
for the Follow-up visit at Week 26 to document any adverse events or serious
adverse events.
A minimum of 10 surgical procedures
comprising major surgeries in a least 5 patients will be targeted to assess
control and prevention of bleeding in the surgical setting. |