CTRI

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CTRI Number

CTRI/2025/03/081608 [Registered on: 04/03/2025] Trial Registered Prospectively

Last Modified On:

28/02/2025

Post Graduate Thesis

Type of Trial

Interventional

Type of Study

Drug

Study Design

Single Arm Study

Public Title of Study

Study of absorption distribution metabolism and excretion of Human Plasma Derived Factor IX in severe Factor IX Deficiency patients

Scientific Title of Study

A prospective single-arm open label study to evaluate Pharmacokinetics properties of Human Plasma Derived Factor IX in severe Factor IX Deficiency

Trial Acronym

nil

Secondary IDs if Any

Secondary ID	Identifier
NIL	NIL

Details of Principal Investigator or overall Trial Coordinator (multi-center study)

Name	Dr Nimish Narkar
Designation	Pharmacologist
Affiliation	K J SOMAIYA HOSPITAL AND RESEARCH CENTRE
Address	K J Somaiya Hospital and Research Centre 3rd Floor CTRU Department SS building Somaiya Ayurvihar Eastern Express Highway Sion Chunabhatti Mumbai 400022 Mumbai MAHARASHTRA 400022 India
Phone	9767870637
Fax
Email	nimish.narkar@somaiya.edu

Details of Contact Person
Scientific Query

Name	Dr Nimish Narkar
Designation	Pharmacologist
Affiliation	K J SOMAIYA HOSPITAL AND RESEARCH CENTRE
Address	K J Somaiya Hospital and Research Centre 3rd Floor CTRU Department SS building Somaiya Ayurvihar Eastern Express Highway Sion Chunabhatti Mumbai 400022 Mumbai MAHARASHTRA 400022 India
Phone	9767870637
Fax
Email	nimish.narkar@somaiya.edu

Details of Contact Person
Public Query

Name	Dr Nimish Narkar
Designation	Pharmacologist
Affiliation	K J SOMAIYA HOSPITAL AND RESEARCH CENTRE
Address	K J Somaiya Hospital and Research Centre 3rd Floor CTRU Department SS building Somaiya Ayurvihar Eastern Express Highway Sion Chunabhatti Mumbai 400022 Mumbai MAHARASHTRA 400022 India
Phone	9767870637
Fax
Email	nimish.narkar@somaiya.edu

Source of Monetary or Material Support

Reliance Life Science Pvt Ltd Dhirubhai Ambani Life Sciences Centre DALC Plot R 282 TTC Area of MIDC Thane Belapur Road Rabale Navi Mumbai 400 701 Maharashtra, India

Primary Sponsor

Name	Reliance Life Science Pvt.Ltd.
Address	Dhirubhai Ambani Life Sciences Centre Plot R 282 TTC Area of MIDC Thane Belapur Road Rabale Navi Mumbai 400701 Maharashtra India
Type of Sponsor	Pharmaceutical industry-Indian

Details of Secondary Sponsor

Name	Address
NIL	NIL

Countries of Recruitment

India

Sites of Study

No of Sites = 1
Name of Principal Investigator	Name of Site	Site Address	Phone/Fax/Email
Dr Nimish Narkar	K J Somaiya Hospital and Research Centre	CTRU dept 3rd floor SS Building K J Somaiya Hospital and Research Centre Somaiya Ayurvihar Sion Chunabhatti 400022 Mumbai MAHARASHTRA	09767870637 nimish.narkar@somaiya.edu

Details of Ethics Committee

No of Ethics Committees= 1
Name of Committee	Approval Status
Institutional Ethics Committee	Approved

Regulatory Clearance Status from DCGI

Status

Not Applicable

Health Condition / Problems Studied

Health Type	Condition
Patients	(1) ICD-10 Condition: D67\|\|Hereditary factor IX deficiency,

Intervention / Comparator Agent

Type	Name	Details
Intervention	Factor IX	GENERIC NAME Human Coagulation Factor IX QUALITATIVE AND QUANTITATIVE COMPOSITION Each lyophilized powder vial contains Factor IX IP 250 IU Protein IP NMT 0.62 g per L Sodium chloride IP NMT 0.3 M Mannitol IP NMT 0.007 M Calcium chloride IP NMT 0.003 M L Arginine HCl Ph Eur NMT 1 g per L L lysine Ph Eur NMT 1.5 g per L Heparin IP NMT 5 IU per mL Sodium citrate NMT 0.002 M DOSAGE FORM AND STRENGTH Single dose vial of 250 IU
Comparator Agent	Not applicable	Not applicable

Inclusion Criteria

Age From	18.00 Year(s)
Age To	90.00 Year(s)
Gender	Male
Details	Male subjects aged 18 years and above at the start of treatment Documented diagnosis of severe Hemophilia B FIX deficiency FIX coagulant levels of less than 1percent Negative for serology testing of HIV 1 and 2 If serology test results of HBsAg or Hepatitis C is positive subject enrolment will be allowed only if no active disease/virus is confirmed Written and signed informed consent form by adult subject Able to adhere to study schedules and requirements

ExclusionCriteria

Details

Subjects with history of inhibitors to FIX
Any other inherited or acquired bleeding disorder
Subjects who had not been previously exposed to pdFIX
Subjects with abnormal laboratory parameters like
Serum creatinine less than 1 point 5 times of upper normal limit
AST or ALT greater than 3 times of upper normal limit
Platelet count less than 100000 per micro litre
Hemoglobin less than 10 gm per dL
Neutrophils less than 1 point 5 into 103 per micro litre
Presence or history of
Hypersensitivity to pdFIX
Deep vein thrombosis or pulmonary embolism within 1 year prior to start of treatment
for the bleeding episode or surgery
Arterial thrombosis within 1 year prior to start of treatment for the bleeding episode or
surgery
Hypersensitivity to human plasma proteins
Oesophageal varicose bleeding
End-stage liver disease ie Child-Pugh score B or C
Ongoing break-through bleeding
Positive result for HIV 1 and 2
Polytrauma 1 year prior to start of treatment for the bleeding episode or surgery
History of any clinically significant diseases or any acute or chronic medical condition
which may in the opinion of investigator affect the conduct of the study including subjects
receiving immune-modulating drugs other than antiretroviral chemotherapy such as
alpha-interferon prednisone equivalent to 10 mg per day or similar drugs at study start

Subject participation in any other clinical trial 30 days prior to administration of IP
Any other condition which investigator feels would affect interpretation of the results or
render the subject at high risk

Method of Generating Random Sequence

Not Applicable

Method of Concealment

Not Applicable

Blinding/Masking

Not Applicable

Primary Outcome

Outcome	TimePoints
To determine the pharmacokinetics of Factor IX in subjects with severe Hemophilia B FIX deficiency FIX coagulant levels of less than 1 percent	A total of 10 blood samples 3 mL each will be collected First sample will be collected within 1 hour prior to drug administration 0 hour and subsequent samples will be collected at 10 minutes and half hour 1hr 2hr 4hr 8hr 24hr 32hr and 48 hours after infusion

Secondary Outcome

Outcome	TimePoints
To assess the safety of Factor IX in subjects with severe Hemophilia B	Vital signs will be assessed at screening & within 1 hour prior to infusion of Factor IX & then at 1hr 3hr 6hr 24hr & 48 hours post infusion Physical examination will be performed at Screening Routine clinical laboratory assessment including coagulation parameters Adverse events including early signs of allergic or hypersensitivity reactions

Target Sample Size

Total Sample Size="12"
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 4

Date of First Enrollment (India)

17/03/2025

Date of Study Completion (India)

Applicable only for Completed/Terminated trials

Date of First Enrollment (Global)

Date Missing

Date of Study Completion (Global)

Applicable only for Completed/Terminated trials

Estimated Duration of Trial

Years="1"
Months="0"
Days="0"

Recruitment Status of Trial (Global)

Not Applicable

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

A prospective, single-arm, open-label study to evaluate Pharmacokinetics properties of Human Plasma-Derived Factor IX in severe Factor IX Deficiency.

Primary Objective is to determine the pharmacokinetics of Factor IX in subjects with severe Hemophilia B (FIX deficiency, FIX coagulant levels of <1%).

Secondary Objectives is to assess the safety of Factor IX in subjects with severe Hemophilia B

12 adult male subjects with severe Hemophilia B will be enrolled into the study.

PK will be evaluated following a 10-minute intravenous infusion of a single dose of 50 IU/kg

body weight. For PK assessment, patients should not have received Factor IX or any other

concerned therapy for bleeding event within 7 days prior to dosing.

Screening period validity will be of three weeks. Subjects will receive a single infusion

of factor IX at a dose of 50 IU/ kg of body weight on Day 1 of PK testing (PK-Day 1).

Before start of the PK phase, there must be an at least 7 days wash-out period of any

Factor IX containing product or any other product used for bleeding events.

A total of 10 blood samples (3 mL each) will be collected for adult male subjects.

First sample will be collected within 1 hour prior to drug administration (0.00 hour) and

subsequent samples will be collected at 10 minutes and 0.5, 1, 2, 4, 8, 24, 32 and 48

hours post infusion.

Before the start of infusion, vital signs will be recorded within 1 hour. Blood will be

withdrawn for baseline Factor IX levels assessment. The vital signs will be recorded at 1,

3, 6, 24 and 48 hours’ post-infusion.

The adverse events and concomitant medications will be recorded, including potential

thromboembolic events.

The pharmacokinetic phase of the study will end at the last PK sample, i.e. 48 hours post infusion.

After completing the last PK sample at 48 hours post infusion, all the participating

subjects will enter into the observational phase of the study from next day, i.e. 72 hours

post infusion. During this observational phase, subjects will report to the site about any

bleeding events occurring over the next at least 5-6 months. All the subjects will receive

Factor IX treatment for their bleeding episodes during this observational study period.

Any other treatment will be at the investigator’s discretion. The site can enroll additional

subjects in this observational phase in order to have total 18 subjects in this study phase

(including 12 subjects from the PK phase of the study).

At the end of the observational study phase, all the patients from this observational

study could be evaluated and considered for further participation in an upcoming gene

therapy clinical trial program depending on their willingness and eligibility. The

observational study will conclude once the gene therapy clinical trial is initiated and all

study related activities will also be completed. All the subjects who are selected for the

gene therapy clinical trial program will then undergo activities for that trial.