A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe Hemophilia

Phase 3

• Conditions: Health Condition 1: D683- Hemorrhagic disorder due to circulating anticoagulants

• Registration Number: CTRI/2023/06/054541
• Lead Sponsor: Sanofi Healthcare India Private Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 25 July 2023

Recruitment & Eligibility

• Status: ot Yet Recruiting
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

Participants are eligible to be included in the study only if all of the following criteria apply:

Age

I 01. Males (adult and adolescent) >= 12 years of age at the time of signing the informed consent

and/or assent as applicable.

Type of participant and disease characteristics

I 02. Diagnosis of severe congenital hemophilia A or B (FVIII < 1% or FIX level <= 2%) as

evidenced by a central laboratory measurement at screening or documented medical record

evidence.

I 03. For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of

4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor

participants) treatment within the last 6 months prior to screening.

I 04. Must meet either the definition of inhibitor or non-inhibitor patient as below:

Inhibitor:

Requiring use of BPA/ emicizumab (Hemlibra®) for prophylaxis or BPA as on-demand

therapy for any bleeding episodes for at least the last 6 months prior to screening, and

meet one of the following Nijmegen-modified Bethesda assay results criteria:

- Inhibitor titer of >= 0.6 Bethesda units (BU)/mL at Screening, or

- Inhibitor titer of < 0.6 BU/mL at Screening with medical record evidence of

2 consecutive titers >= 0.6 BU/mL, or

- Inhibitor titer of < 0.6 BU/mL at Screening with medical record evidence of 1 inhibitor

titer >= 0.6 BU/mL and a history of severe allergic reaction (eg, anaphylaxis) or

nephrotic syndrome

Non-inhibitor:

Requiring use of CFC/ emicizumab (Hemlibra®) for prophylaxis or CFC as on-demand

therapy for any bleeding episodes for at least the last 6 months prior to screening, and

meet each of the following criterion:

- Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and

- No use of BPA to treat bleeding episodes for at least the last 6 months prior to Screening and

- No history of ITI therapy within the past 3 years prior to Screening.

Weight

Not Applicable

Sex, contraceptive/barrier method and pregnancy testing requirements

I 05. Male.

There are no contraceptive requirements for this study except where required by local

regulations.

Informed Consent

I 06. Willing and able to comply with the study requirements and to provide written informed

consent and assent in the case of participants under the age of legal consent, per local and national requirements

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:

Medical conditions

E 01. Known co-existing bleeding disorders other than congenital hemophilia A or B, ie, Von

Willebrand disease, additional factor deficiencies, acquired hemophilia or platelet

disorders.

E 02. History of antiphospholipid antibody syndrome.

E 03. History of arterial or venous thromboembolism, atrial fibrillation, significant valvular

disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants

who have experienced thrombosis associated with indwelling venous access may be

enrolled.

E 04. History of malignancy within 2 years prior to screening, except for basal or squamous cell

carcinoma of the skin that has been successfully treated.

E 05. Completion of a surgical procedure within 14 days prior to screening, or currently

receiving additional CFC or BPA infusion for postoperative hemostasis

E 06. At screening, anticipated need of surgery during the study or planned surgery scheduled to

occur during the study.

E 07. History of intolerance to SC injection(s).

E 08. History of alcohol abuse within the 12 months before Screening. Alcohol abuse in this

study is defined as regular weekly intake of more than 14 units (1 unit equals to 1 glass of wine

(Approximately 125 Ml) equals to 1 measure of spirits (approximately 1 fluid ounce) equals to half pint of

beer (approximately 284 mL).

E 09. Presence of clinically significant liver disease or any of the conditions below:

• History of portal hypertension, esophageal varices, or hepatic encephalopathy;

• Presence of ascites by physical exam

Study & Design

• Study Type: Interventional
• Study Design: Not specified