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A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

Information source: Alnylam Pharmaceuticals
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Hemophilia A; Hemophilia B

Intervention: ALN-AT3SC (Drug); Sterile Normal Saline (0.9% NaCl) (Drug)

Phase: Phase 1

Status: Recruiting

Sponsored by: Alnylam Pharmaceuticals

Official(s) and/or principal investigator(s):
Benny Sorensen, MD PhD, Study Director, Affiliation: Alnylam Pharmaceuticals

Overall contact:
Alnylam Clinical Trials Hotline Call for Complete Site List, Phone: 617-575-7400

Summary

The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of ALN-AT3SC in healthy volunteers and Hemophilia A or B patients.

Clinical Details

Official title: A Phase 1 Single-ascending and Multiple-ascending Dose, Safety, Tolerability and Pharmacokinetics Study of Subcutaneously Administered ALN-AT3SC in Healthy Adult Volunteers and Hemophilia A or B Patients (Moderate or Severe Hemophilia)

Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment

Primary outcome: The safety of ALN-AT3SC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicities (DLTs), and AEs leading to study drug discontinuation.

Secondary outcome:

The pharmacokinetics (PK) of ALN-AT3SC as characterized by plasma PK profiles and urine samples.

The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma AT levels.

The pharmacodynamic (PD) effect of ALN-AT3SC, evaluated by Plasma TG.

Eligibility

Minimum age: 18 Years. Maximum age: 65 Years. Gender(s): Male.

Criteria:

Inclusion Criteria: Part A (SAD phase) inclusion:

- Healthy adult males aged 18 to 40 years inclusive at Screening.

- Subjects with adequate complete blood counts and liver function tests.

- Willing to provide written informed consent and willing to comply with study

requirements. Part B & C (MAD & MD phase) inclusion:

- Adult male hemophilia patients aged 18 to 65 years inclusive at Screening.

- Patients with adequate complete blood counts and liver function tests.

- Patients with moderate or severe, clinically stable hemophilia A or B (Factor VIII or

Factor IX ≤5%).

- Willing to provide written informed consent and willing to comply with study

requirements Exclusion Criteria: Part A (SAD phase) exclusion:

- Subjects with a personal history and/or family history of venous thromboembolism

(VTE)

- Subjects with a known co-existing thrombophilic disorder

- Subjects with a history of multiple drug allergies or history of allergic reaction to

an oligonucleotide or GalNAc.

- Subjects with a history of serious mental illness that includes, but is not limited

to schizophrenia, bipolar disorder, severe depression requiring hospitalization or pharmacological intervention.

- Subjects who have a clinically relevant history or presence of cardiovascular,

respiratory, gastrointestinal, renal, hematological, lymphatic, neurological, musculoskeletal, genitourinary, immunological including osteoarthritis and other inflammatory diseases, dermatological including rash, eczema, dermatitis, or connective tissue diseases or disorders. Part B & C (MAD & MD phase) exclusion:

- Patients with a current serious mental illness that, in the judgment of the

Investigator, may compromise patient safety, ability to participate in all study assessments, or study integrity.

- Patients who have a clinically relevant history or presence of cardiovascular,

respiratory, gastrointestinal, renal, neurological, inflammatory or other diseases that in the judgment of the investigator precludes their participation in the study.

- Patients with a known co-existing thrombophilic disorder

- Patients with a history of multiple drug allergies or history of allergic reaction to

an oligonucleotide or GalNAc.

- Patients who are known to be HIV positive and have a CD4 count <400 cells/μL

Locations and Contacts

Alnylam Clinical Trials Hotline Call for Complete Site List, Phone: 617-575-7400

Clinical Trial Site, Plovdiv, Bulgaria; Recruiting

Clinical Trial Site, Sofia, Bulgaria; Recruiting

Clinical Trial Site, Varna, Bulgaria; Recruiting

Clinical Trial Site, Kirov, Russian Federation; Recruiting

Clinical Trial Site, Moscow, Russian Federation; Recruiting

Clinical Trial Site, St. Petersburg, Russian Federation; Recruiting

Clinical Trial Site, St. Gallen, Switzerland; Recruiting

Clinical Trial Site, Zurich, Switzerland; Recruiting

Clinical Trial Site, London NW3 2QG, United Kingdom; Recruiting

Clinical Trial Site, London SE1 1YR, United Kingdom; Recruiting

Clinical Trial Site, Manchester, United Kingdom; Recruiting

Clinical Trial Site, Truro, United Kingdom; Recruiting

Additional Information

Starting date: January 2014
Last updated: April 16, 2015

Page last updated: August 20, 2015

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