Trial to Evaluate Safety and Tolerability of ALN-TTR02 in Transthyretin (TTR) Amyloidosis
This study is currently recruiting participants.
Verified April 2013 by Alnylam Pharmaceuticals
Sponsor:
Alnylam Pharmaceuticals
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01617967
First received: June 7, 2012
Last updated: April 23, 2013
Last verified: April 2013
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of ALN-TTR02 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
| Condition | Intervention | Phase |
|---|---|---|
|
TTR-mediated Amyloidosis |
Drug: ALN-TTR02 |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR Amyloidosis |
Resource links provided by NLM:
Further study details as provided by Alnylam Pharmaceuticals:
Primary Outcome Measures:
- Safety and tolerability in ATTR patients [ Time Frame: Up to 56 Days ] [ Designated as safety issue: Yes ]The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Secondary Outcome Measures:
- Pharmacokinetics (PK) of ALN-TTR02 [ Time Frame: Up to 208 days ] [ Designated as safety issue: Yes ]Cmax, Area Under Curve, Tmax
- Serum transthyretin (TTR) protein [ Time Frame: Up to 208 days ] [ Designated as safety issue: No ]Determination of % Lowering to pretreatment/Baseline Levels
| Estimated Enrollment: | 20 |
| Study Start Date: | May 2012 |
| Estimated Study Completion Date: | November 2013 |
| Estimated Primary Completion Date: | May 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: ALN-TTR02 |
Drug: ALN-TTR02
Ascending doses of ALN-TTR02 administered by intravenous (IV) infusion
|
Eligibility| Ages Eligible for Study: | 18 Years to 85 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Body mass index must be between 17 kg/m2 and ≤ 33 kg/m2;
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;
- Males agree to use appropriate contraception;
- Diagnosis of TTR amyloidosis;
- Adequate blood counts, liver and renal function;
- Willing to give written informed consent and are willing to comply with the study requirements.
Exclusion Criteria:
- Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
- Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;
- Prior liver transplant;
- Poor cardiac function;
- Considered unfit for the study by the Principal Investigator;
- Employee or family member of the sponsor or the clinical study site personnel.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01617967
Contacts
| Contact: Contact: Alnylam Clinical Trials Hotline. Call for Complete Site List. | 617-575-7400 or 1-866-330-0326 |
Locations
| France | |
| Clinical Trial Site | Recruiting |
| Le Kremlin-bicetre, France | |
| Clinical Trial Site | Recruiting |
| Marseille Cedex, France | |
| Germany | |
| Clinical Trial Site | Recruiting |
| Munster, Germany | |
| Portugal | |
| Clinical Trial Site | Recruiting |
| Lisbon, Portugal | |
| Clinical Trial Site | Recruiting |
| Porto, Portugal | |
| Spain | |
| Clinical Trial Site | Recruiting |
| Barcelona, Spain | |
| Clinical Trial Site | Recruiting |
| Majorca, Spain | |
| Sweden | |
| Clinical Trial Site | Recruiting |
| Umeå, Sweden | |
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
| Study Director: | Jared Gollob, MD | Alnylam Pharmaceuticals |
More Information
No publications provided
| Responsible Party: | Alnylam Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT01617967 History of Changes |
| Other Study ID Numbers: | ALN-TTR02-002, 2012-000467-24 |
| Study First Received: | June 7, 2012 |
| Last Updated: | April 23, 2013 |
| Health Authority: | Portugal: National Pharmacy and Medicines Institute Sweden: Medical Products Agency France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Spain: Agencia Española de Medicamentos y Productos Sanitarios Germany: Federal Institute for Drugs and Medical Devices |
Additional relevant MeSH terms:
|
Amyloidosis Proteostasis Deficiencies Metabolic Diseases |
ClinicalTrials.gov processed this record on May 16, 2013