TTR-mediated AmyloidosisPhase 1NCT01559077
Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects
A Phase 1 study testing ALN-TTR02 and Sterile Normal Saline (0.9% NaCl) in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects. Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- TTR-mediated Amyloidosis
- What is being tested?
- ALN-TTR02 and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Mar 2012to Nov 2012
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
- Non-smokers for at least 3 months preceding screening;
- Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
- Males agree to use appropriate contraception;
- Medical history must be verified by either a personal physician or medical practitioner as appropriate;
- Willing to give written informed consent and are willing to comply with the study requirements.
Who cannot join
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
- Received prescription drugs within 4 weeks of first dosing
Show all 10Show fewer
- Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
- Subjects who have used prescription drugs within 4 weeks of first dosing
- Considered unfit for the study by the Principal Investigator
- Employee or family member of the sponsor or the clinical study site personnel
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Clinical Site
Leeds, LS2 9LH
Clinical Site
London, NW10 7EW
About this study
Official title: A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
- Conditions
- TTR-mediated Amyloidosis
- Being tested
- ALN-TTR02, Sterile Normal Saline (0.9% NaCl)
- Keywords
- ATTR
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-TTR02Drug
Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)Drug
Calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| ALN-TTR02 | Active comparator | Not described Receives: Drug: ALN-TTR02 |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Measured: Up to 28 days
Secondary outcomes (2)
Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).
Measured: Up to 180 days
Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)
Measured: Up to 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2012 (estimated)
- Study startMar 2012
- Primary completionJun 2012
- Study completionNov 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for TTR-mediated AmyloidosisStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jared Gollob, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01559077
- Sponsor's protocol ID
- ALN-TTR02-001
- Status last verified
- Jan 2013
- First submitted
- 19 Mar 2012
- Last updated
- 15 Jan 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.
PubMed 23984729
Study at a glance
From the registry record, last updated by the sponsor on 15 Jan 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT01559077
- Acronym / sponsor ID
- ALN-TTR02-001
- Conditions
- TTR-mediated Amyloidosis
- Keywords
- ATTR
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2012
- Primary completion
- Jun 2012 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 21 Mar 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-TTR02 | Active comparator | Drug: ALN-TTR02 |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
- Non-smokers for at least 3 months preceding screening;
- Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
- Males agree to use appropriate contraception;
- Medical history must be verified by either a personal physician or medical practitioner as appropriate;
Key exclusion criteria (10 listed)
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALN-TTR02Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion | Drug | ALN-TTR02 |
| Sterile Normal Saline (0.9% NaCl)Calculated volume to match active comparator | Drug | Sterile Normal Saline (0.9% NaCl) |
Primary outcome
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Time frame: Up to 28 days
2 secondary outcomes
Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).
Time frame: Up to 180 days
Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)
Time frame: Up to 56 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jan 2013.
- Derived from this study: PubMed 23984729Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.
Related trials for TTR-mediated Amyloidosis
Active, not recruitingPhase 3
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular Disease, Neuromuscular Diseases (NMD)
RecruitingPhase 3
MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Active, not recruitingPhase 3
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Not yet recruitingObservational study
PET/CT Imaging in Carriers of TTR Mutations
ATTR, ATTR Gene Mutation
Enrolling by invitationPhase 3
A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jared Gollob, MD study director
Alnylam Pharmaceuticals
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-TTR02 and Sterile Normal Saline (0.9% NaCl) in people with TTR-mediated Amyloidosis, run by Alnylam Pharmaceuticals at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jan 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. (time frame: Up to 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01559077, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2013.