Transthyretin Mediated Amyloidosis (ATTR)Phase 1NCT01148953
Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis
A Phase 1 study testing ALN-TTR01 and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin Mediated Amyloidosis (ATTR), run by Alnylam Pharmaceuticals at 4 sites in 4 countries.
Trial summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Mediated Amyloidosis (ATTR)
- What is being tested?
- ALN-TTR01 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
- 32 participants
- Where
- 4 sitesin 4 countries
- Study dates
- Jun 2010to Feb 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of TTR amyloidosis
- Adequate blood counts, liver and renal function
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
- Males agree to use appropriate contraception
- Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.
Who cannot join
- Known human immunodeficiency virus (HIV) positive status
- Receiving antibiotics for bacterial infection within 7 days of screening
- Known or suspected systemic viral, parasitic or fungal infection
- Receiving an investigational agent within 30 days prior to study drug administration
- Poor cardiac function
- Considered unfit for the study by the Principal Investigator
- Known sensitivity to oligonucleotides
- Employee or family member of the sponsor or the clinical study site personnel.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
About this study
Official title: A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
- Being tested
- ALN-TTR01, Sterile Normal Saline (0.9% NaCl)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-TTR01Drug
Dose levels between 0.01 and 1.0 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-TTR01 | Active comparator | Not described Receives: Drug: ALN-TTR01 |
| 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 patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Measured: Up to 28 Days
Secondary outcomes (2)
Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Measured: Up to 70 days
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
Measured: up to 70 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2010 (estimated)
- Study startJun 2010
- Primary completionJan 2012
- Study completionFeb 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 Transthyretin Mediated Amyloidosis (ATTR)Studies 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
- 32 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
- NCT01148953
- Sponsor's protocol ID
- ALN-TTR01-001
- Other IDs
- 2009-017383-16, 2009
- Status last verified
- May 2012
- First submitted
- 21 Jun 2010
- Last updated
- 24 May 2012
- 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 24 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis
- ClinicalTrials.gov ID
- NCT01148953
- Acronym / sponsor ID
- ALN-TTR01-001
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Jan 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 23 Jun 2010 (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-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-TTR01 | Active comparator | Drug: ALN-TTR01 |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of TTR amyloidosis
- Adequate blood counts, liver and renal function
- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
- Males agree to use appropriate contraception
- Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.
Key exclusion criteria (8 listed)
- Known human immunodeficiency virus (HIV) positive status
- Receiving antibiotics for bacterial infection within 7 days of screening
- Known or suspected systemic viral, parasitic or fungal infection
- Receiving an investigational agent within 30 days prior to study drug administration
- Poor cardiac function
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-TTR01Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion | Drug | ALN-TTR01 |
| Sterile Normal Saline (0.9% NaCl)Calculated volume to match active comparator | Drug | Sterile Normal Saline (0.9% NaCl) |
Primary outcome
The proportion of patients 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-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Time frame: Up to 70 days
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
Time frame: up to 70 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 24 May 2012.
- 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.
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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
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-TTR01 and Sterile Normal Saline (0.9% NaCl) in people with Transthyretin Mediated Amyloidosis (ATTR), run by Alnylam Pharmaceuticals at 4 sites in 4 countries.
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 5 inclusion and 8 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 24 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France, Portugal, Sweden, 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 patients 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 NCT01148953, 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 24 May 2012.