Nemaline MyopathyPhase 2NCT02035501
Treatment of TNNT1-Myopathy With L-Tyrosine.
A Phase 2 study testing L-Tyrosine in people with Nemaline Myopathy, run by Hadassah Medical Organization at 1 site in Israel.
Trial summary
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy. Read more about this study
- Who is it for?
- Ages Up to 20 years
- Conditions the study is for
- Nemaline Myopathy
- What is being tested?
- L-Tyrosine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteIsrael
- Study dates
- Jan 2014to Jan 2015
- Phase
- Phase 2
- 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 Up to 20 years
- All sexes
- Child, Adult
Who may be able to join
- Male or female patients
- any age with TNNT1-related myopathy
Who cannot join
- Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
The purpose of the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
- Conditions
- Nemaline Myopathy
- Being tested
- L-Tyrosine, Placebo
- Medical subject headings
- Myopathies, Nemaline
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-TyrosineDrug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| L-Tyrosine | Active comparator | Not described Receives: Drug: L-Tyrosine |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. goal attainment score
Measured: 3 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2014 (estimated)
- Study startJan 2014
- Primary completionJan 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nemaline MyopathyStudies of the same condition, open and under way.
- More trials run by Hadassah Medical OrganizationThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Shimon Edverson, Hadassah Medical Organization
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02035501
- Sponsor's protocol ID
- SimonEdvardson1
- Status last verified
- Jan 2014
- First submitted
- 2 Jul 2013
- Last updated
- 14 Jan 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of TNNT1-Myopathy With L-Tyrosine. A Double-blind, Placebo-controlled Crossover Trial.
- ClinicalTrials.gov ID
- NCT02035501
- Acronym / sponsor ID
- SimonEdvardson1
- Conditions
- Nemaline Myopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Study start
- Jan 2014
- Primary completion
- Jan 2015 (estimated)
- First posted
- 14 Jan 2014 (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 the study is to investigate if treatment with L-Tyrosine improves selected outcome measures of TNNT1 myopathy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| L-Tyrosine | Active comparator | Drug: L-Tyrosine |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- Up to 20 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Male or female patients
- any age with TNNT1-related myopathy
Key exclusion criteria (1 listed)
- Patients who are non-cooperative or parents/ legal guardians who are unwilling to sign consent form
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 |
|---|---|---|
| L-Tyrosine | Drug | L-Tyrosine |
| Placebo | Drug | Placebo |
Primary outcome
1. goal attainment score
Time frame: 3 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing L-Tyrosine in people with Nemaline Myopathy, run by Hadassah Medical Organization at 1 site in Israel.
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 2 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 14 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
1. goal attainment score (time frame: 3 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 Jan 2014.