Unverricht-Lundborg DiseasePhase 3NCT03351569
Intravenous Immunoglobulin for Unverricht-Lundborg Disease.
A Phase 3 study testing Intravenous immunoglobulin in people with Unverricht-Lundborg Disease, run by Azienda Socio Sanitaria Territoriale di Mantova.
Trial summary
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease. Read more about this study
- Who is it for?
- Ages 18 to 25 years, men only
- Conditions the study is for
- Unverricht-Lundborg Disease
- What is being tested?
- Intravenous immunoglobulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- 9 Dec 2015to 30 Dec 2017
- Phase
- Phase 3
- 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 25 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- Malattia di Unverricht-Lundborg (genetic diagnosis)
Who cannot join
- Contraindications to intravenous immunoglobulin
About this study
Official title: Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease.
Read the detailed description
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis).
The patient was randomized to be treated with intravenous immunoglobulin or placebo 1:1 (crossover) once a month for at least one year.
Main objective: improvement of the action myoclonus. Secondary objectives: Improvement in the overall score and in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial.
The first analyst was scheduled at one year from the start of the trial. The program was to discuss the patient's analysis data and to let the patient decide in three possible ways: to continue the trial, to continue treatment with immunoglobulins, to suspend the treatment. Depending on the decision, it was planned to follow the patient throughout the year after the analysis, at least for one year.
- Conditions
- Unverricht-Lundborg Disease
- Being tested
- Intravenous immunoglobulin
- Keywords
- Unverricht-Lundborg disease, progressive myoclonus epilepsy, pharmacoresistant epilepsy
- Medical subject headings
- Unverricht-Lundborg Syndrome, Myoclonic Epilepsies, Progressive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous immunoglobulinDrug
Also called Venital
Intravenous drip.
| Study group | Role | What they receive |
|---|---|---|
| Immunoglobulin | Experimental | Intravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year. Receives: Drug: Intravenous immunoglobulin |
| Saline solution | Placebo comparator | Intravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year. Receives: Drug: Intravenous immunoglobulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.
The range for Action Myoclonus Score is 0 (best) - 160 (worst, , i.e. more severe involuntary movements). Percent change = 100 X (Placebo UMRS4 - Treatment UMRS4) / Placebo UMRS4).
Measured: monthly for one year
Secondary outcomes (2)
Unified Myoclonus Rating Scale (UMRS) overall score improvement.
The total value of the UMRS (range from 0 - best - to 365 - worst) is composed of the sum of 6 sections: (1) Patient Questionnaire (range 0-48), (2) Myoclonus at rest (range 0-108), (3) Stimulus Sensitivity (range 0-17), (4) Myoclonus with Action (range 0-160), (5) Functional Tests (0-28), (6) Global Disability Score (range 0-4).
Measured: monthly for one year
Patient's preference
The program was to discuss the patient's analysis data with the patient himself and to let him decide in three possible ways: (1) to continue the trial, (2) to continue treatment with immunoglobulins, (3) to suspend the treatment.The choice number 2 is considered a favorable outcome.
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2017
- Study start9 Dec 2015
- Primary completion6 Dec 2016
- Study completion30 Dec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Unverricht-Lundborg DiseaseStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- sigle patient trial
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Azienda Socio Sanitaria Territoriale di Mantova
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Alfonso Ciccone, Azienda Socio Sanitaria Territoriale di Mantova
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03351569
- Sponsor's protocol ID
- 200200
- Other IDs
- 2017-002147-15, 2017
- Status last verified
- Nov 2017
- First submitted
- 27 Oct 2017
- Last updated
- 27 Nov 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: We planned to publish the results of this single patient trial.
Publications
Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme.11.7.
PubMed 21695041Guatelli JC, Gingeras TR, Richman DD. Alternative splice acceptor utilization during human immunodeficiency virus type 1 infection of cultured cells. J Virol. 1990 Sep;64(9):4093-8. doi: 10.1128/JVI.64.9.4093-4098.1990.
PubMed 2384914
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intravenous Immunoglobulin for Unverricht-Lundborg Disease: Single-patient Trial.
- ClinicalTrials.gov ID
- NCT03351569
- Acronym / sponsor ID
- 200200
- Conditions
- Unverricht-Lundborg Disease
- Keywords
- Unverricht-Lundborg disease, progressive myoclonus epilepsy, pharmacoresistant epilepsy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Azienda Socio Sanitaria Territoriale di MantovaUniversity, hospital or other
- Study start
- 9 Dec 2015 (actual)
- Primary completion
- 6 Dec 2016 (actual)
- Study completion
- 30 Dec 2017 (estimated)
- First posted
- 24 Nov 2017 (actual)
- 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.
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease.
Detailed description
Single patient randomized double blind trial to assess whether intravenous immunoglobulin can improve the clinical outcome of a case suffering from Unverricht-Lundborg disease (clinical and genetic diagnosis).
The patient was randomized to be treated with intravenous immunoglobulin or placebo 1:1 (crossover) once a month for at least one year.
Main objective: improvement of the action myoclonus. Secondary objectives: Improvement in the overall score and in individual sections of the Unified Myoclonus Rating Scale at one year; patient preferences based on results at the end of the trial.
The first analyst was scheduled at one year from the start of the trial. The program was to discuss the patient's analysis data and to let the patient decide in three possible ways: to continue the trial, to continue treatment with immunoglobulins, to suspend the treatment. Depending on the decision, it was planned to follow the patient throughout the year after the analysis, at least for one year.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- sigle patient trial
| Arm | Type | Receives |
|---|---|---|
| Immunoglobulin | Experimental | Drug: Intravenous immunoglobulinIntravenous immunoglobulin 25 grams (five 100 ml bottles, 5g/100ml), in 3 hours, once a month for one year. |
| Saline solution | Placebo comparator | Drug: Intravenous immunoglobulinIntravenous saline solution 500 ml (five 100 ml bottles), in 3 hours, once a month for one year. |
Who may qualify
- Ages
- 18 to 25 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (1 listed)
- Malattia di Unverricht-Lundborg (genetic diagnosis)
Key exclusion criteria (1 listed)
- Contraindications to intravenous immunoglobulin
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 |
|---|---|---|
| Intravenous immunoglobulinAlso: VenitalIntravenous drip. | Drug | Immunoglobulin, Saline solution |
Primary outcome
Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale.
Time frame: monthly for one year
The range for Action Myoclonus Score is 0 (best) - 160 (worst, , i.e. more severe involuntary movements). Percent change = 100 X (Placebo UMRS4 - Treatment UMRS4) / Placebo UMRS4).
2 secondary outcomes
Unified Myoclonus Rating Scale (UMRS) overall score improvement.
Time frame: monthly for one year
Patient's preference
Time frame: one year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2017.
- Background: PubMed 21695041Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme.11.7.
- Background: PubMed 2384914Guatelli JC, Gingeras TR, Richman DD. Alternative splice acceptor utilization during human immunodeficiency virus type 1 infection of cultured cells. J Virol. 1990 Sep;64(9):4093-8. doi: 10.1128/JVI.64.9.4093-4098.1990.
- Individual participant dataSharing planned: Yes. We planned to publish the results of this single patient trial.
Related trials for Unverricht-Lundborg Disease
RecruitingObservational study
Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1)
Progressive Myoclonus Epilepsy Type 1, EPM1
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.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Intravenous immunoglobulin in people with Unverricht-Lundborg Disease, run by Azienda Socio Sanitaria Territoriale di Mantova.
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 1 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 27 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Improvement of at least 20% of the action myoclonus at one year, measured with section 4 (Action Myoclonus) of the Unified Myoclonus Rating Scale. (time frame: monthly for one year). 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 NCT03351569, which also lists 2 linked publications.
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 27 Nov 2017.