Bilateral Blepharospasm (BEB)Phase 3NCT01896895

Efficacy and Safety Study of Botulinum Toxin Type A Against Placebo to Treat Abnormal Contraction or Twitch of the Eyelid

A Phase 3 study testing IncobulinumtoxinA (Xeomin), 25 Units, IncobotulinumtoxinA (Xeomin), 12.5 Units and IncobotulinumtoxinA (Xeomin), 35 Units in people with Bilateral Blepharospasm (BEB), run by Merz Pharmaceuticals GmbH at 11 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blepharospasm (BEB) who are BTX treatment-naïve. Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Bilateral Blepharospasm (BEB)
What is being tested?: IncobulinumtoxinA (Xeomin), 25 Units, IncobotulinumtoxinA (Xeomin), 12.5 Units and IncobotulinumtoxinA (Xeomin), 35 Units
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 61 participants
Where: 11 sitesin 3 countries
Study dates: Nov 2013to Nov 2016
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male or female out-patients age ≥ 18 and ≤ 80 years.
A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of orbicular oculi muscles.
A need for injection of BTX defined as a Jankovic Rating Scale [JRS] severity subscore ≥ 2.
Treatment-naïve subject defined as at least 12 months without BTX of any serotype for the treatment of BEB before administration of IP.

Who cannot join

Subject with any previous unsuccessful treatment with BTX of any serotype for the treatment of BEB.
Atypical variant of BEB (e.g., apraxia of the eyelid opening) caused by inhibition of levator palpebrae muscle.
Neuroleptic-induced blepharospasm.
Myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation, spinal cord stimulation) and surgery in the upper face.
Generalized disorders of muscles activity (e.g., myasthenia gravis in particular ocularis, Lambert-Eaton-Syndrome, amyotrophic lateral sclerosis) or any other significant neuromuscular dysfunction which might interfere with the study.

Trial locations and contacts

11 sites in 3 countries.

Country

City, postcode or hospital

11 sites in 3 countries

Malaysia5 sites

Merz Investigational Site #060007
George Town, Pulau Pinang, 10990
Merz Investigational Site #060004
Kota Kinabalu, Sabah, 88586
Merz Investigational Site #060006
Kuala Lumpur, 50586
Merz Investigational Site #060002
Kuala Lumpur, 56000
Merz Investigational Site #060003
Kuala Selangor, 43000

Other trials in Malaysia

Sri Lanka4 sites

Merz Investigational Site #094001
Colombo, 07
Merz Investigational Site #094005
Colombo, 10350
Merz Investigational Site #094006
Kurunegala, 60000
Merz Investigational Site #094002
Nugegoda, 10250

Other trials in Sri Lanka

Greece2 sites

Merz Investigational Site #030002
Athens, 11521
Merz Investigational Site #030001
Athens, 11526

Other trials in Greece

About this study

Official title: Prospective, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multi-center Study With an Open-label Extension Period to Investigate the Efficacy and Safety of Two Different Doses of NT 201 in Botulinum Toxin Treatment-naïve Subjects With Blepharospasm

This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blepharospasm (BEB) who are BTX treatment-naïve.

In this study, BTX treatment-naïve subjects are defined as those who have not received BTX treatment within the last 12 months for the treatment of BEB. This definition aims to avoid bias by comparison of treatment effects in the subject's assessments. Furthermore, this study will substantiate the existing efficacy and safety database for the indication BEB.

Read the detailed description

Subjects to receive one injection with NT 201 or placebo at baseline of the placebo-controlled first cycle. Thereafter, all subjects entering the Open-Label Extension Period (OLEX) to receive a second injection of NT 201 (second injection cycle).

Conditions: Bilateral Blepharospasm (BEB)
Being tested: IncobulinumtoxinA (Xeomin), 25 Units, IncobotulinumtoxinA (Xeomin), 12.5 Units, Placebo, IncobotulinumtoxinA (Xeomin), 35 Units

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IncobulinumtoxinA (Xeomin), 25 UnitsDrug
Also called NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 25 Units per eye.
IncobotulinumtoxinA (Xeomin), 12.5 UnitsDrug
Also called NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 12.5 Units per eye.
PlaceboDrug
Main Period: Placebo to IncobotulinumtoxinA (Xeomin), powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
IncobotulinumtoxinA (Xeomin), 35 UnitsDrug
Also called NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins
IncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
Open-Label Extension: up to 35 Units per eye.

Study groups in NCT01896895
Study group	Role	What they receive
IncobotulinumtoxinA (Xeomin) 25U per eye	Experimental	Main Period: one injection session, 25 Units per eye. Open-Label Extension: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection. Receives: Drug: IncobulinumtoxinA (Xeomin), 25 Units; Drug: IncobotulinumtoxinA (Xeomin), 35 Units
IncobotulinumtoxinA (Xeomin) 12.5U per eye	Experimental	Main Period: one injection session, 12.5 Units per eye. Open-Label Extension Period: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection. Receives: Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units; Drug: IncobotulinumtoxinA (Xeomin), 35 Units
Placebo	Placebo comparator	Main Period: Placebo to IncobotulinumtoxinA (Xeomin)(12.5 or 25U/eye), one injection session. Open-Label Extension: IncobotulinumtoxinA (Xeomin), one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection. Receives: Drug: Placebo; Drug: IncobotulinumtoxinA (Xeomin), 35 Units

What is being measured

The primary outcome is the main question the study is designed to answer.

Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4)
JRS severity subscore was used to classify individual symptoms of blepharospasm and to determine therapeutic efficacy. JRS severity subscore ranges from 0 to 4, where 0: None; 1: increased blinking present with external stimuli; 2: Mild but spontaneous eyelid fluttering, definitely noticeable, possibly embarrassing, but not functionally disabling, 3: Moderate, very noticeable spasm of eyelids only, mildly incapacitating, 4: Severe, incapacitating spasm of eyelids and possibly other facial muscles. Values represent least square (LS) mean differences between baseline and visit 4 resulting from analysis of covariance (ANCOVA) with treatment group, pooled site, and gender as fixed factors and baseline JRS severity subscore and age as covariates and missings replaced using the last observation carried forward (LOCF) method. Negative values denote improvement, while positive values denote deterioration vs. baseline.
Measured: Baseline, Day 43 (Visit 4)

Secondary outcomes (2)

Double-blind MP: Change From Baseline in Blepharospasm Disability Index (BSDI) at Day 43 (Visit 4)
BSDI is a scale for assessment of impairment of specific activities of daily living caused by blepharospasm. BSDI consists of six items (driving a vehicle; reading; watching TV; shopping; getting about on foot (walking); doing everyday activities), each ranging from 0 (=no impairment) to 4 (=no longer possible due to illness). The BSDI total score is a mean score for non-missing items ranging from 0 to 4. It is calculated by adding scores of all applicable and answered items, and dividing the resulting sum by the number of items answered. Outcome values represent LS mean differences between baseline and visit 4 (visit 4 value minus baseline value) resulting from ANCOVA with treatment group, pooled site, gender as fixed factors and baseline BSDI total score, age as covariates. Missings were replaced by the LOCF method. Negative values denote an improvement, while positive values denote deterioration vs. baseline.
Measured: Baseline, Day 43 (Visit 4)
Double-blind MP: Patient Evaluation of Global Response (PEGR) at Final Visit (Day 43-Day 141)
PEGR scale is a descriptive subjective 9-point response self-rating scale ranging from "complete abolishment of signs and symptoms" (value=+4) down to "very marked worsening" (value=-4). Outcome values represent least square means at visit 4 resulting from an ANCOVA with treatment group, pooled site, gender as fixed factors and age as covariates. Missing were set to a zero effect (value=0). Positive values denote an improvement, while negative values denote deterioration.
Measured: Baseline, Final Visit (Day 43-Day 141)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Nov 2013Nov 2016

First posted11 Jul 2013 (estimated)
Study startNov 2013
Primary completionApr 2016
Study completionNov 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Bilateral Blepharospasm (BEB)Studies 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: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 61 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01896895
Sponsor's protocol ID: MRZ60201_3074_1
Other IDs: 2012-004821-26
Status last verified: Jan 2018
First submitted: 8 Jul 2013
Last updated: 1 Mar 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Mitsikostas DD, Dekundy A, Hanschmann A, Althaus M, Scheschonka A, Pagan F, Jankovic J. Duration and onset of effect of incobotulinumtoxinA for the treatment of blepharospasm in botulinum toxin-naive subjects. Curr Med Res Opin. 2021 Oct;37(10):1761-1768. doi: 10.1080/03007995.2021.1965975. Epub 2021 Aug 24.
PubMed 34384301
Duarte GS, Rodrigues FB, Marques RE, Castelao M, Ferreira J, Sampaio C, Moore AP, Costa J. Botulinum toxin type A therapy for blepharospasm. Cochrane Database Syst Rev. 2020 Nov 19;11(11):CD004900. doi: 10.1002/14651858.CD004900.pub3.
PubMed 33211907
Mitsikostas DD, Dekundy A, Sternberg K, Althaus M, Pagan F. IncobotulinumtoxinA for the Treatment of Blepharospasm in Toxin-Naive Subjects: A Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial. Adv Ther. 2020 Oct;37(10):4249-4265. doi: 10.1007/s12325-020-01427-6. Epub 2020 Aug 10.
PubMed 32779096

Study at a glance

From the registry record, last updated by the sponsor on 1 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Prospective, Double-blind, Placebo-controlled, Randomized, Parallel-group, Multi-center Study With an Open-label Extension Period to Investigate the Efficacy and Safety of Two Different Doses of NT 201 in Botulinum Toxin Treatment-naïve Subjects With Blepharospasm
ClinicalTrials.gov ID: NCT01896895
Acronym / sponsor ID: MRZ60201_3074_1
Conditions: Bilateral Blepharospasm (BEB)
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 61 participants (actual)
Lead sponsor: Merz Pharmaceuticals GmbHIndustry (pharma, biotech, devices)
Study start: Nov 2013
Primary completion: Apr 2016 (actual)
Study completion: Nov 2016 (actual)
First posted: 11 Jul 2013 (estimated)
Results: Posted 1 Mar 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This phase 3 study will serve to collect efficacy and safety data of two different doses of NT 201 in subjects suffering from Bilateral Blepharospasm (BEB) who are BTX treatment-naïve.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
IncobotulinumtoxinA (Xeomin) 25U per eye	Experimental	Drug: IncobulinumtoxinA (Xeomin), 25 Units, Drug: IncobotulinumtoxinA (Xeomin), 35 UnitsMain Period: one injection session, 25 Units per eye. Open-Label Extension: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.
IncobotulinumtoxinA (Xeomin) 12.5U per eye	Experimental	Drug: IncobotulinumtoxinA (Xeomin), 12.5 Units, Drug: IncobotulinumtoxinA (Xeomin), 35 UnitsMain Period: one injection session, 12.5 Units per eye. Open-Label Extension Period: one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.
Placebo	Placebo comparator	Drug: Placebo, Drug: IncobotulinumtoxinA (Xeomin), 35 UnitsMain Period: Placebo to IncobotulinumtoxinA (Xeomin)(12.5 or 25U/eye), one injection session. Open-Label Extension: IncobotulinumtoxinA (Xeomin), one injection session, up to 35 Units per eye. Mode of administration: intramuscular injection.

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Male or female out-patients age ≥ 18 and ≤ 80 years.
A clinical diagnosis of bilateral BEB characterized by spontaneous, spasmodic, intermittent or persistent involuntary contractions of orbicular oculi muscles.
A need for injection of BTX defined as a Jankovic Rating Scale [JRS] severity subscore ≥ 2.
Treatment-naïve subject defined as at least 12 months without BTX of any serotype for the treatment of BEB before administration of IP.

Key exclusion criteria (5 listed)

Subject with any previous unsuccessful treatment with BTX of any serotype for the treatment of BEB.
Atypical variant of BEB (e.g., apraxia of the eyelid opening) caused by inhibition of levator palpebrae muscle.
Neuroleptic-induced blepharospasm.
Myotomy or denervation surgery in the affected muscles (e.g., peripheral denervation, spinal cord stimulation) and surgery in the upper face.
Generalized disorders of muscles activity (e.g., myasthenia gravis in particular ocularis, Lambert-Eaton-Syndrome, amyotrophic lateral sclerosis) or any other significant neuromuscular dysfunction which might interfere with the study.

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
IncobulinumtoxinA (Xeomin), 25 UnitsAlso: NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsIncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 25 Units per eye.	Drug	IncobotulinumtoxinA (Xeomin) 25U per eye
IncobotulinumtoxinA (Xeomin), 12.5 UnitsAlso: NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsIncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Main Period: 12.5 Units per eye.	Drug	IncobotulinumtoxinA (Xeomin) 12.5U per eye
PlaceboMain Period: Placebo to IncobotulinumtoxinA (Xeomin), powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).	Drug	Placebo
IncobotulinumtoxinA (Xeomin), 35 UnitsAlso: NT 201, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteinsIncobotulinumtoxinA (active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins) powder for solution for injection, prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl). Open-Label Extension: up to 35 Units per eye.	Drug	IncobotulinumtoxinA (Xeomin) 12.5U per eye, IncobotulinumtoxinA (Xeomin) 25U per eye, Placebo

Primary outcome

Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4)
Time frame: Baseline, Day 43 (Visit 4)
JRS severity subscore was used to classify individual symptoms of blepharospasm and to determine therapeutic efficacy. JRS severity subscore ranges from 0 to 4, where 0: None; 1: increased blinking present with external stimuli; 2: Mild but spontaneous eyelid fluttering, definitely noticeable, possibly embarrassing, but not functionally disabling, 3: Moderate, very noticeable spasm of eyelids only, mildly incapacitating, 4: Severe, incapacitating spasm of eyelids and possibly other facial muscles. Values represent least square (LS) mean differences between baseline and visit 4 resulting from analysis of covariance (ANCOVA) with treatment group, pooled site, and gender as fixed factors and baseline JRS severity subscore and age as covariates and missings replaced using the last observation carried forward (LOCF) method. Negative values denote improvement, while positive values denote deterioration vs. baseline.

2 secondary outcomes

Double-blind MP: Change From Baseline in Blepharospasm Disability Index (BSDI) at Day 43 (Visit 4)
Time frame: Baseline, Day 43 (Visit 4)
Double-blind MP: Patient Evaluation of Global Response (PEGR) at Final Visit (Day 43-Day 141)
Time frame: Baseline, Final Visit (Day 43-Day 141)

Results and safety

Results posted to the registry on 1 Mar 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Double-blind MP: Placebo	20	19
Double-blind MP: IncobotulinumtoxinA 25 Units	22	20
Double-blind MP: IncobotulinumtoxinA 50 Units	19	16
OLEX: IncobotulinumtoxinA 70 Units	0	0

Primary outcome: Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4)

Time frame: Baseline, Day 43 (Visit 4). Least squares mean (95% Confidence Interval). Unit: score on a scale

Measure	Double-blind MP: Placebo n = 20	Double-blind MP: IncobotulinumtoxinA 25 Units n = 22	Double-blind MP: IncobotulinumtoxinA 50 Units n = 19
Value	-0.6 (-1.0 to -0.1)	-1.0 (-1.5 to -0.6)	-1.8 (-2.3 to -1.3)

Double-blind MP: Placebo vs Double-blind MP: IncobotulinumtoxinA 50 Units: LS mean difference: -1.2 (95% CI -1.9 to -0.6), p =0.0004, ANCOVA
Double-blind MP: Placebo vs Double-blind MP: IncobotulinumtoxinA 25 Units: LS mean difference: -0.5 (95% CI -1.1 to 0.2), p =0.1452, ANCOVA

Population analysed: FAS was subset of participants in the SES of the double-blind MP for whom at least a baseline value of the JRS severity subscore was available.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Double-blind MP: From the time point of first injection up to final visit (Day 43 to 141); OLEX Period: From the time point of first injection up to end of study visit (Day 43 to 141). Other adverse events reported at 4.5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Double-blind MP: Placebo	0 of 20 (0%)	6 of 20 (30%)	0 of 20
Double-blind MP: IncobotulinumtoxinA 25 Units	2 of 22 (9.1%)	7 of 22 (31.8%)	0 of 22
Double-blind MP: IncobotulinumtoxinA 50 Units	1 of 19 (5.3%)	7 of 19 (36.8%)	0 of 19
OLEX: IncobotulinumtoxinA 70 Units	0 of 39 (0%)	4 of 39 (10.3%)	0 of 39

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Mar 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 34384301Mitsikostas DD, Dekundy A, Hanschmann A, Althaus M, Scheschonka A, Pagan F, Jankovic J. Duration and onset of effect of incobotulinumtoxinA for the treatment of blepharospasm in botulinum toxin-naive subjects. Curr Med Res Opin. 2021 Oct;37(10):1761-1768. doi: 10.1080/03007995.2021.1965975. Epub 2021 Aug 24.
Derived from this study: PubMed 33211907Duarte GS, Rodrigues FB, Marques RE, Castelao M, Ferreira J, Sampaio C, Moore AP, Costa J. Botulinum toxin type A therapy for blepharospasm. Cochrane Database Syst Rev. 2020 Nov 19;11(11):CD004900. doi: 10.1002/14651858.CD004900.pub3.
Derived from this study: PubMed 32779096Mitsikostas DD, Dekundy A, Sternberg K, Althaus M, Pagan F. IncobotulinumtoxinA for the Treatment of Blepharospasm in Toxin-Naive Subjects: A Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial. Adv Ther. 2020 Oct;37(10):4249-4265. doi: 10.1007/s12325-020-01427-6. Epub 2020 Aug 10.

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.

Merz Medical Expert study director

Merz Pharmaceuticals GmbH

11 sites in 3 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 4 inclusion and 5 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 1 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 sites in Malaysia, Sri Lanka, Greece. 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 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?

Yes. Results are posted in the registry (first posted 1 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Double-blind MP: Change From Baseline in JRS Severity Subscore at Day 43 (Visit 4) (time frame: Baseline, Day 43 (Visit 4)). 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 NCT01896895, which also lists 3 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 1 Mar 2018.