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Multiple System AtrophyPhase 2/3DOPS-AMSNCT02071459

Efficacy of L-threo DOPS on Orthostatic Hypotension Symptoms and Other Non-motor Symptoms in Patients With MSA

A Phase 2/3 study testing L-Threo DOPS in people with Multiple System Atrophy, run by University Hospital, Toulouse at 13 sites in France.

Completed

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

Official record
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Trial summary

Evaluate the effects of L-Threo DOPS on orthostatic hypotension symptoms and other non-motor symptoms in patients with Multiple System Atrophy (MSA) after 12 weeks following randomization to continued therapy with droxidopa or placebo. Read more about this study

Who is it for?
Ages 30 to 80 years
Conditions the study is for
Multiple System Atrophy
What is being tested?
L-Threo DOPS
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
107 participants
Where
13 sitesFrance
Study dates
21 Jan 2014to 28 Oct 2019
Phase
Phase 2/3
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 30 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • MSA patients (possible or probable, MSA-P or C (according to revised criteria, Gilman et al 2008)).
  • Aged 30 to 80 years,
  • Able to walk at least 10 meters
  • With symptomatic OH (score of at least 3 at one of the items of Part I of the OH scale (OHQ))
  • Documented fall in systolic blood pressure of at least 20 mmHg, and/or in diastolic blood pressure of at least 10 mmHg, within 3 minutes after standing.
  • Able to fill in the evaluation questionnaires with or without help
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  • With no significant problems with swallowing.
  • Stable anti-parkinsonian, dysautonomia and depression treatments for the 4 weeks before the study and during the entire study
  • Signed written informed consent for the present study.

Who cannot join

  • Dementia (DSM-IV, Mini-Mental State Examination (MMSE) < 24/30)
  • Concomitant use of vaso-constrictive drugs, other than midodrine. Patients taking vasoconstrictor agents such as ephedrine, dihydroergotamine, must stop taking these drugs at least 2 days or 7 half-lives prior to their baseline visit (Visit 1); the association with midodrine may be kept at a stable dose not exceeding 3 tablets (7.5 mg) / day if the patient has no CV history. This will be discussed case by case with the coordinating center and the safety committee of this study.
  • Taking anti-hypertensive medication

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

France13 sites

  • Centre hospitalier d'Angers

    Angers, 49933

  • CHU bordeaux

    Bordeaux

  • CHU de Clermont-Ferrand

    Clermont-Ferrand, 63000

  • CHU de Dijon

    Dijon, 21000

  • CHRU de lille

    Lille, 59037

  • CHU de limoges

    Limoges, 87042

  • Hôpital La Timone

    Marseille, 13000

  • Hôpital G. & R. Laennec

    Nantes, 44093

  • Hôpital Pitié-Salpétrière

    Paris, 75013

  • CHU de Poitiers

    Poitiers, 86021

  • CHU Pontchaillou

    Rennes, 35033

  • CHU de Rouen

    Rouen, 76031

  • chu de Strasbourg

    Strasbourg, 67091

Other trials in France

About this study

Official title: Evaluate the Long-term (3 Months) Efficacy of L-threo DOPS (DroxiDopa) on Orthostatic Hypotension Symptoms and Other Non-motor Symptoms in Patients With Multiple System Atrophy (MSA). Comparative Study Versus Placebo

Evaluate the effects of L-Threo DOPS on orthostatic hypotension symptoms and other non-motor symptoms in patients with Multiple System Atrophy (MSA) after 12 weeks following randomization to continued therapy with droxidopa or placebo.

Read the detailed description

Background :

Multiple system atrophy (MSA) is a rare, sporadic progressive neurodegenerative disorder, rapidly leading to severe disability and impairment of quality of life. MSA is characterized by a variable combination of a poor levodopa parkinsonism and /or cerebellar ataxia and autonomic failure (cardiovascular and / or bladder and sexual dysfunction) (Gilman et al, 2008). The prevalence is approximately 4-5 cases per 100 000 inhabitants.

Orthostatic hypotension (OH) is one of the major symptoms of MSA, present in a large majority of patients, leading to significant disability because of impaired balance, falls and possibly syncope. Drugs available to treat OH in this disease are very limited.

L-ThreoDOPS (L DOPS or DroxiDopa) is an orally administered synthetic catecholamine acid that is converted to the sympathetic neurotransmitter norepinephrine (NE) through a single step of decarboxylation by the endogenous enzyme 3,4-dihydroxyphenylalanine (DOPA) decarboxylase. It prevents symptoms related to OH by central and/peripheral mechanisms. This drug is currently developed for "neurogenic OH" by Chelsea Therapeutics on the basis of short duration placebo-controlled randomized trials. Besides an expected effect on OH, L-DOPS may also, by noradrenergic stimulation, improve some motor and non-motor symptoms common and disabling in MSA patients such as akinesia and fatigue.

In this context, the French reference center for MSA and the 12 national centers with identified skills to manage this disease, propose to conduct a national multicenter randomized clinical trial versus placebo to evaluate the long term efficacy (3 months) of L-threo DOPS on the OH and other non-motor symptoms in MSA patients.

Being tested
L-Threo DOPS, placebo
Keywords
L-threo DOPS (DroxiDopa), Orthostatic Hypotension, Multiple system atrophy
Medical subject headings
Multiple System Atrophy, Hypotension, Orthostatic

What participants receive

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

  • L-Threo DOPSDrug

    Also called L DOPS or DroxiDopa

    initial dose titration period (4 weeks) followed by 8 weeks at the max tolerated dose

  • placeboDrug

    initial period (4 weeks) followed by 8 weeks

Study groups in NCT02071459
Study groupRoleWhat they receive
L-Threo DOPSExperimentalpatients with Multiple System Atrophy (MSA) after 12 weeks to continued therapy with L-Threo DOPS
Receives: Drug: L-Threo DOPS
placeboPlacebo comparatorpatients with Multiple System Atrophy (MSA) after 12 weeks to continued therapy with placebo
Receives: Drug: placebo

What is being measured

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

  1. Evaluate the efficacy of long term efficacy of L-threo DOPS

    Evaluate the efficacy of long term efficacy of L-threo DOPS (droxidopa) in MSA patients (probable or possible - cerebellar (C) or parkinsonian (P) type) with symptomatic NOH as measured by the relative change in mean score of Orthostatic Hypotension Symptom Assessment (OHSA) (Part I of the questionnaire on the symptoms OH (OHQ) (Kaufmann et al., 2011)) 12 weeks following randomization to therapy with droxidopa or placebo (including 8 weeks to maximum tolerated dose).

    Measured: 12 weeks

Secondary outcomes (4)

  1. efficacy of L-ThreoDOPS on symptomatic OH

    Evaluate and compare the efficacy of L-ThreoDOPS on symptomatic OH (measured by the relative change in mean score of Item 1 of the Orthostatic Hypotension Symptom Assessment (OHSA)) in MSA patients 4, 8 and 12 weeks following randomization to continued therapy with droxidopa or placebo

    Measured: 12 weeks

  2. effects of L-Threo DOPS on motor symptoms

    Evaluate the effects of L-Threo DOPS on motor symptoms (UMSARS I and II) in MSA patients after 12 weeks following randomization to continued therapy with droxidopa or placebo

    Measured: 12 weeks

  3. effect of L-Threo DOPS on dysautonomic symptoms

    Evaluate the effect of L-Threo DOPS on dysautonomic symptoms (COMPASS) in MSA patients after 4, 8 and 12 weeks following randomization to continued therapy with droxidopa or placebo

    Measured: 12 weeks

  4. safety of high doses of L-ThreoDOPS

    Determine the safety of high doses of L-ThreoDOPS in MSA patients based on the occurrence of treatment-emergent adverse events

    Measured: 12 Weeks

Timeline

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

  1. First posted25 Feb 2014 (estimated)
  2. Study start21 Jan 2014
  3. Primary completion28 Oct 2019
  4. Study completion28 Oct 2019

Resources

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
107 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02071459
Sponsor's protocol ID
12 554 01
Other IDs
12-018-0200
Acronym
DOPS-AMS
Status last verified
Apr 2026
First submitted
21 Feb 2014
Last updated
8 Apr 2026
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • Dierickx LO, Bettiol C, Huglo D, Marcelli F, Sibert L, Gouel P, Ferretti L, Orlhac F, Huyghe E. Is 18F-fluorodexyglucose testicular uptake using positron emission tomography/computed tomography correlated with the results of a testicular sperm extraction procedure in the case of azoospermia (French prospective multicenter AZOPREDHISTOTEP study)? Eur J Nucl Med Mol Imaging. 2026 Apr;53(5):3283-3290. doi: 10.1007/s00259-025-07583-7. Epub 2025 Nov 21.

    PubMed 41269291

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing L-Threo DOPS in people with Multiple System Atrophy, run by University Hospital, Toulouse at 13 sites in France.

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 9 inclusion and 3 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 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in France. 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 combined 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.

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?

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?

Evaluate the efficacy of long term efficacy of L-threo DOPS (time frame: 12 weeks). 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 NCT02071459, 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 8 Apr 2026.

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