CMT1APhase 2UPACOMTNCT02600286
Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A
A Phase 2 study testing EllaOne in people with CMT1A, run by University Hospital, Strasbourg, France at 5 sites in France.
Trial summary
The disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness. It is autosomal dominant, associated with a duplication of the chromosome 17p11.2 region which leads to overexpression of the gene and the protein-peripheral… Read more about this study
- Who is it for?
- Ages 18 to 70 years, men only
- Conditions the study is for
- CMT1A
- What is being tested?
- EllaOne
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 5 sitesFrance
- Study dates
- 23 Oct 2015to 2 Nov 2017
- 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 18 to 70 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male 18-70 years
- CMT1A proven genetically (17p11.2 duplication)
- symptomatic CMT1A (MRC score <5 in at least one muscle group)
- Non severe axonal impairment (amplitude of the motor evoked potential on the median nerve and / or ulnar than 50% of normal)
- Subject contacted with a valid phone number
- Subject affiliated to a social security scheme
- Subject has been informed of the results of the medical examination prior
Who cannot join
- Another cause of neuropathy: Chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulin, B12 deficiency, hepatitis B / C, HIV, Lyme or poliomyelitis
- Liver failure
- Lapp lactase deficiency, malabsoprtion syndrome glucose / galactose
- Support long-term drug interacting with the CYP3A4
- Patients with indication against xylocaine adrenaline
- In the biopsy site: surgery, skin disease or local infection
Show all 13Show fewer
- Immunosuppression innate or acquired
- Hypersensitivity to the active substance / excipient
- uncontrolled severe asthma
- Treatment with vitamin C or vitamin B3 in the four weeks preceding randomization
- Orthopaedic surgery of the lower limbs in the 6 months prior to randomization or planned
- Against indication xylocaine adrenaline
- Malfunction of the innate or acquired coagulation
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
France5 sites
Service d'Explorations et pathologies neuro- musculaires
Besançon, 25030
Département de Neurologie
Dijon, 21079
Département de Neurologie
Nancy, 54035
Unité de pathologie neuro-musculaire
Paris, 75561
Département de Neurologie Centre de Référence des Maladies Neuromusculaires Grand Est (CERNEST) Hôpital de Hautepierre
Strasbourg, 67098
About this study
Official title: LONG-TERM EFFECTS TOLERANCE AND THE Ulipristal Acetate IN DISEASE Charcot-MARIE-TOOTH TYPE OF 1A
The disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness. It is autosomal dominant, associated with a duplication of the chromosome 17p11.2 region which leads to overexpression of the gene and the protein-peripheral myelin protein-22 (PMP22), a major component of peripheral myelin.
In animals and humans, PMP22 mRNA level of glutathione S-transferase theta 2 and Cathepsin A (markers of oxidative stress), detected in a skin biopsy are markers that may play a role in the prognosis evolution of the disease. Furthermore, several studies have shown that the administration of progesterone increased the expression of PMP22 gene (measured in a skin biopsy) and worsening symptoms. In contrast, anti-progestins reduce the synthesis of PMP22 and improve symptoms in rat CMT1A.
The long-term safety of anti-progesterone was evaluated for mifepristone (RU486) ulipristal acetate and (EllaOne®). Few side effects have been reported including a few cases of endometrial hyperplasia reversible upon discontinuation of treatment. With the RU486, rare cases of adrenal androgen and failure have been observed. However, EllaOne® has low antagonistic action on the glucocorticoid receptor and no action on androgen receptors. The investigators therefore believe that it will be well tolerated in humans and will reduce the synthesis of PMP22 and the action of oxidative stress by improving disability of patients.
- Conditions
- CMT1A
- Being tested
- EllaOne, EllaOne placebo
- Keywords
- Physical medicine and rehabilitation, Neurology
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EllaOneDrug
Also called 1: Experimental, 2: Experimental, 3: Experimental
- 5 mg/per os of EllaOne every day through 12 months.
- 10 mg/per os of EllaOne every day through 12 months.
- EllaOne placebo/per os every day through 12 months.
EllaOne placeboDrug
Also called 1: Experimental, 2: Experimental, 3: Experimental
- 5 mg/per os of EllaOne every day through 12 months.
- 10 mg/per os of EllaOne every day through 12 months.
- EllaOne placebo/per os every day through 12 months.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Arm (1) will be randomised to receive either :
* 5 mg/per os of EllaOne every day through 12 months.
* 10 mg/per os of EllaOne every day through 12 months.
* EllaOne placebo/per os every day through 12 months. Receives: Drug: EllaOne; Drug: EllaOne placebo |
| 2 | Experimental | Arm (2) will be randomised to receive either :
* 5 mg/per os of EllaOne every day through 12 months.
* 10 mg/per os of EllaOne every day through 12 months.
* EllaOne placebo/per os every day through 12 months. Receives: Drug: EllaOne; Drug: EllaOne placebo |
| 3 | Experimental | Arm (3) will be randomised to receive either :
* 5 mg/per os of EllaOne every day through 12 months.
* 10 mg/per os of EllaOne every day through 12 months.
* EllaOne placebo/per os every day through 12 months. Receives: Drug: EllaOne; Drug: EllaOne placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Analysis of the effectiveness of Ellaone. This will be assessed by the production of RNA PMP22 using skin biopsy.
Measured: 12 months after treatment with EllaOne
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2015 (estimated)
- Study start23 Oct 2015
- Primary completionNov 2017
- Study completion2 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CMT1AStudies of the same condition, open and under way.
- More trials run by University Hospital, Strasbourg, FranceThe 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
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Strasbourg, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Andoni Echaniz-Laguna, MD, Study director, University Hospital, Strasbourg, FranceNicolas Collongues, MD, Principal investigator, University Hospital, Strasbourg, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02600286
- Sponsor's protocol ID
- 6100
- Acronym
- UPACOMT
- Status last verified
- Jul 2022
- First submitted
- 4 Nov 2015
- Last updated
- 25 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- LONG-TERM EFFECTS TOLERANCE AND THE Ulipristal Acetate IN DISEASE Charcot-MARIE-TOOTH TYPE OF 1A
- ClinicalTrials.gov ID
- NCT02600286
- Acronym / sponsor ID
- UPACOMT / 6100
- Conditions
- CMT1A
- Keywords
- Physical medicine and rehabilitation, Neurology
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 23 participants (actual)
- Lead sponsor
- University Hospital, Strasbourg, FranceUniversity, hospital or other
- Study start
- 23 Oct 2015 (actual)
- Primary completion
- Nov 2017 (actual)
- Study completion
- 2 Nov 2017 (actual)
- First posted
- 9 Nov 2015 (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 disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness. It is autosomal dominant, associated with a duplication of the chromosome 17p11.2 region which leads to overexpression of the gene and the protein-peripheral myelin protein-22 (PMP22), a major component of peripheral myelin.
In animals and humans, PMP22 mRNA level of glutathione S-transferase theta 2 and Cathepsin A (markers of oxidative stress), detected in a skin biopsy are markers that may play a role in the prognosis evolution of the disease. Furthermore, several studies have shown that the administration of progesterone increased the expression of PMP22 gene (measured in a skin biopsy) and worsening symptoms. In contrast, anti-progestins reduce the synthesis of PMP22 and improve symptoms in rat CMT1A.
The long-term safety of anti-progesterone was evaluated for mifepristone (RU486) ulipristal acetate and (EllaOne®). Few side effects have been reported including a few cases of endometrial hyperplasia reversible upon discontinuation of treatment. With the RU486, rare cases of adrenal androgen and failure have been observed. However, EllaOne® has low antagonistic action on the glucocorticoid receptor and no action on androgen receptors. The investigators therefore believe that it will be well tolerated in humans and will reduce the synthesis of PMP22 and the action of oxidative stress by improving disability of patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: EllaOne, Drug: EllaOne placeboArm (1) will be randomised to receive either : * 5 mg/per os of EllaOne every day through 12 months. * 10 mg/per os of EllaOne every day through 12 months. * EllaOne placebo/per os every day through 12 months. |
| 2 | Experimental | Drug: EllaOne, Drug: EllaOne placeboArm (2) will be randomised to receive either : * 5 mg/per os of EllaOne every day through 12 months. * 10 mg/per os of EllaOne every day through 12 months. * EllaOne placebo/per os every day through 12 months. |
| 3 | Experimental | Drug: EllaOne, Drug: EllaOne placeboArm (3) will be randomised to receive either : * 5 mg/per os of EllaOne every day through 12 months. * 10 mg/per os of EllaOne every day through 12 months. * EllaOne placebo/per os every day through 12 months. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male 18-70 years
- CMT1A proven genetically (17p11.2 duplication)
- symptomatic CMT1A (MRC score <5 in at least one muscle group)
- Non severe axonal impairment (amplitude of the motor evoked potential on the median nerve and / or ulnar than 50% of normal)
- Subject contacted with a valid phone number
Key exclusion criteria (13 listed)
- Another cause of neuropathy: Chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulin, B12 deficiency, hepatitis B / C, HIV, Lyme or poliomyelitis
- Liver failure
- Lapp lactase deficiency, malabsoprtion syndrome glucose / galactose
- Support long-term drug interacting with the CYP3A4
- Patients with indication against xylocaine adrenaline
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 |
|---|---|---|
| EllaOneAlso: 1: Experimental, 2: Experimental, 3: Experimental* 5 mg/per os of EllaOne every day through 12 months. * 10 mg/per os of EllaOne every day through 12 months. * EllaOne placebo/per os every day through 12 months. | Drug | 1, 2, 3 |
| EllaOne placeboAlso: 1: Experimental, 2: Experimental, 3: Experimental* 5 mg/per os of EllaOne every day through 12 months. * 10 mg/per os of EllaOne every day through 12 months. * EllaOne placebo/per os every day through 12 months. | Drug | 1, 2, 3 |
Primary outcome
Analysis of the effectiveness of Ellaone. This will be assessed by the production of RNA PMP22 using skin biopsy.
Time frame: 12 months after treatment with EllaOne
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2022.
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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.
Andoni Echaniz-Laguna, MD study director
University Hospital, Strasbourg, France
Nicolas Collongues, MD principal investigator
University Hospital, Strasbourg, France
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing EllaOne in people with CMT1A, run by University Hospital, Strasbourg, France at 5 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 7 inclusion and 13 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: stopped early, as of the sponsor's last update on 25 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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. 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?
The study was stopped early. 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?
Analysis of the effectiveness of Ellaone. This will be assessed by the production of RNA PMP22 using skin biopsy. (time frame: 12 months after treatment with EllaOne). 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 25 Jul 2022.