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Trial record 3 of 328 for:    CMT

Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A (UPACOMT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02600286
Recruitment Status : Terminated
First Posted : November 9, 2015
Last Update Posted : May 25, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital, Strasbourg, France

Brief 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 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.


Condition or disease Intervention/treatment Phase
CMT1A Drug: EllaOne Drug: EllaOne placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: LONG-TERM EFFECTS TOLERANCE AND THE Ulipristal Acetate IN DISEASE Charcot-MARIE-TOOTH TYPE OF 1A
Actual Study Start Date : October 23, 2015
Actual Primary Completion Date : November 2017
Actual Study Completion Date : November 2, 2017


Arm Intervention/treatment
Experimental: 1

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.
Drug: EllaOne
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental

Drug: EllaOne placebo
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental

Experimental: 2

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.
Drug: EllaOne
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental

Drug: EllaOne placebo
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental

Experimental: 3

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.
Drug: EllaOne
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental

Drug: EllaOne placebo
  • 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.
Other Names:
  • 1: Experimental
  • 2: Experimental
  • 3: Experimental




Primary Outcome Measures :
  1. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02600286


Locations
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France
Département de Neurologie Centre de Référence des Maladies Neuromusculaires Grand Est (CERNEST) Hôpital de Hautepierre
Strasbourg, France, 67098
Sponsors and Collaborators
University Hospital, Strasbourg, France
Investigators
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Study Director: Andoni Echaniz-Laguna, MD University Hospital, Strasbourg, France
Principal Investigator: Nicolas Collongues, MD University Hospital, Strasbourg, France

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Responsible Party: University Hospital, Strasbourg, France
ClinicalTrials.gov Identifier: NCT02600286     History of Changes
Other Study ID Numbers: 6100
First Posted: November 9, 2015    Key Record Dates
Last Update Posted: May 25, 2018
Last Verified: May 2018

Keywords provided by University Hospital, Strasbourg, France:
Physical medicine and rehabilitation
Neurology

Additional relevant MeSH terms:
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Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Ulipristal acetate
Contraceptive Agents, Female
Contraceptive Agents
Reproductive Control Agents
Physiological Effects of Drugs