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Trial record 6 of 6 for:    Neurodegeneration with Brain Iron Accumulation (NBIA)

Compassionate Use of Deferiprone in Patients With PKAN

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ClinicalTrials.gov Identifier: NCT02635841
Expanded Access Status : Available
First Posted : December 21, 2015
Last Update Posted : June 28, 2017
Sponsor:
Information provided by (Responsible Party):
ApoPharma

Brief Summary:
Patients with pantothenate kinase-associated neurodegeneration (PKAN) who have completed the ApoPharma-sponsored study TIRCON2012V1-EXT and who wish to continue to take deferiprone will be offered the opportunity to receive it on a compassionate basis. Patients will be followed locally by their own neurologist or other appropriate specialist.

Condition or disease Intervention/treatment
Pantothenate Kinase-Associated Neurodegeneration Drug: Deferiprone

Detailed Description:
Patients with PKAN who have completed both the randomized, blinded, placebo-controlled trial TIRCON2012V1 and its follow-up, the single-arm open-label extension study TIRCON2012V1-EXT, may enroll in this compassionate-use/expanded access program in order to continue receiving the iron chelator deferiprone. Participants in this program will be followed locally by their own neurologist or other appropriate specialist. Patients will take deferiprone oral solution 80 mg/mL at the same dose they were prescribed in the TIRCON2012V1-EXT clinical trial. Safety monitoring of the patients will be performed. At a minimum, the program will continue until the analysis of the currently blinded TIRCON2012V1 trial is completed (approximately Q4 2016), at which time it will be re-evaluated.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration



Intervention Details:
  • Drug: Deferiprone
    Deferiprone oral solution (80 mg/mL)
    Other Name: Ferriprox

Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

Inclusion Criteria:

  1. Completed study TIRCON2012V1-EXT
  2. Able to adhere to program appointments and evaluation schedules
  3. Females of childbearing potential must have a negative pregnancy test result at the start of the program, unless the treating physician determines there is no reasonable risk of pregnancy because of significant incapacity. If applicable, they must meet at least one of the following criteria: Use an effective method of contraception during the program and for 30 days following the last dose of deferiprone, OR participate in a non-heterosexual lifestyle, OR have a male sexual partner who has been sterilized, OR be abstinent during the program and for at least 30 days after its completion.
  4. Fertile sexually active heterosexual males must agree to use an effective method of contraception during the program and for at least 30 days after its completion
  5. Patients and/or their authorized legal representatives must provide signed and dated written informed consent, and minors must additionally sign an assent form as per local regulatory requirements.

Exclusion Criteria:

Patients will be excluded from taking part in the compassionate use program if the treating physician determines the presence of any medical, psychological, or psychiatric condition which in his/her opinion would cause participation in the program to be unwise


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): NCT02635841


Contacts
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Contact: Caroline Fradette, PhD 1-416-401-7543 cfradett@apopharma.com
Contact: Fernando Tricta, MD 1-416-401-7332 ftricta@apopharma.com

Sponsors and Collaborators
ApoPharma

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Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT02635841     History of Changes
Other Study ID Numbers: TIRCON2012V1-COMP
First Posted: December 21, 2015    Key Record Dates
Last Update Posted: June 28, 2017
Last Verified: November 2015
Keywords provided by ApoPharma:
Pantothenate Kinase-Associated Neurodegeneration
PKAN
Neurodegeneration with Brain Iron Accumulation
NBIA
Deferiprone
Ferriprox
Additional relevant MeSH terms:
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Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
Brain Diseases
Pathologic Processes
Basal Ganglia Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action