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Copper Histidine

Phase 3

Menkes Disease | Small molecule | Other |Fortress Biotech, Inc.|Last Updated: Nov 19, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment93
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00811785Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper DeficiencyPHASE3 COMPLETED 93 — — Feb 27, 2009Aug 28, 2020Nov 19, 20201 United States
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Study Endpoints
Primary Endpoints
To assess neurological improvement in patients with OHS or unexplained copper deficiency treated with subcutaneous CuHis injections.
Three years

Neurological Improvement: reduction in dysautonomia symptoms in OHS, and improved nerve conduction tests in unexplained copper deficiency

Secondary Endpoints
To assess survival in classic Menkes disease subjects treated with subcutaneous CuHis injections in comparison with classic Menkes patients who did not receive any type of copper treatment.
Continuously
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Interventions
NameTypeDescription
Copper HistidineDRUGDaily subcutaneous injections for three years
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Eligibility Criteria
Age RangeN/A — 80 Years
SexALL
Healthy VolunteersNo
Study Sites1

* INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Provision of signed and dated informed consent form by parent or legal guardian, or the subject himself/herself. 2. Male or female, aged 0 to 80 years. 3. Diagnosed...

Countries:United States
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