EUCTR2020-000105-92-HUActive, not recruitingPhase 1
A Randomized, Double-Blind, Placebo-Controlled Dose Range Finding Study with Open-Label Extension to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LMI070/branaplam when Administered as Weekly Oral Doses in Participants with Early Manifest Huntington’s Disease
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- ovartis Pharma AG
- Enrollment
- 75
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •1. Signed informed consent must be obtained prior to participation in the study
- •2. Must be capable of providing informed consent
- •3. Clinically diagnosed Stage 1 or 2 HD with a diagnostic confidence interval (DCL) = 4 and a UHDRS Total Functional Capacity (TFC) >8 at screening
- •4. Genetically confirmed HD, with presence of =40 CAG repeats in the huntingtin gene
- •- For participants without prior documentation, a sample must be sent to the CAG lab vendor and confirmation of the CAG repeat length for these participants must be obtained prior to randomization
- •- For participants with previously existing documentation of their CAG repeat length, it is acceptable to use this prior data to qualify for randomization. These participants must also submit a sample for CAG repeat testing to be conducted by the central study laboratory.
- •5. Male and female participants between 25 to 75 years of age, inclusive, on the day of Informed Consent signature
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 50
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 25
Exclusion Criteria
- •1. Use of other investigational drugs within 5 half-lives of the first dose of study drug or within 30d, whichever is longer.
- •2. Prior participation in clinical trial investigating a huntingtin-lowering therapy (unless participant received only placebo)
- •3. History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes
- •4. Participants taking medications prohibited by the protocol (see
- •5. Any medical history, lumbar surgery or condition that would interfere with the ability to complete the protocol specified assessments
- •6. History of malignancy of any organ system, treated or untreated, regardless of whether there is evidence of local recurrence or metastases
- •7. Participant has other severe, acute or chronic medical conditions including unstable psychiatric conditions or laboratory abnormalities that in the opinion of the Investigator may increase the risk associated with study participation, or that may interfere with the interpretation of study results
- •8. Score yes on item 4 or 5 of the C-SSRS, if this ideation occurred in the past 6m from screening or yes on any item of the Suicidal Behavior section, except for the Non-Suicidal Self-Injurious Behavior, if this behavior occurred in the past 2y
- •9. Pregnant or nursing (lactating) women. WOCB potential should not become pregnant during the study or 7 months after stopping study medication.
- •10. Sexually active males unwilling to use a condom together with a spermicidal agent during intercourse while taking study drug and for 120d (in total) after the last dose of the study drug. A condom is required to be used also by vasectomized men as well during intercourse with a male partner of the study subject in order to prevent delivery of the genotoxic drug via semen. Male participants must not donate sperm for the time period specified above.
- •11. Women of child-bearing potential, defined as all heterosexually
- •active women physiologically capable of becoming pregnant, unless they are using one highly effective methods of contraception during dosing and for 7 months after stopping the study medication. Highly effective methods of birth control are those methods that have a less than 1% chance of an unwanted pregnancy for 1 year (see protocol).
- •In addition to one highly effective method of contraception, a condom is required for all male partners of female participants to prevent fathering a child AND to prevent exposure of study treatment via vaginal fluid to your partner, until at least 7 months following the last dose of study treatment. WOCB potential must not donate their eggs for 7 months after the last dose of study treatment.
- •Total abstinence, Periodic abstinence and withdrawal are NOT acceptable methods of contraception for heterosexually active
- •participants.. Oral contraception cannot be considered due to potential decreased efficacy as potential DDI with branaplam.
- •In case local regulations are more stringent than the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the ICF.
- •12. History of:
- •- Gene therapy or cell transplantation or any other experimental brain surgery
- •- Hepatitis B or hepatitis C or serologic evidence for active viral hepatitis
- •- Immunodeficiency diseases, including a positive HIV test
- •- History or current evidence of drug or alcohol abuse in the 12m prior to screening, as defined by the DSM-V criteria for substance abuse. For former abusers, abstinence
Investigators
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