Novartis decision based on Cohort 1 results

The purpose of this study was to determine if SOM230 is safe and effective for the treament of cluster headache.

The purpose of this non-confirmatory study was to determine if SOM230 has adequate efficacy and safety to warrant further clinical development in cluster headache (CH). This study was a sequential design of SOM230 vs. Placebo.

Inclusion Criteria:

• Subject is male or female age 18-65 inclusive.
• Written informed consent must be obtained before any assessment is performed.
• Subjects must have established diagnosis of episodic cluster headaches (CH) or chronic CH, averaging 2-6 headache attacks per day each lasting at least 45 minutes without treatment, not to exceed 6 attacks per day within the last year.
• Able to communicate well with the investigator, to understand and comply with the requirements of the study, as well as accepting NOT to share any study information through social media during their participation in the study.
• Subject is able to self-inject medication subcutaneously or have the assistance of a partner on an out-patient basis.

Exclusion Criteria:

• Subjects that have a history of greater than 6 CH attacks per day within the last year.

• Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during the duration dosing of the study treatment. Or men who are sexually active with women of child bearing potential, unless the male subjects always use condoms during the study.

• History of multiple and recurring allergies or allergy to the investigational compound/compound class being used in this study.

• Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations.

• A history of clinically significant heart diseases, ECG abnormalities, continued use of drugs known to prolong QTc during the study conduct, or any of the following ECG abnormalities at screening or baseline:

  • QTcF > 450 msec (males)
  • QTcF > 460 msec (females)

• Uncontrolled diabetes as evidenced by screening HbA1c > 8.0%

• A positive Hepatitis B surface antigen or Hepatitis C test result.

• A positive pregnancy test or lactating mothers.

• History of drug or alcohol abuse within the 12 months prior to dosing other than prescription medications to manage their CH attacks, or evidence of such abuse as indicated by the laboratory assays conducted during screening.

• Significant acute illness which has not resolved within two (2) weeks prior to initial dosing.

• Any surgical or medical condition which might significantly jeopardize the subject's safety in case of participation in the study. The Investigator should make this determination in consideration of the subject's medical history and/or clinical or laboratory evidence of any of the following:

• Liver disease or liver injury as indicated by abnormal liver function tests. ALT (SGPT), AST (SGOT), γ-GT, alkaline phosphatase and serum bilirubin will be tested.

• ALT must be within the normal range

• Serum bilirubin must not exceed 1.2 x ULN

• γ-GT, AST and alkaline phosphatase must not exceed 2 x ULN [If necessary, laboratory testing may be repeated on one occasion (as soon as possible) prior to treatment, to rule out any laboratory error]

• Acute cholecystitis or symptomatic cholelithiasis in subjects without H/O cholecystectomy

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Safety analysis set excluded 2 subjects due to not being dosed.

Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 26 months.

Terminated (Non-efficacy in first Phase 2a cohort.)