A Long Term Safety Study of BCX7353 in Hereditary Angioedema

Browse trials for

• Angioedema 164
• Angioedemas, Hereditary 171

Keywords

• BCX7353
• Hereditary Angioedema
• HAE

Eligibility criteria

Study arms

• Experimental
  BCX7353 150 mg once daily

  Drug: BCX7353

Interventions

• DrugBCX7353

  BCX7353 mg oral capsules administered once daily

Primary outcomes

1. Safety & Tolerability

   The number and percentage of subjects with treatment-emergent adverse events.

   Time frame: Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).

Secondary outcomes

1. Incidence of Acute Attacks of Angioedema in Subjects During Treatment

   Number of 'adjusted' attacks were assessed. Adjusted attacks included at least 1 symptom of swelling, had a response of 'no' to the diary question, 'In retrospect, could there be an alternative explanation for your symptoms other than an HAE attack (i.e., allergic reaction, viral cold etc.)?', and were considered unique (attack began \> 24 hours from the end of the prior attack). Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.

   Time frame: Up to 96 weeks (US) / 216 weeks (ROW)

2. The Durability of Response to Treatment

   To evaluate if the rate of attacks remains consistent (durable) over time, the monthly attack rate was assessed at 0 to 24 weeks, 24 to 48 weeks, 48 to 96 weeks and 96 weeks until the end of the study. Monthly attack rate was defined as the total number of adjusted HAE attacks experienced during the treatment period adjusted for the length of a month (defined as 28 days) and the number of days the subject was on treatment during that month.

   Time frame: Up to 96 weeks (US) / 216 weeks (ROW)

3. Patient Reported Quality of Life (QoL) During Treatment

   Quality of Life (QoL) specific to hereditary angioedema (HAE) was assessed at baseline and at each study visit until the end of the study. The questionnaire (i.e. AE-QoL) consisted of 17 questions spanning 4 domains (functioning, fatigue/mood, fear/shame, and nutrition). Each AE-QoL question had 5 answer options (scored 1-5), with lower and higher scores indicting less and more adverse impact, respectively. Per-subject scores for each domain were computed using the appropriate scoring algorithm applied to the question response scores for each domain. Per-subject total scores (including all 4 domains) were similarly computed using the question response scores for all 17 questions. The outputs from the scoring algorithm were normalized on a scale ranging from 0 (less adverse impact) to 100 (most adverse impact). The Mean change from baseline (CFB) in AE-QoL total score over time is presented below.

   Time frame: Up to 96 weeks (US) / 216 weeks (ROW)

4. Patient's Satisfaction With Medication During Long Term Administration of Berotralstat

   The Treatment Satisfaction Questionnaire for Medication (TSQM) was completed by subjects at baseline and at each study visit until the end of the study. TSQM scores consisted of 14 items of which 13 items were made up of 3 specific scales (Effectiveness, Side Effects, and Convenience) and 1 global satisfaction scale (Global Satisfaction). At baseline, TSQM questionnaires were completed based on subject's satisfaction with usual medications. At all other time points for collection of TSQM, subjects were asked about their level of satisfaction or dissatisfaction with the study drug. Scale scores were calculated for each scale and were transformed into scores ranging from 0 to 100, with higher scores indicating higher satisfaction. TSQM score and corresponding change from baseline values were calculated at each visit. Note: Subjects in Hong Kong did not complete the TSQM.

   Time frame: Up to 96 weeks (US) / 216 weeks (ROW)

Baseline characteristics

Publications

• Riedl MA, Neville D, Cloud B, Desai B, Bernstein JA. Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives. Allergy Asthma Proc. 2022 Sep 1;43(5):397-405. doi: 10.2500/aap.2022.43.220050. Epub 2022 Jul 12. PubMed 35820771 ↗
• Farkas H, Stobiecki M, Peter J, Kinaciyan T, Maurer M, Aygoren-Pursun E, Kiani-Alikhan S, Wu A, Reshef A, Bygum A, Fain O, Hagin D, Huissoon A, Jesenak M, Lindsay K, Panovska VG, Steiner UC, Zubrinich C, Best JM, Cornpropst M, Dix D, Dobo SM, Iocca HA, Desai B, Murray SC, Nagy E, Sheridan WP. Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study. Clin Transl Allergy. 2021 Jun;11(4):e12035. doi: 10.1002/clt2.12035. PubMed 34161665 ↗

Study documents

• Study protocol · Mar 23, 2020
• Statistical analysis plan · Feb 26, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No