Prophylaxis Impact on Quality of Life Impariment of HAE Patients with Lower Annual Base Attack Rates
Organizational Data
- DRKS-ID:
- DRKS00028582
- Recruitment Status:
- Recruiting ongoing
- Date of registration in DRKS:
- 2022-03-30
- Last update in DRKS:
- 2024-05-08
- Registration type:
- Prospective
Acronym/abbreviation of the study
PIQHAR
URL of the study
No Entry
Brief summary in lay language
PIQHAR is an observational study in patients with type 1 or type 2 Hereditary angioedema (HAE) who, despite a lower yearly attack rate, suffer from a clinically significant impairment in their quality of life. Patients will be enrolled after decision for treatment with lanadelumab (TAKHZYRO®) according to current product labelling was made. Comprehensive data from routine patient visits and patient questionnaires will be captured by study sites at enrollment and every six months thereafter up to month 24. As so far little clinical experience exists for this HAE patient group, the main study aim is to evaluate the efficacy of lanadelumab in improving health-related quality of life. Further aims are to evaluate the percentage of attack-free days while on different dosing intervals as well as the reduction of HAE attack frequency.
Brief summary in scientific language
Hereditary angioedema (HAE) is a rare disease caused by autosomal dominant mutations in the C1-esterase inhibitor (C1-INH) gene. With the introduction of new prophylactic therapies, goals for HAE treatment have recently evolved from treating the more severe and life-threatening attacks to preventing their occurrence. One of these novel therapies is lanadelumab (TAKHZYRO®). So far, the majority of real-world evidence for the use of lanadelumab has predominantly been collected in HAE patients with a high disease activity who were insufficiently controlled by other available treatment options. The PIQHAR study, as non-interventional, prospective, multicenter study, aims to collect real-world clinical evidence on the long-term, quality of life (QoL)-related Patient Reported Outcomes (PROs), effectiveness and HAE attack characteristics of lanadelumab in patients who experience less frequent HAE attacks (≤ 24 attacks/year), coupled with a considerable impairment in their quality of life (measured via validated questionnaires). Patients (12 years and older) will be enrolled at participating sites over a period of approximately 12 months and will be followed for up to 24 months after the enrollment date. Comprehensive data from routine patient visits will be captured by study sites at enrollment (prior to the first dose of lanadelumab) and every six months thereafter up to month 24. For the primary outcome measure, the Angioedema Quality of Life Questionnaire (AE-QoL), data will be additionally reported at month 1, 2, 3, 9, 15 and 21.
Health condition or problem studied
- ICD10:
- D84.1 - Defects in the complement system
- Healthy volunteers:
- No
Interventions, Observational Groups
- Arm 1:
- Patients with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling, a documented history of ≤ 24 attacks in the year prior to enrollment and a baseline AE-QoL Score of ≥ 30 points.
Endpoints
- Primary outcome:
- Effectiveness of lanadelumab to significantly improve health-related quality of life in HAE patients with less frequent HAE attacks, measured via the Angioedema Quality of Life Questionnaire (AE-QoL) and its minimal clinically important difference (MCID) of 6 points in the total score.
- Secondary outcome:
- - Effectiveness of lanadelumab q4w dosing interval compared to non-q4w dosing intervals; - Effectiveness of lanadelumab in reducing HAE attack frequency in real-world clinical practice.
Study Design
- Purpose:
- Prevention
- Retrospective/prospective:
- Prospective
- Study type:
- Non-interventional
- Longitudinal/cross-sectional:
- Longitudinal study
- Study type non-interventional:
- No Entry
Recruitment
- Recruitment Status:
- Recruiting ongoing
- Reason if recruiting stopped or withdrawn:
- No Entry
Recruitment Locations
- Recruitment countries:
-
- Germany
- Number of study centers:
- Multicenter study
- Recruitment location(s):
-
- Doctor's practice Deutschland
- Medical center Deutschland
- University medical center Deutschland
Recruitment period and number of participants
- Planned study start date:
- 2022-04-01
- Actual study start date:
- 2022-07-15
- Planned study completion date:
- 2026-07-15
- Actual Study Completion Date:
- No Entry
- Target Sample Size:
- 50
- Final Sample Size:
- No Entry
Inclusion Criteria
- Sex:
- All
- Minimum Age:
- 12 Years
- Maximum Age:
- no maximum age
- Additional Inclusion Criteria:
- - Confirmed diagnosis of HAE Type 1/2. - Voluntarily provision of written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study. - Initiation of treatment with lanadelumab in accordance with current product labelling. - Documented history of ≤ 24 attacks in the year prior to enrollment AND baseline AE-QoL Score of ≥ 30 points. - 12 years or older.
Exclusion Criteria
- Inability to provide written, signed, and dated informed consent. - Documented history of > 24 confirmed HAE attacks/year in the year prior to study enrollment OR baseline AE-QoL Score of < 30 points. - Participation in the study ruled out by the treating physician/investigator. - Pregnant or breastfeeding. - Current or planned participation in interventional studies.
Addresses
Primary Sponsor
- Address:
- Takeda GmbHByk Gulden Str. 278467 KonstanzGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
- Investigator Sponsored/Initiated Trial (IST/IIT):
- No
Contact for Scientific Queries
- Address:
- Takeda Pharma Vertrieb GmbH & Co. KGDr. Christoph HirchePotsdamer Str. 12510783 BerlinGermany
- Telephone:
- +49 162 / 2352202
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Contact for Public Queries
- Address:
- Sacura GmbHDr. Agnes MusiolTechnologiehof Mendelstraße 1148149 MünsterGermany
- Telephone:
- (+49) 251 - 980 1490
- Fax:
- (+49) 251 - 980 1491
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Principal Investigator
- Address:
- Takeda Pharma Vertrieb GmbH & Co. KGDr. Christoph HirchePotsdamer Str. 12510783 BerlinGermany
- Telephone:
- +49 162 / 2352202
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Sources of Monetary or Material Support
Commercial (pharmaceutical industry, medical engineering industry, etc.)
- Address:
- Takeda GmbHByk Gulden Str. 278467 KonstanzGermany
- Telephone:
- No Entry
- Fax:
- No Entry
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Ethics Committee
Address Ethics Committee
- Address:
- Ethik-Kommission der Landesärztekammer HessenHanauer Landstraße 15260314 Frankfurt am MainGermany
- Telephone:
- +49-69-97672209
- Fax:
- +49-69-97672377
- Contact per E-Mail:
- Contact per E-Mail
- URL:
- No Entry
Vote of leading Ethics Committee
- Vote of leading Ethics Committee
- Date of ethics committee application:
- 2021-07-20
- Ethics committee number:
- 2021-2576-evBO
- Vote of the Ethics Committee:
- Approved
- Date of the vote:
- 2022-01-07
Further identification numbers
- Other primary registry ID:
- No Entry
- EudraCT Number:
- No Entry
IPD - Individual Participant Data
- Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
- No
- IPD Sharing Plan:
- No Entry
Study protocol and other study documents
- Study protocols:
- No Entry
- Study abstract:
- No Entry
- Other study documents:
- No Entry
- Background literature:
- No Entry
- Related DRKS studies:
- No Entry
Publication of study results
- Planned publication:
- No Entry
- Publikationen/Studienergebnisse:
- No Entry
- Date of first publication of study results:
- No Entry
- DRKS entry published for the first time with results:
- No Entry
Basic reporting
- Basic Reporting / Results tables:
- No Entry
- Brief summary of results:
- No Entry