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Hereditary Angioedema (HAE)Phase 3NCT02741596

Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

A Phase 3 study testing DX-2930 in people with Hereditary Angioedema (HAE), run by Shire at 43 sites in 7 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in participants with Type I and Type II HAE. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Hereditary Angioedema (HAE)
What is being tested?
DX-2930
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
212 participants
Where
43 sitesin 7 countries
Study dates
26 May 2016to 31 Oct 2019
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male and female HAE participants who are 12 years of age or older at the time of screening
  • Documented diagnosis of HAE (Type I or II) based on
  • Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling episodes without accompanying urticaria).
  • Diagnostic testing results obtained during screening (or a prior DX-2930 study) that confirm HAE Type I or II: C1 inhibitor (C1-INH) functional level less than (<) 40 percentage (%) of the normal level. Participants with functional C1-INH level 40-50% of the normal level may be enrolled if they also have a C4 level below the normal range. Participants may be retested if results are incongruent with clinical history or believed by the investigator to be confounded by long-term prophylactic (LTP) use. (It is understood that C1-INH therapy may alter the lab results of C1-INH assessments; therefore, the investigator's discretion in collaboration with Medical Monitor is advised for proper documentation of eligibility).
  • At least one of the following: Age at reported onset of first angioedema symptoms less than or equal to (<=) 30 years, a family history consistent with HAE Type I or II, or C1q within normal range.
  • A historical baseline HAE attack rate of at least 1 attack per 12 weeks
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  • Adult participants and caregivers of participants under the age of 18 are willing and able to read, understand, and sign an informed consent form. Participants age 12 to 17, whose caregiver has provided informed consent, are willing and able to read, understand and sign an assent form.
  • Males and females who are fertile and sexually active must adhere to contraception requirements for the duration of the study as
  • Females (NOTE: Female rollover participants (those who previously participated in Study DX-2930-03 [NCT02586805]) of childbearing potential may continue to use the birth control method used during Study DX-2930-03 (NCT02586805).) of childbearing potential must agree to be abstinent or it is recommended to use highly effective forms of contraception from the screening period through 30 days after the final study visit. This includes stable doses (for 3 months prior to study screening) of combined estrogen and progestin-containing hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), progestin-only hormonal contraception associated with inhibition of ovulation, intra-uterine device (IUD, all types) or intrauterine hormone releasing systems (IUS). Notes: 1) A female whose male partner has had a vasectomy must agree to use one additional form of medically acceptable contraception. 2) Use of a male condom with or without spermicide or cervical cap, diaphragm or sponge with spermicide or a combination (double barrier methods) are not considered highly effective.
  • Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal for at least 12 months do not require contraception during the study.
  • Males, including males who are surgically sterile (post vasectomy), with female partners of childbearing potential must agree to be abstinent or else use a medically acceptable form of contraception from the screening period through 60 days after the final study visit.

Who cannot join

  • Discontinued from DX-2930-03 (NCT02586805) after enrollment for any reason.
  • If rolling over from DX-2930-03 (NCT02586805), presence of important safety concerns that would preclude participation in this study.
  • Concomitant diagnosis of another form of chronic, recurrent angioedema, such as acquired angioedema (AAE), HAE with normal C1 inhibitor (also known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria.
  • Dosing with an investigational drug (not including DX-2930 or other HAE therapies) or exposure to an investigational device within 4 weeks prior to screening.
  • Exposure to angiotensin-converting enzyme (ACE) inhibitors within 4 weeks prior to screening or any newly initiated or dose modification of estrogen-containing medications with systemic absorption (such as oral contraceptives or hormonal replacement therapy) 3 months prior to the screening visit.
  • Unwilling to discontinue use of long-term prophylactic therapy for HAE (C1-INH, attenuated androgens, or anti-fibrinolytics) within 3 weeks after starting DX-2930 treatment.
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  • Any of the following liver function test abnormalities: alanine aminotransferase (ALT) > 3x upper limit of normal, or aspartate aminotransferase (AST) > 3x upper limit of normal, or total bilirubin > 2x upper limit of normal (unless the bilirubin elevation is a result of Gilbert's Syndrome).
  • Pregnancy or breastfeeding.
  • Participant has any condition that, in the opinion of the investigator or Sponsor, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results (eg, history of substance abuse or dependence, a significant pre-existing illness or other major comorbidity that the investigator considers may confound the interpretation of study results).

Trial locations and contacts

43 sites in 7 countries.

43 sites in 7 countries

United States31 sites

  • University of Michigan

    Ann Arbor, Michigan, 48106

  • Austin Regional Clinic

    Austin, Texas, 78731

  • Hudson-Essex Allergy, LLC

    Belleville, New Jersey, 07109

  • Clinical Research Center of Alabama

    Birmingham, Alabama, 35209

  • Massachusetts General Hospital

    Boston, Massachusetts, 02421

  • IMMUNOe Research Centers

    Centennial, Colorado, 80112

  • Clinical Research Center of Charlotte

    Charlotte, North Carolina, 28277

  • Institute Asthma and Allergy

    Chevy Chase, Maryland, 20815

  • Bernstein Clinical Research Center, LLC

    Cincinnati, Ohio, 45231

  • Asthma and Allergy Associates, PC

    Colorado Springs, Colorado, 80907

  • Optimed Research, LTD.

    Columbus, Ohio, 43235

  • AARA Research Center

    Dallas, Texas, 75231

  • Intermountain Clinical Research

    Draper, Utah, 84020

  • Duke Asthma, Allergy, and Airway Center

    Durham, North Carolina, 27705

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • Medical College of Wisconsin, Childrens Hospital

    Milwaukee, Wisconsin, 53226

  • Winthrop University Hospital

    Mineola, New York, 11501

  • Allergy Associates of Utah

    Murray, Utah, 84107

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Atlantic Research Center, LLC

    Ocean City, New Jersey, 07712

  • Midwest Immunology Clinic

    Plymouth, Minnesota, 55446

  • Virginia Commonwealth University

    Richmond, Virginia, 23219

  • University of California San Diego

    San Diego, California, 92122

  • AIRE Medical of Los Angeles

    Santa Monica, California, 90404

  • Medical Research of Arizona

    Scottsdale, Arizona, 85251

  • Premier Clinical Research

    Spokane, Washington, 99202

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • University of South Florida

    Tampa, Florida, 33613

  • Toledo Institute of Clinical REsearch

    Toledo, Ohio, 43617

  • Allergy & Asthma Clinical Research

    Walnut Creek, California, 94598

Other trials in United States

Canada4 sites

  • University of Alberta Hospital

    Edmonton, Alberta, T6G 2V2

  • Yang Medicine

    Ottawa, Ontario, K1G 6C6

  • Clinique Spécialisée en Allergie de la Capitale

    Québec, Quebec, G1V 4M6

  • Gordon Sussman Clinical Research, Inc.

    Toronto, Ontario, M4V 1R2

Other trials in Canada

Germany4 sites

  • Charité - Universitätsmedizin Berlin

    Berlin, 10117

  • Universitätsklinikum Frankfurt

    Frankfurt, 60596

  • Hautklinik der Universitätsmedizin Mainz

    Mainz, 55101

  • HZRM Hamophilie Zentrum Rhein Main GmbH

    Mörfelden-Walldorf, 64546

Other trials in Germany

Italy1 site

  • University of Milan Luigi Sacco Hospital

    Milan, 20157

Other trials in Italy

Jordan1 site

  • Triumpharma Clinical Evaluation Centre

    Amman, 11941

Other trials in Jordan

Puerto Rico1 site

  • Adler Medical Plaza

    San Juan, 00918

Other trials in Puerto Rico

United Kingdom1 site

  • Royal London Hospital

    London, E1 1BB

Other trials in United Kingdom

About this study

Official title: HELP Study ExtensionTM: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of DX-2930 for Prevention Against Acute Attacks of Hereditary Angioedema (HAE)

This study is an open-label, long term safety and efficacy study to evaluate DX-2930 in preventing acute angioedema attacks in participants with Type I and Type II HAE.

Being tested
DX-2930
Keywords
DX-2930(SHP643), Dyax, Hereditary Angioedema
Medical subject headings
Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DX-2930Drug

    Participants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.

  • DX-2930Drug

    Participants who were not participants in DX-2930-03 will receive 300 milligram (mg) DX-2930 subcutaneous injection once in every 2 weeks until the end of the treatment period (up to 924 days).

Study groups in NCT02741596
Study groupRoleWhat they receive
Rollover ParticipantsExperimentalParticipants who rollover from the DX-2930-03 study will receive 300 milligram (mg) DX-2930 subcutaneous injection at Day 0 followed by second dose following the first HAE attack and then once in every 2 weeks until the end of the treatment period (up to 924 days). A wash-out period of a minimum of 10 days and a maximum of 18 days is required between subsequent administrations.
Receives: Drug: DX-2930
Non-rollover ParticipantsExperimentalParticipants who were not participants in DX-2930-03 will receive 300 milligram (mg) DX-2930 subcutaneous injection once in every 2 weeks until the end of the treatment period (up to 924 days).
Receives: Drug: DX-2930

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    An adverse event (AE) was any untoward medical occurrence in a clinical trial Participant whether or not it appeared to have a causal relationship with the treatment administered. Treatment-emergent AEs were defined as AEs with onset at the time of or following the first exposure to open-label DX-2930 in this study, or medical conditions present prior to the start of treatment but increasing in severity or relationship at the time of or following the start of treatment.

    Measured: From start of the study up to follow-up (Day 952)

Secondary outcomes (5)

  1. Rate of Investigator Confirmed Hereditary Angioedema (HAE) Attacks During the Treatment Period

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. The treatment period investigator-confirmed HAE attack rate was calculated for each participant as the number of investigator-confirmed HAE attacks occurring during the treatment period (regular dosing stage of the treatment period for rollover participants) divided by the number of days the participant contributed to the treatment period multiplied by 28 days. Rate of investigator-confirmed HAE attacks during the treatment period was reported.

    Measured: Up to Day 924

  2. Rate of Investigator-Confirmed Hereditary Angioedema (HAE) Attacks Requiring Acute Treatment During the Treatment Period

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Rate of investigator-confirmed HAE attacks requiring acute treatment for each participant as the number of investigator-confirmed HAE attacks occurring during the treatment period (regular dosing stage of the treatment period for rollover participants) divided by the number of days the participant contributed to the treatment period multiplied by 28 days. Rate of investigator-confirmed HAE attacks requiring acute treatment during the treatment period was reported.

    Measured: Up to Day 924

  3. Rate of Moderate or Severe Hereditary Angioedema (HAE) Attacks During the Treatment Period

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Moderate and severe investigator-confirmed HAE attacks were the attacks that were moderate or severe as per the HAE attack assessment and reporting procedures (HAARP) defined severity. The overall severity of attack was determined by the investigator using following definitions: mild (transient or mild discomfort), moderate (mild to moderate limitation in activity), severe (marked limitation in activity). Rate of moderate or severe HAE attacks during the treatment period was reported.

    Measured: Up to Day 924

  4. Rate of High-Morbidity Hereditary Angioedema (HAE) Attacks During the Treatment Period

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. High-morbidity Hereditary Angioedema (HAE) attack was defined as any attack that had at least one of the following characteristics: severe, resulted in hospitalization (except hospitalization for observation lesser than [<] 24 hours), hemodynamically significant (systolic blood pressure <90 millimeter of mercury [mmHg], required intravenous hydration, or associated with syncope or near-syncope) or laryngeal edema. Number of high-morbidity HAE attacks during the treatment period was analyzed and reported using the methods for the overall number of investigator-confirmed HAE attacks with the exception of the monthly line graphs. Rate of high-morbidity HAE attacks during the treatment period was reported.

    Measured: Up to Day 924

  5. Time to First Investigator-Confirmed Hereditary Angioedema (HAE) Attacks in Rollover Participants

    HAE attack was defined as a discrete episode during which the participant progressed from no angioedema to symptoms of angioedema. Time to first investigator-confirmed HAE attack was calculated from the time of first open-label dose to the start time of the first investigator-confirmed HAE attack. Time to the first investigator-confirmed HAE attack was analyzed and reported only in rollover safety population.

    Measured: Up to Day 924

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Apr 2016 (estimated)
  2. Study start26 May 2016
  3. Primary completion31 Oct 2019
  4. Study completion31 Oct 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Prevention
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
212 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02741596
Sponsor's protocol ID
DX-2930-04
Other IDs
2015-005255-27, 2015
Status last verified
May 2021
First submitted
8 Apr 2016
Last updated
8 Jun 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Publications

  • Craig T, Tachdjian R, Bernstein JA, Anderson J, Nurse C, Watt M, Yu M, Juethner S. Long-term prevention of hereditary angioedema attacks with lanadelumab in adolescents. Ann Allergy Asthma Immunol. 2024 Dec;133(6):712-719.e1. doi: 10.1016/j.anai.2024.08.001. Epub 2024 Aug 10.

    PubMed 39128590

  • Lumry WR, Maurer M, Weller K, Riedl MA, Watt M, Yu M, Devercelli G, Meunier J, Banerji A; HELP OLE Study Group. Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2023 Jul;131(1):101-108.e3. doi: 10.1016/j.anai.2023.03.028. Epub 2023 Apr 5.

    PubMed 37028510

  • Craig TJ, Zaragoza-Urdaz RH, Li HH, Yu M, Ren H, Juethner S, Anderson J; HELP and HELP OLE Study Investigators. Effectiveness and safety of lanadelumab in ethnic and racial minority subgroups of patients with hereditary angioedema: results from phase 3 studies. Allergy Asthma Clin Immunol. 2022 Sep 24;18(1):85. doi: 10.1186/s13223-022-00721-y.

    PubMed 36153561

  • Banerji A, Bernstein JA, Johnston DT, Lumry WR, Magerl M, Maurer M, Martinez-Saguer I, Zanichelli A, Hao J, Inhaber N, Yu M, Riedl MA; HELP OLE Investigators. Long-term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study. Allergy. 2022 Mar;77(3):979-990. doi: 10.1111/all.15011. Epub 2021 Aug 13.

    PubMed 34287942

  • Riedl MA, Bernstein JA, Craig T, Banerji A, Magerl M, Cicardi M, Longhurst HJ, Shennak MM, Yang WH, Schranz J, Baptista J, Busse PJ. An open-label study to evaluate the long-term safety and efficacy of lanadelumab for prevention of attacks in hereditary angioedema: design of the HELP study extension. Clin Transl Allergy. 2017 Oct 6;7:36. doi: 10.1186/s13601-017-0172-9. eCollection 2017.

    PubMed 29043014

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing DX-2930 in people with Hereditary Angioedema (HAE), run by Shire at 43 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 43 sites in United States, Canada, Germany, Italy, Jordan and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 May 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: From start of the study up to follow-up (Day 952)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02741596, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2021.

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