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ecallantide

Phase 3

Hereditary Angioedema (HAE) | Small molecule | Immunology |Takeda Pharmaceutical Company Limited|Last Updated: Jun 11, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment334

FDA Designations

No designations recorded

Clinical trial landscape

ecallantide · 3 trials · 1 indication

Phase 3 3
NCT00456508Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)Hereditary Angioedema (HAE)
COMPLETED147 Analytics
NCT00457015Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)Hereditary Angioedema (HAE)
COMPLETED96 Analytics
NCT00262080Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)Hereditary Angioedema (HAE)
COMPLETED91 Analytics
PHASE3COMPLETED
Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)Unlock trial analytics
PHASE3COMPLETED
Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)Unlock trial analytics
PHASE3COMPLETED
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)Unlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hrs Post Dosing
4 hrs post dose after every episode

Mean Symptom Complex Severity (MSCS) score is a validated point-in-time measure of symptom severity. At baseline and 4 hrs, patients rated the severity on a categorical scale (0=normal, 1=mild, 2=moderate, 3=severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose
baseline, 4 hours post-dose

The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.

Treatment Outcome Score at 4 Hours Post-Dose
4 hours post-dose (DOUBLE-BLIND PART)

Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.

Secondary Endpoints

Treatment Outcome Score (TOS) at 4 Hrs Post Dosing, Based on the Patient Assessment of Baseline Severity of Symptoms
4 hrs post dose after every episode
Time to Significant Improvement
15 min - 4 hrs post dose after every episode
Treatment Outcome Score at 4 Hours Post-Dose
4 hours post-dose
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
DX-88 (ecallantide)EXPERIMENTALDX-88 (ecallantide) Patients were treated with DX-88 (ecallantide) when they experienced an HAE attack. 30 mg dose of ecallantide given via 3 SC injections; a second 30 mg dose can be administered if needed. Patients were to be assessed until 4 hrs post-dose. Patients were asked to return for 3 follow-up visits: 7 days, 28 days and 90 days post-dose.
PlaceboPLACEBO_COMPARATORPlacebo, Phosphate Buffer Saline (PBS), pH 7.0 given as 3 subcutaneous injections.

Interventions

NameTypeDescription
ecallantideDRUGsolution for SC injection, one 30 mg dose per HAE attack
Phosphate Buffer Saline (PBS), pH 7.0DRUGgiven as three 1mL subcutaneous injections.
Phosphate Buffer Saline (PBS),DRUGgiven as three 1mL subcutaneous injections.
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Eligibility Criteria

Age Range10 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria: * 10 years of age or older * Documented diagnosis of HAE (Type I or II) * Willing and able to give informed consent * Acute HAE attack at time of presentation Exclusion Criteria: * Receipt of an investigational drug or device, within 30 days prior to study treatment, other tha...

Countries:United StatesCanadaJordan
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Competitive Landscape -Hereditary Angioedema 9 trials (matched to "Hereditary Angioedema (HAE)")

Frequently asked questions about ecallantide

What is ecallantide used for?

Ecallantide is used for the treatment of acute attacks of Hereditary Angioedema (HAE), a rare genetic condition characterized by recurrent episodes of severe swelling. It is an investigational small molecule being developed by Takeda Pharmaceutical Company Limited (TAK) and is currently in Phase 3 clinical development.

What does ecallantide target?

Ecallantide is a small molecule that targets the plasma kallikrein pathway, which plays a central role in the generation of bradykinin, a key mediator of swelling in Hereditary Angioedema. By inhibiting this pathway, ecallantide aims to reduce the severity and duration of acute HAE attacks.

Who makes ecallantide?

Ecallantide is being developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol TAK. The drug is currently in Phase 3 clinical trials for the treatment of Hereditary Angioedema.

What phase is ecallantide in?

Ecallantide is in Phase 3 clinical development for the treatment of Hereditary Angioedema (HAE). It is an investigational drug and has not yet been approved by regulatory authorities. Three Phase 3 trials have been completed, with a total of 334 patients enrolled across studies.

What clinical trials is ecallantide in?

Ecallantide has completed three Phase 3 clinical trials: NCT00262080, NCT00456508, and NCT00457015. These randomized, double-blind, placebo-controlled studies evaluated the efficacy and safety of ecallantide in treating acute attacks of Hereditary Angioedema in patients aged 10 years and older across the United States, Canada, and Jordan.

Is ecallantide the same as DX-88?

Yes, ecallantide is also known as DX-88. The clinical trials NCT00262080, NCT00456508, and NCT00457015 refer to the drug as DX-88 (ecallantide) in their titles, confirming that these names refer to the same investigational therapy for Hereditary Angioedema.