Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects

NCT02663687 | Completed Phase 1 Results

• 1 other identifier: SHP623-100
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers. This study will also collect pharmacokinetic data (how the body absorbs and breaks down the study drug).

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs)

  An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. An AE was considered to be a TEAE in a specific treatment period of the study if the date and time of onset were after investigational product administration in that period and if it occurred less than equals to (\<=) Day 28 and was both not present at the start of that period and was not a chronic condition that was part of the participant's medical history, or it was present at the start of that period or as part of the participant's medical history but the severity or frequency increased during that period \<= Day 28. An SAE was defined as any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose.

  From the start of study treatment up to 28 days after the last dose of the study treatment (up to 56 days)

Secondary Outcomes (10)

• Maximum Observed Plasma Concentration (Cmax) of SHP623 Occurring at Time of Maximum Observed Concentration During a Dosing Interval (Tmax)

  Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

• Time of Maximum Plasma Concentration (Tmax) of SHP623 Sampled During a Dosing Interval

  Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

• Terminal Half-life (t1/2) of SHP623

  Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

• Area Under the Plasma Concentration Curve From Time Zero to Infinity (AUC 0-inf) of SHP623

  Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

• Area Under the Plasma Concentration Curve From Time Zero to 168 Hours Postdose (AUC 0-168) of SHP623

  Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

Study Arms (2)

Treatment 1- 4

EXPERIMENTAL

Treatment A: 9 Subjects will receive dose level I of SHP623 intravenously (IV). B: 9 Subjects will receive dose level I of SHP623 subcutaneously(SC).

Drug: Recombinant human C1 esterase inhibitor; Drug: SHP623

Placebo

PLACEBO COMPARATOR

3 Subjects will receive placebo for each cohort

Drug: Placebo

Interventions

Recombinant human C1 esterase inhibitor DRUG

Subjects will receive escalating doses I-IV as both IV and SC injections

Also known as: SHP623

Treatment 1- 4

Placebo DRUG

Subjects will receive matching placebo

Placebo

SHP623 DRUG

SHP623

Treatment 1- 4

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Must be considered healthy. Healthy status is defined by absence of evidence of any active or chronic disease
• Male, or non-pregnant, non-lactating female, who agrees to comply with any applicable contraceptive requirements of the protocol, or females of non-child-bearing potential.
• Hemoglobin ≥12.0g/ld.

You may not qualify if:

• Have a history of allergic reaction to C1 INH products (e.g. C1 Inhibitor \[Human\], Berinert \[C1 Estrace Inhibitor (Human)\] and C1 estrace \[recombinant\]
• Known history of alcohol or other substance abuse within the last year.
• Donation of blood or blood products within 60 days prior to receiving investigational product.
• Current use of any medication except hormonal replacement therapy, hormonal contraceptives and occasional use of any over-the-counter non-steroidal anti-inflammatory drug (NSAID) or acetaminophen.
• Have a history of hypercoagulability or other predisposition to thrombotic events.

Sponsors & Collaborators

• Shire: lead

Study Sites (1)

Clinical Pharmacology of Miami

Miami, Florida, 33014, United States

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

conestat alfa

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2015
• First Posted: January 26, 2016
• Study Start: February 19, 2016
• Primary Completion: December 5, 2016
• Study Completion: December 5, 2016
• Last Updated: June 3, 2021
• Results First Posted: September 6, 2018

Record last verified: 2021-05

Locations

US (1)