Clinical trial NCT03401112: A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Official title: A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: Cardurion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Imara, Inc.
First posted: 2018-01-17
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).

Detailed description: This is a proof-of-concept study in adult SCA participants, ages 18 to 55 years old, to examine the safety, tolerability, and pharmacokinetic (PK), as well as the potential pharmacodynamic (PD) effects and clinical efficacy, of IMR-687 across a range of doses.

IMR-687 was administered in 2 populations of participants with SCA: those who were not receiving hydroxyurea (HU) and those who were receiving a stable dose of HU according to standard of care.

Eligibility criteria: Key Inclusion Criteria:

* Male or female participants with confirmed SCA
* Age 18 to 55 years, inclusive
* For participants on HU, must have been on a stable dose for at least 60 days prior to screening

Key Exclusion Criteria:

* Total hemoglobin \>12.5 or \<6 grams/deciliter
* Red blood cell transfusion within 60 days of baseline
* \>7 hospitalizations for vaso-occlusive crises (VOCs) within the last year
* Estimated glomerular filtration rate \<50 milliliter/minute
* Aspartate aminotransferase/alanine aminotransferase \>3x the upper limit of normal

Primary outcomes:
measure: Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs)
timeFrame: Day 1 (after dosing) through up to Week 24
Secondary outcomes:
measure: Pharmacokinetic (PK) Of Participants Who Did Not Concomitantly Receive HU: Maximum Plasma Concentration (Cmax) Of IMR-687
timeFrame: Day 1 and Week 25
measure: PK Of Participants Who Did Not Concomitantly Receive HU: Area Under The Concentration-time Curve (AUC) From Time 0 To 24 Hours Postdose (AUC0-24h) Of IMR-687
timeFrame: Day 1 and Week 25
measure: PK Of Participants Who Concomitantly Received HU: Cmax Of IMR-687
timeFrame: Day 1 and Week 17
measure: PK Of Participants Who Concomitantly Received HU: AUC0-24h Of IMR-687
timeFrame: Day 1 and Week 17
measure: PK Of Participants Who Concomitantly Received HU: Cmax Of HU
timeFrame: Baseline (1 and 2) and Week 17
measure: PK Of Participants Who Concomitantly Received HU: AUC0-24h Of HU
timeFrame: Baseline (1 and 2) and Week 17
Other outcomes:
measure: Change From Baseline In F-Cells
timeFrame: Baseline, EOT (Week 25 for participants without HU and Weeks 17 or 25 for participants with HU)
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Farmington
country: United States
facility: University of Connecticut Health Center
state: Connecticut
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Chicago
country: United States
facility: University of Illinois
state: Illinois
city: Chicago
country: United States
facility: Loretto Hospital
state: Illinois
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Temple
country: United States
facility: Baylor Scott & White Health
state: Texas
city: Birmingham
country: United Kingdom
facility: Sandwell & West Birmingham Hospital
city: Bristol
country: United Kingdom
facility: Bristol Haematology and Oncology Centre
city: London
country: United Kingdom
facility: Royal London Hospital
city: London
country: United Kingdom
facility: University College London Hospital
city: London
country: United Kingdom
facility: Guy's Hospital
city: Oxford
country: United Kingdom
facility: Oxford Cancer & Haematology Centre, The Churchill Hospital
Study officials:
affiliation: Cardurion Pharmaceuticals
name: Regulatory Operations
role: STUDY_DIRECTOR
References: