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A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03181633
Recruitment Status : Active, not recruiting
First Posted : June 9, 2017
Last Update Posted : August 20, 2019
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE June 5, 2017
First Posted Date  ICMJE June 9, 2017
Last Update Posted Date August 20, 2019
Actual Study Start Date  ICMJE June 22, 2017
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 7, 2017)
  • Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), and AEs leading to discontinuation [ Time Frame: Through study completion, an average of 2 years ]
  • Serum lactate dehydrogenase (LDH) levels [ Time Frame: Through study completion, an average of 2 years ]
  • Hemoglobin (Hgb) levels [ Time Frame: Through study completion, an average of 2 years ]
  • Number of red blood cell (RBC) transfusions [ Time Frame: Through study completion, an average of 2 years ]
  • Reticulocyte counts [ Time Frame: Through study completion, an average of 2 years ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 7, 2017)
  • FACIT Fatigue Scale scores [ Time Frame: Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years ]
  • EORTC QLQ-C30 scores [ Time Frame: Week 1, Week 13, Week 37, and then every 6 months through study completion, an average of 2 years ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official Title  ICMJE An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100
Brief Summary The purpose of this study is to evaluate long term safety and efficacy of ACH-0144471 in patients with PNH who have demonstrated clinical benefit from ACH-0144471 in Clinical Study ACH471-100. This study is designed to include up to 12 patients.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Paroxysmal Nocturnal Hemoglobinuria
Intervention  ICMJE Drug: ACH-0144471
ACH-0144471 will be administered to all patients enrolled in the study.
Study Arms  ICMJE Experimental: ACH-0144471
All patients will receive ACH-0144471 during the treatment period.
Intervention: Drug: ACH-0144471
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: August 19, 2019)
8
Original Estimated Enrollment  ICMJE
 (submitted: June 7, 2017)
12
Estimated Study Completion Date  ICMJE December 30, 2020
Estimated Primary Completion Date December 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Study designed to include up to 12 patients who completed treatment in study ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant safety or tolerability concerns.
  • Negative pregnancy test for females prior to dosing and throughout the study.

Exclusion Criteria:

  • Have developed any clinically relevant co-morbidities while participating in study ACH471-100 that would make the patient inappropriate for continuation of treatment with ACH-0144471, in the opinion of the investigator.
  • Have developed any clinically significant laboratory abnormalities while participating in study ACH471-100 that, in the opinion of the investigator, would make the patient inappropriate for the study or put the patient at undue risk.
  • Females who are pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or patients with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE New Zealand
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03181633
Other Study ID Numbers  ICMJE ACH471-103
2017-000665-79 ( EudraCT Number )
U1111-1196-0653 ( Other Identifier: UTN )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alexion Pharmaceuticals
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP