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

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ClinicalTrials.gov Identifier: NCT03181633
Recruitment Status : Recruiting
First Posted : June 9, 2017
Last Update Posted : June 4, 2018
Sponsor:
Information provided by (Responsible Party):
Achillion Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: ACH-0144471 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100
Actual Study Start Date : June 22, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
Experimental: ACH-0144471
All patients will receive ACH-0144471 during the treatment period.
Drug: ACH-0144471
ACH-0144471 will be administered to all patients enrolled in the study.




Primary Outcome Measures :
  1. 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 ]
  2. Serum lactate dehydrogenase (LDH) levels [ Time Frame: Through study completion, an average of 2 years ]
  3. Hemoglobin (Hgb) levels [ Time Frame: Through study completion, an average of 2 years ]
  4. Number of red blood cell (RBC) transfusions [ Time Frame: Through study completion, an average of 2 years ]
  5. Reticulocyte counts [ Time Frame: Through study completion, an average of 2 years ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03181633


Contacts
Contact: Clinical Operations +1 203-752-5577 PNHTrialInquiries@achillion.com

Locations
New Zealand
Auckland Clinical Studies Limited Recruiting
Auckland, New Zealand
Contact       Fauna_Extn@clinicalstudies.co.nz   
Sponsors and Collaborators
Achillion Pharmaceuticals

Responsible Party: Achillion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03181633     History of Changes
Other Study ID Numbers: ACH471-103
2017-000665-79 ( EudraCT Number )
U1111-1196-0653 ( Other Identifier: UTN )
First Posted: June 9, 2017    Key Record Dates
Last Update Posted: June 4, 2018
Last Verified: June 2018

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

Keywords provided by Achillion Pharmaceuticals:
PNH
Paroxysmal
Hemoglobinuria
ACH-0144471

Additional relevant MeSH terms:
Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases