A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100

Phase 1

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-000665-79-GB
• Lead Sponsor: Achillion Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-04-27
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 12

Inclusion Criteria

1. Patients must have completed treatment in study ACH471-100 and must have demonstrated a clinical benefit from ACH-0144471, in the opinion of the PI, with no significant safety or tolerability concerns.
2. Female participants of childbearing potential must agree to use an acceptable method of contraception (as defined in Section 5.5.4) from the date of signing the informed consent to the first day of dosing (Day 1), and must agree to use a highly effective method of contraception (as defined in Section 5.5.4) from the first day of dosing to 30 days after their last dose of study drug. Female participants of childbearing potential must also have a negative serum pregnancy test during Screening and negative urine pregnancy test on Day 1.
Female participants of non-childbearing potential need not employ a method of contraception.
3. Non-sterile male participants must agree to use a highly effective method of contraception (as defined in Section 5.5.4) with their partner(s) of childbearing potential from the first day of dosing to 90 days after their last dose of study drug.
Males who are surgically sterile need not employ additional contraception.
Males must agree not to donate sperm while enrolled in this study and for 90 days after their last dose of study drug.
4. Must agree to provide written informed consent.
5. Must be willing, at all times, to have transportation and telephone access, and to be within one hour of an emergency medical center
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Have developed any clinically relevant co-morbidities while participating in ACH471-100 that would make the patient inappropriate for continuation of treatment with ACH-0144471, in the opinion of the investigator.
2. Have developed a clinically significant laboratory abnormality while participating in ACH471-100 that, in the opinion of the investigator, would make the patient inappropriate for the study or put the patient at undue risk.
3. 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified