A Phase 2 interventional study of Cemdisiran in Atypical Hemolytic Uremic Syndrome, sponsored by Alnylam Pharmaceuticals. Withdrawn at 12 sites in 10 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-10-01.
Sponsored by Alnylam Pharmaceuticals · Phase 2, Interventional, and Treatment
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
45 studies on the registry are indexed under Atypical Hemolytic Uremic Syndrome; 14 are open to participants now.
Browse Atypical Hemolytic Uremic Syndrome studies →Alnylam Pharmaceuticals is the lead sponsor of 84 studies on the registry; 19 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 15 (83%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: Cemdisiran
Subcutaneous (sc) injection of Cemdisiran
The effect of Cemdisiran on platelet count
Time frame: Week 32
The effect of Cemdisiran on hematological response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on serum creatinine levels
Time frame: up to 84 weeks
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
Time frame: up to 84 weeks
The effect of Cemdisiran on adverse events (AEs)
Time frame: up to 108 weeks
Plan to share: Undecided
No publications or documents are linked to this record.
Ran from Sep 2017 to primary completion in Sep 2018, 11 months.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in Sep 2018. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Cemdisiran
Atypical Hemolytic Uremic Syndrome →