Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome
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- STATUS
- Recruiting
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- End date
- Dec 31, 2024
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- participants needed
- 3
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- sponsor
- Kunming Hope of Health Hospital
Summary
It is a single-center, open, single-arm, non-randomized investigator-initiated trial evaluating the efficacy and safety of recombinant adeno-associated virus administration for patients with Menkes syndrome.
Description
Who can participate? Patients with Menkes syndrome were diagnosed by combining clinical and genetic diagnoses.
How to conduct this study? In this study, the constructed recombinant adeno-associated virus preparation will be delivered to patients with Menkes syndrome. After administration, close follow-up will be conducted for at least three months, followed by regular follow-up until at least the 12th month. The efficacy and safety of treatment will be observed and evaluated. After the completion of the study, participants will decide whether to participate in the long-term follow-up program for five years.
What are the possible benefits and risks of participating? Benefits: This study may prolong the life span and improve the prognosis of Menkes syndrome subjects. The information obtained from this study will help determine what treatment may be safe and effective for other subjects with similar conditions.
Risks: Subjects may have adverse reactions to the treatment. These adverse reactions may include abnormal liver injury, fever, thrombocytopenia, thrombotic microangiopathy, and possibly others.
Where is the study run? Kunming Hope of Health Hospital.
Details
| Condition | Menkes Syndrome |
|---|---|
| Treatment | Recombinant adeno-associated virus administration |
| Clinical Study Identifier | NCT05507996 |
| Sponsor | Kunming Hope of Health Hospital |
| Last Modified on | 4 October 2022 |
How to participate?
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn moreIf you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn moreComplete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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